...MS TOOLKIT... The Latest in Research and Treatment

Merck KGaA: Oral Investigational Treatment Cladribine Tablets for Multiple Sclerosis Significantly Reduced Relapse Rate in Phase III Pivotal Trial

2009-01-26T10:16:00.006-07:00

• Two-year primary efficacy endpoint of CLARITY trial met: 58% relative
reduction in annualized relapse rate in the low total dose treatment group
and 55% in the high total dose treatment group

• Submission for registration of cladribine tablets planned for mid-2009

• Cladribine tablets are the first oral investigational multiple sclerosis
treatment to complete a two-year pivotal study

Darmstadt, Germany, January 23, 2009 – Merck KGaA and its Merck Serono division
announced today that the CLARITY1 Phase III pivotal trial of its proprietary oral
formulation of cladribine (cladribine tablets) met the two-year primary endpoint of
clinical relapse rate reduction in patients with relapsing-remitting multiple sclerosis
(MS).

The two cladribine tablet treatment groups of the study, assessing different dose
regimens, demonstrated a statistically significant reduction in the annualized rate of
relapses compared to placebo. Patients from the lower total dose group experienced a
58% relative reduction in annualized relapse rates with respect to placebo (0.14 versus
0.33 for the placebo group; p<0.001). Patients from the higher total dose group
experienced a 55% relative reduction in annualized relapse rates with respect to
placebo (0.15 versus 0.33; p<0.001).

Overall, the frequencies of adverse events were low in the cladribine tablet treatment
groups and were comparable to that observed in the placebo group. Lymphopenia, an expected event based on the presumed mechanism of action of cladribine, occurred
more frequently in the cladribine tablet treatment groups. With the exception of
lymphopenia, the most frequently reported adverse events in the three study groups
were headaches and nasopharyngitis.

“We believe the CLARITY data mark an important milestone in the assessment of
investigational oral treatments for multiple sclerosis and that cladribine tablets have the potential to make a real difference in the lives of patients,” said Elmar Schnee, Executive Board Member with responsibility for the Pharmaceuticals business sector.

“Based on the successful completion of the CLARITY study, we plan to submit cladribine tablets for registration to the EMEA and to the FDA for mid-2009."

Secondary endpoints of the CLARITY study were also met, including reduction of lesion activity as measured by magnetic resonance imaging (MRI), proportion of subjects relapse-free and disability progression. Full study results will be submitted for
presentation at an upcoming scientific meeting.

The CLARITY study was a two-year (96 weeks), randomized, double-blind, placebo controlled, international trial. It enrolled 1,326 patients with relapsing-remitting MS
according to the revised McDonald criteria2. Study participants were randomized to one
of three different treatment groups consisting of two different dose regimens of
cladribine tablets or matching placebo tablets (1:1:1 ratio). Cladribine tablets were
given in two or four treatment courses in the first year, with each course consisting of once daily administration for four to five consecutive days, which means study patients took cladribine tablets for only 8 to 20 days during the year. In the second year, two treatment courses were administered to all patient groups. The primary endpoint of the CLARITY study was the qualifying relapse rate at 96 weeks. Secondary endpoints included MRI endpoints3, proportion of subjects relapse-free and disability progression at 96 weeks. Out of the 1,326 randomized patients, 90% of patients treated with cladribine tablets completed the study (92% in the lower total dose group and 89% in the higher total dose group) compared to 87% in the placebo group.

1 CLARITY: CLAdRIbine Tablets Treating MS OrallY

2 The McDonald criteria are diagnostic criteria for MS. In April 2001 an international panel in association with the National Multiple Sclerosis Society (NMSS) of America recommended revised diagnostic criteria for MS. They make use of advances in MRI imaging techniques and are intended to replace the Poser criteria. The new criteria facilitate the diagnosis of MS in patients who present with signs and symptoms
suggestive of the disease. The McDonald criteria for the diagnosis of multiple sclerosis were revised in 2005 to simplify and speed diagnosis, while maintaining adequate sensitivity and specificity.

3 The exact correlation between MRI findings and the current or future clinical status of patients, including disability progression, is unknown.

About cladribine tablets

Merck Serono’s proprietary oral formulation of cladribine (cladribine tablets) is currently being evaluated in Phase III as a treatment for patients with relapsing forms of multiple sclerosis (MS). Cladribine is a small molecule that may interfere with the behavior and the proliferation of certain white blood cells, particularly
lymphocytes, which are thought to be involved in the pathological process of MS.
The clinical development program for cladribine tablets includes:

- The CLARITY (CLAdRIbine Tablets Treating MS OrallY) extension study: a two-year placebo-controlled extension of the CLARITY study, designed to provide data on the long-term safety and efficacy of extended administration of cladribine tablets for up to four years

- The ORACLE MS (ORAl CLadribine in Early MS) study: a two-year Phase III placebo-controlled trial designed to evaluate the efficacy and safety of cladribine tablets as a monotherapy in patients at risk of developing MS (patients who have experienced a first clinical event suggestive of MS). This trial was announced in September 2008.

- The ONWARD (Oral Cladribine Added ON To Rebif New Formulation in Patients With Active Relapsing Disease) study: a Phase II placebo-controlled trial designed primarily to evaluate the safety and tolerability of adding cladribine tablets treatment to patients with relapsing forms of MS, who have experienced breakthrough disease while on established interferon-beta therapy. This trial was announced in January 2007.

Cladribine tablets have been granted a fast track designation by the US Food and Drug Administration based on the need for an oral therapy in a subset of patients with relapsing forms of multiple sclerosis.

About Merck Serono and multiple sclerosis

Merck Serono is a leader in multiple sclerosis (MS) with Rebif® (interferon beta-1a), a disease-modifying drug used to treat relapsing forms of MS, which is registered in more than 80 countries worldwide. Full prescribing information for this product can be obtained by contacting the Company or visiting its website.

Additional therapeutic options are currently under development at Merck Serono, including cladribine tablets, currently in Phase III and potentially the first oral therapy for MS, as well as several products in early stage development. Merck Serono also is taking a leading role in developing an understanding of the role of genetics in MS.

About multiple sclerosis

Multiple sclerosis (MS) is a chronic, inflammatory condition of the nervous system and is the most common, non-traumatic, disabling neurological disease in young adults. The World Health Organization estimates that up to 2.5 million people suffer from MS worldwide. While symptoms can vary, the most common symptoms of MS include blurred vision, numbness or tingling in the limbs and problems with strength and coordination. The relapsing forms of MS are the most common.

All Merck Press Releases are distributed by e-mail at the same time they become available on the Merck Website. Please go to http://www.subscribe.merck.de to register online, change your selection or discontinue this service.

Merck is a global pharmaceutical and chemical company with total revenues of € 7.1 billion in 2007, a history that began in 1668, and a future shaped by 32,458 employees in 59 countries. Its success is characterized by innovations from entrepreneurial employees. Merck's operating activities come under the umbrella of Merck KGaA, in which the Merck family holds an approximately 70% interest and free shareholders own the remaining approximately 30%. In 1917 the U.S. subsidiary Merck & Co. was expropriated and has been an independent company ever since.

Merck KGaA
Frankfurter Strasse 250
64293 Darmstadt
Hotline +49 (0) 6151 72-5000
www.merck.de

Head External Communications -2386
Spokesmen: -9591 / -7144 / -6328
Fax +49 (0) 6151 72-7707
media.relations@merck.de

Novartis launches Extavia®, a new therapeutic option to help patients combat devastating symptoms of multiple sclerosis

2009-01-26T10:05:00.005-07:00

# Multiple sclerosis (MS) causes progressive disability and affects 2.5 million people worldwide including many young adults[1]

# Extavia offers patients and physicians a new branded version of standard-of-care interferon beta-1b

# Approved to treat MS patients from first signs of active disease to more advanced, relapsing forms

# Launch marks start of planned long-term partnership between Novartis and MS community

Basel, January 22, 2009 - Novartis has today announced the launch of Extavia®, a new version of the standard-of-care for relapsing forms of multiple sclerosis (MS), providing patients and physicians with an alternative option to help manage this devastating disease.

Extavia, a new branded version of interferon beta-1b, is available initially in Germany and Denmark with other European launches to follow during 2009. It is approved to treat a broad range of patients, from those with early signs of MS to those with more advanced relapsing forms of the disease.

"Extavia will provide patients and physicians with an additional option for receiving a mainstay of care in MS," said Trevor Mundel, MD, Global Head of Development at Novartis Pharma AG. "This important first step also opens the way for Novartis to build supportive partnerships with the MS community and lays the foundations for providing innovative approaches to MS care."

Extavia is the same medicinal product as Betaferon®*, an interferon beta-1b. This has a well characterised efficacy and safety profile with more than 700,000 patient-years' experience[2] and a 17-year track record of clinical use - the longest for any interferon beta in the treatment of MS[3].

MS is estimated to affect up to 2.5 million patients worldwide and is one of the leading causes of neurological disability in young adults1. The disease typically presents in relapsing forms involving acute self-limiting attacks of neurological dysfunction (or "relapses"), followed by complete or partial restoration of function.
Data have shown that interferon beta-1b produces a 34% reduction in annualized relapse rates (p<0.001), and patients are almost twice as likely to remain relapse-free for over two years compared to those on placebo (31% vs. 16%, p=0.007)[4]. Treatment with interferon beta-1b can also slow disease progression. After two years, nearly three-quarters of patients who had experienced a single episode of neurological disease lasting at least 24 hours did not progress to clinically definite MS[5].

The launch of Extavia in Europe by the Pharmaceuticals Division of Novartis marks the beginning of a long-term commitment to meet the therapeutic needs of the MS community. This will include the establishment of a support program for Extavia users that will foster cross-communication between patients and their physicians and nurses. In turn, this will lay the foundations for future potential innovations in MS therapy. The rollout of Extavia in key EU countries is expected during the coming months.

Novartis acquired the rights to its own branded version of interferon beta-1b in an agreement with Bayer Schering, the company that markets Betaferon. In backing Extavia, Novartis brings over 50 years of neuroscience expertise and resources to the MS community. This expertise has helped to pioneer early breakthrough treatments for a number of neurological and pathological conditions, some of which remain important therapies to this day.

MS is a chronic autoimmune disease of the central nervous system that causes inflammation and neurodegeneration. Pathology is characterised by the destruction of myelin, which helps neurons carry electrical signals in the brain[6]. The disease causes problems with muscle control and strength, vision, balance, sensation and mental function[6].

The beneficial effects of interferon beta are believed to be due to its modulation of the immune system to reduce inflammatory damage. Specifically, interferon beta limits the activation of immune cells that attack myelin, suppresses the production of inflammatory cytokines - a type of protein that amplifies the inflammatory response causing damage to myelin - and stimulates the production of anti-inflammatory cytokines.

Extavia has been filed with the US Food and Drug Administration for the treatment of relapsing forms of MS to reduce the frequency of clinical exacerbations (or relapses). Patients with MS in whom efficacy has been demonstrated include those who have experienced a first clinical episode and have features consistent with MS as shown by magnetic resonance imaging (MRI)[7].

Extavia is administered by subcutaneous (or under the skin) injection. Patients will have the choice of using either a fine (30 gauge) needle for manual injection or a convenient autoinjector.

* Betaferon® is a registered trademark of Bayer Schering Pharma AG.

Disclaimer

The foregoing release contains forward-looking statements that can be identified by terminology such as "launches," "planned," "long-term," "can," "will," "likely," "commitment," "future," "potential," "expected," "estimated," "believed," or similar expressions, or by express or implied discussions regarding potential additional marketing approvals for Extavia, the roll-out of Extavia in potential additional markets, the potential development of additional MS therapies, or regarding potential future revenues from Extavia or additional MS therapies. Such forward-looking statements reflect the current views of the Company regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that will be approved for sale in any additional markets. Nor can there be any guarantee that Extavia will be launched in any additional markets. Neither can there be any guarantees that Novartis will successfully develop and bring to market any additional MS therapies. Nor can there be any guarantee that Extavia or such additional therapies will achieve any particular levels of revenue in the future. In particular, management's expectations regarding Extavia and any such additional MS therapies could be affected by, among other things, unexpected regulatory actions or delays or government regulation generally; unexpected clinical trial results, including unexpected new clinical data and unexpected additional analysis of existing clinical data; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; competition in general; government, industry and general public pricing pressures, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis

Novartis AG provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2007, the Group's continuing operations (excluding divestments in 2007) achieved net sales of USD 38.1 billion and net income of USD 6.5 billion. Approximately USD 6.4 billion was invested in R&D activities throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 97,000 full-time associates and operate in over 140 countries around the world. For more information, please visit http://www.novartis.com.

References

[1] World Health Organization. Neurology atlas, 2004. Accessed 16 Jan 2009. http://www.who.int/mental_health/neurology/neurogy_atlas_review_references.pdf
[2] FDA approves Betaseron® for use after the first event suggestive of multiple sclerosis [press release]. Wayne, NJ: Berlex: 23 October 2006.
[3] Ebers G, Traboulsee A, Langdon D, Goodin D, Konieczny A. The interferon beta-1b 16-year long-term follow-up study: the results. Presented at the 16th meeting of the European Neurological Society; 27-31 May, 2006.
[4] The IFNB Multiple Sclerosis Study Group. Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. Neurology. 1993;43:655-661.
[5] Kappos L, Freedman MS, Polman CH, et al. Effect of early versus delayed interferon beta-1b treatment on disability after a first clinical event suggestive of multiple sclerosis: a 3-year follow-up analysis of the BENEFIT study. Lancet. 2007;370:389-97.
[6] National Multiple Sclerosis Society website. http://www.nationalmssociety.org/about-multiple-sclerosis/symptoms/index.aspx. Accessed January 12, 2009.
[7] Extavia proposed US Prescribing Information.

# # #

Novartis Media Relations
Central media line: +41 61 324 2200

Eric Althoff
Novartis Global Media Relations
+41 61 324 7999 (direct)
+41 79 593 4202 (mobile)
eric.althoff@novartis.com

John Taylor
Novartis Pharma Communications
+41 61 324 6715 (direct)
+41 79 593 4279 (mobile)
john.taylor@novartis.com
e-mail: media.relations@novartis.com

Novartis Investor Relations
Central phone: +41 61 324 7944

Ruth Metzler-Arnold +41 61 324 9980

North America:

Pierre-Michel Bringer +41 61 324 1065

Richard Jarvis +1 212 830 2433

John Gilardi +41 61 324 3018

Jill Pozarek +1 212 830 2445

Thomas Hungerbuehler +41 61 324 8425

Edwin Valeriano +1 212 830 2456

Isabella Zinck +41 61 324 7188

e-mail: investor.relations@novartis.com

Peptimmune Grants Major Pharmaceutical Company Exclusive Option to License PI-2301 for Multiple Sclerosis

2009-01-26T09:56:00.004-07:00

Cambridge, Mass., January 15, 2009 — Peptimmune, Inc. (“Peptimmune”), a privately held biotechnology company, announced today that it has granted Novartis (NYSE: NVS) an exclusive option to obtain exclusive worldwide rights to develop and commercialize PI-2301, Peptimmune’s multiple sclerosis drug candidate. In a separate but related agreement, the MPM Bio IV NVS Strategic Fund L.P. made an equity investment in Peptimmune.In the event that Novartis exercises the option to PI-2301, Novartis would assume the global clinical development, manufacturing, and marketing of PI-2301 and all associated costs. Peptimmune would receive payments upon the option exercise and upon successful completion of certain development, regulatory, and commercial milestones. These payments together could total more than $500 million. In addition, Peptimmune shall be eligible to receive royalties on product sales. Additional terms were not disclosed.“We believe that Novartis represents an outstanding future partner for Peptimmune and the global development of PI-2301. This option agreement provides Peptimmune with access to both Novartis’ world class development expertise and the necessary capital to invest in this proprietary product,” said Thomas P. Mathers, President and CEO of Peptimmune.Todd Foley, Managing Director at MPM Capital commented, “Peptimmune and its world-leading expertise in peptide copolymers represent an attractive investment opportunity.”

About PI-2301PI-2301 is a peptide copolymer developed using Peptimmune’s novel platform peptide chemistry. PI-2301 is designed to enhance the regulatory response of the immune system, thereby controlling the pathogenic autoimmune response in certain diseases. PI-2301 is currently in Phase 1b development by Peptimmune.

About Peptimmune

Peptimmune, Inc. is a privately held clinical stage biotechnology company focused on the development of peptide therapies to improve the management of chronic autoimmune and inflammatory disorders. The Company is in clinical development with second-generation therapeutics that are expected to result in safer and more effective products for multiple sclerosis and pemphigus vulgaris. Current investors include New Enterprise Associates, MPM Capital, Hunt Ventures, Boston Medical Investors, Silicon Valley Bank Capital, and Genzyme Corporation. For additional information, please contact us.

New Interferon Formulations Promise to Eliminate Injections in Multiple Sclerosis Treatment

2009-01-26T09:42:00.005-07:00

SAN DIEGO, CA January 12, 2009/MarketWire/-- Nerveda Inc. and Aegis Therapeutics
LLC today announced preclinical results from their joint collaboration aimed at
developing non-injectable formulations of the beta-interferons. The beta interferons,
beta-1a (tradename Rebif®), and beta 1b (tradenames Betaseron® and Betaferon®) are
closely related injectable protein drugs in the interferon family that are used to treat both the relapsing-remitting and secondary-progressive forms of multiple sclerosis (MS). The beta interferons are currently administered by subcutaneous injection and have been proven clinically to slow the advance of multiple sclerosis and reduce the frequency of attacks. Current worldwide combined annual sales of Rebif®, Betaseron® and Betaferon® are approximately $4 Billion.

Because proteins are large and fragile molecules, they cannot be administered orally
and are typically administered by injection. They are often subject to instability due to aggregation of the protein molecules – particularly upon storage and handling at nonrefrigerated temperatures. The resulting protein aggregates are more poorly absorbed
into the blood stream upon injection due to their increased size, and induce development of circulating antibodies to interferon in patients that reduce the effectiveness of the drug over time.

Leading medical scientists at Johns Hopkins University, expert in the treatment of
neurological diseases, in collaboration with Nerveda and Aegis have applied Aegis’
Intravail® transmucosal absorption enhancement, and ProTek® protein stabilization
technologies to address these problems and have demonstrated for the first time that the beta interferons can be administered intranasally to prevent nerve damage in preclinical animal models of multiple sclerosis. In addition, the new formulations were shown to reduce or eliminate the immunogenicity of Betaseron® and Rebif®, administered either by injection or intranasally, while substantially increasing stability in a stress test involving constant agitation at elevated temperatures for extended periods of time.

Dr. Edward Maggio, Ph.D., CEO of Aegis Therapeutics, who participated in the
research, said, “since interferons will continue to be the foundation of MS therapy, it is critical that non-invasive delivery options for patients be developed.” Maggio also indicated, “the reduction in immunogenicity and the increase in stability also address a significant unmet need of the currently available beta-interferon therapies.”
Nerveda plans to begin testing the new formulation in clinical trials in early 2009 in
collaboration with clinicians and scientists at John Hopkins University Medical Center
and other sites.

* Rebif® is a registered trademark of Pfizer, Inc.
* Betaseron® is a registered trademark of Bayer Healthcare Pharmaceuticals
* Betaferon® is a registered trademark of Bayer Schering Pharma AG
* Intravail® and ProTek® are registered trademarks of Aegis Therapeutics, LLC

About Nerveda Inc.

Nerveda is a privately funded specialty pharmaceutical and diagnostic company focused
on improving the quality of life for patients suffering from neurodegenerative diseases
and their caregivers. Nerveda supports the clinical development of products licensed
from Johns Hopkins University, including neuroprotective compounds and stem cell
therapeutics that show promise in treating auto-immune disorders.

About Aegis Therapeutics

Aegis Therapeutics LLC is a drug delivery technology company commercializing its
patented or proprietary drug delivery and drug formulation technologies through productspecific licenses. Our patented Intravail® drug delivery technology enables the noninvasive delivery of a broad range of protein, peptide and non-peptide macromolecular therapeutics that can currently only be administered by injection. Aegis’ Intravail® absorption enhancement agents provide exceptionally high and unmatched bioavailability performance, comparable in efficiency to subcutaneous injection, via the intranasal administration route. Intravail® has also been successfully applied to buccal, oral, and rectal administration of small molecule, peptide, and nucleotide-analog type drugs. Our patented ProTek® technology allows creation of proprietary, easily manufacturable, and stable aqueous or lyophilized dosage forms that maintain the integrity and physiological activity of many protein and peptide therapeutics. ProTek® technology is applicable to injectable, intranasal, and other dosage forms of peptide or protein therapeutics.

For more information about Aegis, please visit the Aegis website at: htpp://www.aegisthera.com.

Contact:
Aegis Therapeutics LLC
Ralph Barry, Chief Business Officer
1-858-618-1400 Ext. 102
Email: rbarry@aegisthera.com

Contact:
Nerveda Inc.
Cam Gallagher, CEO
1-(858) 705-2365
Email: CGallagher@NervedaBio.com

Apitope and Merck Serono Announce Licensing Agreement on Novel Peptide Therapeutics for the Treatment of Multiple Sclerosis

2009-01-26T09:36:00.006-07:00

Apitope and Merck Serono to collaborate on development and commercialization of ATX-MS-1467, Apitope’s peptide therapeutic for the treatment of multiple sclerosis (MS)

Bristol, England and Hasselt, Belgium, January 13, 2009 – Apitope Technology (Bristol) Ltd., a wholly owned subsidiary of Apitope International NV, announced today the signature of a research, development and commercialization agreement with Merck Serono, a division of Merck KGaA, Darmstadt, Germany. Under this agreement, Apitope has granted exclusive worldwide rights to Merck Serono to develop and commercialize Apitope’s product ATX-MS-1467. This peptide therapeutic has completed an initial clinical study in patients with MS. It is designed to induce immunological tolerance of the body’s T-cells to key autoantigens involved in the pathogenesis of MS.

ATX-MS-1467 is a novel peptide-based therapeutic derived from Apitope’s proprietary technology platform. Under the terms of the agreement, Apitope will receive an upfront payment and will initially be responsible for the further development of ATX-MS-1467, for which Merck Serono will fund the costs. Merck Serono will be responsible for all development activities from the beginning of Phase II clinical trials. Merck Serono will also provide committed funding to Apitope for research on other novel therapeutic peptides for the treatment of MS.

Under the terms of the agreement, Apitope is eligible to receive up to € 154 million in upfront, development and commercialization milestone payments, in addition to royalties on the net sales of products resulting from the collaboration.

“This partnership with Apitope strengthens our position as a leader in the field of innovative research and development in multiple sclerosis,” said Bernhard Kirschbaum, Executive Vice President Research and Development at Merck Serono. “ATX-MS-1467 represents a novel, targeted approach and may have the potential to complement existing MS drugs by offering a novel mode of action. By applying our existing stratified medicine approaches, we will also identify those MS patients who should benefit most from this potential treatment.”

“We are very pleased that ATX-MS-1467 has attracted a major pharmaceutical partner such as Merck Serono with extensive experience and leadership in the development of therapies for multiple sclerosis,” said Keith Martin, CEO of Apitope. “We view this collaboration as confirmation of Apitope's ability to develop early-stage first-in-class therapies for autoimmune diseases. In addition to continuing to build our in-house diagnostic platform in MS, we look forward to progressing ATX-MS-1467 with Merck Serono.”

ATX-MS-1467 consists of four short peptides that are derived from myelin basic protein, a key autoantigen in MS. It is specifically designed to target up to 70 % of MS patients who have a specific genetic profile.

About Apitope

Apitope International NV is a biopharmaceutical company with headquarters in Hasselt, Belgium, and a subsidiary in Bristol, England. The Company is developing novel products to revolutionize the diagnosis and treatment of chronic autoimmune and allergic disorders. Apitope’s therapeutic peptide technology platform is based on established scientific evidence showing that soluble, synthetic peptides can reinstate tolerance and selectively attenuate pathological immune responses. The ApitopesTM (Antigen Processing Independent epiTOPES) inhibit the immune system's harmful attack on the body while preserving normal immune responses to harmful antigens, such as infections. Apitope’s portfolio includes novel peptide therapies for MS as well as other autoimmune diseases and common allergies. Apitope is also developing novel diagnostic products for the early detection of autoimmune diseases such as MS and rheumatoid arthritis (RA). Apitope is backed by LRM; Vesalius Biocapital; Fast Forward, VINNOF; Hasselt University; The Wellcome Trust; the Daniels family, Wyvern Seedcorn Fund and the University of Bristol; Innovator Capital Limited advised on the recent funding rounds.

For more information, please go to www.apitope.com

About multiple sclerosis

Multiple sclerosis (MS) is a chronic, inflammatory condition of the nervous system and is the most common, non-traumatic, disabling neurological disease in young adults. The World Health Organization estimates that up to 2.5 million people suffer from MS worldwide. While symptoms can vary, the most common symptoms of MS include blurred vision, numbness or tingling in the limbs and problems with strength and coordination. The relapsing forms of MS are the most common.

About Merck Serono and multiple sclerosis

Merck Serono is a leader in multiple sclerosis (MS) with Rebif® (interferon beta-1a), a disease-modifying drug used to treat relapsing forms of MS, which is registered in more than 80 countries worldwide. Full prescribing information for this product can be obtained by contacting the Company or visiting its website. Additional therapeutic options are currently under development at Merck Serono, including oral cladribine, currently in Phase III and potentially the first oral therapy for MS, as well as several products in early stage development. Merck Serono is also taking a leading role in developing an understanding of the role of genetics in MS.

About Merck Serono

Merck Serono is the division for innovative prescription pharmaceuticals of Merck, a global pharmaceutical and chemical group. Headquartered in Geneva, Switzerland, Merck Serono discovers, develops, manufactures and markets innovative small molecules and biopharmaceuticals to help patients with unmet medical needs. Its North American business operates in the United States and Canada as EMD Serono.

Merck Serono has leading brands serving patients with cancer (Erbitux®), multiple sclerosis (Rebif®), infertility (Gonal-f®), endocrine and cardiometabolic disorders (Glucophage®, Concor®, Euthyrox®, Saizen®, Serostim®), as well as psoriasis (Raptiva®).

With an annual R&D expenditure of around € 1bn, Merck Serono is committed to growing its business in specialist-focused therapeutic areas including neurodegenerative diseases, oncology, fertility and endocrinology, as well as new areas potentially arising out of research and development in autoimmune and inflammatory diseases.

About Merck

Merck is a global pharmaceutical and chemical company with total revenues of € 7.1 billion in 2007, a history that began in 1668, and a future shaped by 32,458 employees in 59 countries. Its success is characterized by innovations from entrepreneurial employees. Merck's operating activities come under the umbrella of Merck KGaA, in which the Merck family holds an approximately 70% interest and free shareholders own the remaining approximately 30%. In 1917 the U.S. subsidiary Merck & Co. was expropriated and has been an independent company ever since.

For more information, please visit www.merckserono.net or www.merck.de

Acorda expects to seek approval for drug

2009-01-26T09:32:00.001-07:00

January 12, 2009

Acorda Therapeutics Inc., a Hawthorne-based biotech company, said that it is pushing ahead with plans to seek regulatory approval for its most promising product, Fampridine-SR, a drug to help people with multiple sclerosis walk.

The company said that it remains on schedule to file a new drug application for the medication with the U.S. Food and Drug Administration during the first quarter. It also expects to submit applications for the medication to regulators in Europe and Canada.

“We are also beginning discussions with potential marketing partners to explore commercialization options in Europe and the rest of world excluding the U.S.,” Chief Executive Officer Ron Cohen said in a written statement. “Achieving these milestones will bring us closer to our goal of making Fampridine-SR available to the many people with MS who may benefit from it.”

MS Relapse-Rate Higher in Adolescent-Onset Patients

2009-01-26T09:23:00.003-07:00

By Michael Smith, North American Correspondent, MedPage Today
Published: January 12, 2009

Reviewed by Zalman S. Agus, MD; Emeritus Professor
University of Pennsylvania School of Medicine.

BOSTON, Jan. 12 -- When multiple sclerosis symptoms start in adolescence, the later annual relapse rate is nearly three times as great as it is for adult-onset disease, researchers here said.

The finding suggests that adolescent-onset MS has a more inflammatory disease course, according to Tanuja Chitnis, M.D., of Massachusetts General Hospital, and colleagues.

But the biological basis of the difference remains unclear and requires more study, Dr. Chitnis and colleagues said in the January issue of Archives of Neurology.

Several studies have shown that initial disease progression is slower in patients whose disease begins before the age of 18, the researchers said, but have differed on rates of relapse.

To clarify the issue -- and see whether the slower progression is related to relapse rates -- Dr. Chitnis and colleagues studied patients treated in the pediatric and adult MS centers at Massachusetts General and Brigham and Women's after July 2001.

Patients were eligible if treatment started within 12 months of the first symptom and were followed for at least a year. All told, the researchers identified 110 adult-onset patients and 21 whose disease started before they were 18.

The main outcome measure was annualized relapse rate, defined as the number of relapses divided by the number of person-years at risk. Rates for the adult and younger groups were compared using a proportional means model, as well as a negative binomial regression that excluded the first attack.

In both models, the researchers found that the annualized relapse rate was significantly higher in the younger cohort. Specifically:

* When the first attack was included, adolescent patients had an annualized rate of 1.4, compared with 0.65 for adult-onset patients. The difference was significant at P0.001 and yielded an adjusted rate ratio of 2.81 (with a 95% confidence interval from 2.07 to 3.81.

* Excluding the first attack yielded a pediatric rate of 1.13 and an adult rate of 0.4, also significantly different at P0.001.The adjusted rate ratio was 2.93 (with a 95% confidence interval from 1.93 to 4.46).

When the researchers controlled for the effects of disease-modifying treatment -- such as interferons, glatiramer acetate (Copaxone) or oral immunosuppressants -- the difference remained significant, with rate ratios of 2.82 and 3.02, depending on the model.

And when age was treated as a continuous variable, the age at onset was significantly associated with relapse rate at P0.001, Dr. Chitnis and colleagues found.

While several studies have shown that disease progression is slower in adolescent-onset cases, the researchers said they were unable to show that in this case because of the relatively short disease duration.

But if that is so, Dr. Chitnis and colleagues argued, the finding that younger patients have more relapses implies "greater plasticity, less neurodegeneration, and potentially more repair and remyelination in the younger nervous system."

The authors pointed out that "it remains possible that patients with more severe pediatric-onset MS were selectively evaluated at our centers. However, distance from the center, a proxy measure for referral bias, which has been shown to influence clinical characteristics in prior articles in the neurology and oncology literature, was not different between our pediatric-onset and adult-onset groups."

The study was supported by the National Multiple Sclerosis Society. The researchers did not report any conflicts.

Primary source: Archives of Neurology
Source reference:
Gorman MP, et al "Increased relapse rate in pediatric-onset compared with adult-onset multiple sclerosis" Arch Neurol 2009; 66(1): 54-59.

UPDATE 1-Biogen moves long-acting Avonex to final trial-CEO

2009-01-26T09:17:00.003-07:00

By Deena Beasley

LOS ANGELES, Jan 7 (Reuters) - Biogen Idec Inc (BIIB.O: Quote, Profile, Research) has moved a long-acting version of its multiple sclerosis drug, Avonex, directly into trials designed to meet requirements for regulatory approval, the company's chief executive said on Wednesday.

CEO Jim Mullen said during an investor conference held in New York that the company has completed Phase 1 trials and will study dosing the drug once every two weeks as well as once monthly.

"I think we've got a good chance of similar efficacy as interferons," he said.

Patients in the trials will be treated for one year with the injectable drug and full results will likely be available in two years, Mullen added.

Avonex, with sales of $573 million in the third quarter of 2008, is the leading drug for multiple sclerosis, an autoimmune disease in which the body mistakenly attacks the fatty myelin coating surrounding nerve cells.

Biogen, along with marketing partner Elan Corp Plc (ELN.I: Quote, Profile, Research), also sells MS drug Tysabri, which was taken off the market after its 2004 introduction when it was linked to a potentially fatal brain infection, but reintroduced beginning in 2006 because there were so few good options for patients with MS.

Mullen said investors continue to underestimate the newer drug's sales potential: "We have a much more bullish view on Tysabri than the Street does."

"We've got a 40 share of the overall MS market, which could easily go to 50," the CEO said.

He said other discrepancies between Biogen's outlook and that of Wall Street include the company's "more bullish view on the sustainability of Avonex," growth potential for the franchise surrounding non-Hodgkin's lymphoma drug Rituxan, and additional opportunities for trimming costs.

Mullen said Biogen will no longer report new cases of progressive multifocal leukoencephalopathy, the brain infection associated with Tysabri, through filings with securities regulators, but will instead issue weekly updates on its Web site. (Reporting by Deena Beasley; Editing by Andre Grenon and Matthew Lewis)

© Thomson Reuters 2008. All rights reserved. Users may download and print extracts of content from this website for their own personal and non-commercial use only. Republication or redistribution of Thomson Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters. Thomson Reuters and its logo are registered trademarks or trademarks of the Thomson Reuters group of companies around the world.

Thomson Reuters journalists are subject to an Editorial Handbook which requires fair presentation and disclosure of relevant interests.

Immune Molecule Decreases Severity Of Multiple Sclerosis-like Disease In Mice

2009-01-26T09:04:00.001-07:00

ScienceDaily (Jan. 4, 2009) — A group led by Dr. Cedric Raine at Albert Einstein College of Medicine have explored the expression of an immune molecule (CXCL1) that interacts with myelin-producing cells, finding that CXCL1 decreases the severity of disease in a mouse model of multiple sclerosis (MS).

The autoimmune disease multiple sclerosis (MS) attacks the central nervous system, resulting in demyelination of neurons. Myelin-producing cells in the central nervous system are severely depleted in lesions in patients with MS.

Myelin-producing cells express immune receptors and have been shown to respond to the immune molecule CXCL1, although the role of CXCL1 in MS has not been previously explored. Dr. Raine and colleagues examined the effects of CXCL1 specifically expressed in the nervous system in a mouse model of MS. They observed decreased severity of disease and more prominent remyelination in these mice. CXCL1, therefore, may play a neuroprotective role in CNS autoimmune demyelination.

In future studies, Dr. Raine's group plans to determine how CXCL1 mediates protection in MS. "Exploration of these pathways affords novel therapeutic avenues to enhance the limited remyelination typically seen in MS."

Journal reference:

1. Omari KM, Lutz SE, Santambrogio L, Lira SA, Raine CS. Neuroprotection and remyelination after autoimmune demyelination in mice that inducibly overexpress CXCL1. Am J Pathol, 2009, 174:164-176

Adapted from materials provided by American Journal of Pathology, via EurekAlert!, a service of AAAS.

MLA
American Journal of Pathology (2009, January 4). Immune Molecule Decreases Severity Of Multiple Sclerosis-like Disease In Mice. ScienceDaily. Retrieved January 26, 2009, from http://www.sciencedaily.com /releases/2008/12/081230074742.htm

First Phase III results for FTY720, a novel oral therapy for MS, show superior efficacy compared to interferon beta-1a

2008-12-12T07:22:00.004-07:00

* FTY720 significantly reduced annualized relapse rates by 52% (0.5 mg dose) and 38% (1.25 mg) vs. interferon beta-1a in one-year TRANSFORMS study[1]

* FTY720 generally well-tolerated and safety profile in line with previous experience[1]

* Regulatory submissions for FTY720 in US and EU on track for end of 2009; FREEDOMS and FREEDOMS II placebo-controlled Phase III studies continuing

* Multiple sclerosis, a devastating disease causing progressive disability, affects up to 2.5 million people worldwide including many young adults[2]

Basel, December 12, 2008 - Initial results from the one-year Phase III TRANSFORMS study show the investigational oral compound FTY720 (fingolimod) has superior efficacy to a current standard of care for patients with relapsing-remitting multiple sclerosis (MS). Patients on oral FTY720 experienced significantly fewer relapses than those treated with the injectable medicine interferon beta-1a (Avonex®*)[1].

The study, the first one-year head-to-head Phase III trial against a standard of care in MS, met its primary endpoint for both doses of FTY720.

The annualized relapse rate at one year for patients given FTY720 0.5 mg was 0.16, representing a 52% reduction compared to a relapse rate of 0.33 for interferon beta-1a (p<0.001). The FTY720 1.25 mg dose also showed a significant reduction in relapses with a rate of 0.20 representing a 38% reduction against interferon beta-1a (p<0.001). No statistically significant difference was seen between the two FTY720 doses[1].
Comprehensive analyses of the TRANSFORMS study data are ongoing, and detailed results are planned to be presented at a leading scientific congress in 2009. Regulatory submissions remain on track to be completed in the US and EU at the end of 2009.
"We are encouraged by the early results from TRANSFORMS, which represent a major step towards delivering an effective oral treatment for people with relapsing-remitting MS," said Trevor Mundel, MD, Global Head of Development at Novartis Pharma AG. "These positive results reinforce the potential for FTY720 to provide a significant advance in the future treatment of this devastating disease."

MS is a chronic autoimmune neurodegenerative disease of the central nervous system associated with irreversible progression of disability[3]. As many as 2.5 million people worldwide are affected by the condition[2] that typically begins in early adulthood between the ages of 20 and 40 years when patients are in the prime of life[4].

TRANSFORMS (TRial Assessing injectable interferoN vS FTY720 Oral in RrMS) is the first of three studies to report results in one of the largest Phase III clinical programs ever conducted in MS, involving more than 3,400 patients around the world.
As a head-to-head trial against interferon beta-1a, TRANSFORMS was designed to assess the efficacy of FTY720 compared to an established disease-modifying therapy in reducing relapse rates in patients with relapsing-remitting MS, the most common form of the disease. Two other studies - FREEDOMS and FREEDOMS II - are two-year placebo-controlled Phase III studies to assess the impact of FTY720 in reducing the frequency of relapses and slowing the progression of disability, and to further characterize the benefit-risk profile. Data from these studies to support regulatory submissions are expected in 2009.

TRANSFORMS was a one-year worldwide double-blind, double-dummy study that enrolled 1,292 patients. The study had three arms: oral FTY720 0.5 mg and 1.25 mg once-daily, and the active comparator interferon beta-1a given once-weekly by intra-muscular injection. The patient population in TRANSFORMS was consistent with the demographics and disease state seen in Phase III clinical trials for other disease-modifying treatments for relapsing-remitting MS[5].

The safety profile of FTY720 seen in TRANSFORMS was in line with previous clinical experience. The compound was generally well-tolerated with 87% of FTY720-treated patients completing the study on treatment. The proportion of patients discontinuing therapy was 10% in the FTY720 0.5 mg group, 15% in the FTY720 1.25 mg group, and 12% in the interferon beta-1a group[1].

The most commonly reported adverse events, seen in more than 10% of patients in all three study arms, were headache, nasopharyngitis and fatigue. Influenza-like symptoms were reported in 37% of patients treated with interferon beta-1a and in 4% of patients treated with FTY720[1].

Adverse effects seen in FTY720-treated patients included transient reductions in heart rate at the start of treatment, minor increases in blood pressure, and elevations in liver enzymes (also seen with interferon beta-1a). Macular edema (swelling of the center of the retina) was detected in less than 1% of FTY720-treated patients[1]. Seven cases of localized skin cancer were diagnosed in FTY720-treated patients (four basal cell carcinoma and three melanoma), while one case of squamous cell carcinoma was seen in the interferon beta-1a group. All of these localized skin lesions were successfully removed[1].

As previously reported, two fatal herpes infections occurred in patients treated with FTY720 1.25 mg. Both cases involved confounding factors impacting the outcome, but a role for FTY720 could not be excluded given its immunosuppressive effect.

In general, the safety profile of the FTY720 0.5 mg dose appeared to be better than that of the 1.25 mg dose, including lower rates of infections and bradycardia. Further analyses of the TRANSFORMS data and results from the ongoing Phase III studies will help to provide a more comprehensive assessment of FTY720's benefit-risk profile.

Disclaimer
The foregoing release contains forward-looking statements that can be identified by terminology such as "on track," "planned," "encouraged," "potential," "to assess," "to further characterize," "expected," "appeared to be," "will," or similar expressions, or by express or implied discussions regarding potential regulatory submissions or marketing approvals for FTY720 or regarding potential future revenues from FTY720. You should not place undue reliance on these statements. Such forward-looking statements reflect the current views of management regarding future events, and involve known and unknown risks, uncertainties and other factors that may cause actual results with FTY720 to be materially different from any future results, performance or achievements expressed or implied by such statements. There can be no guarantee that FTY720 will be submitted for approval in any market by the end of 2009 or at any time. Nor can there be any guarantee that FTY720 will ever be approved for sale in any market. Neither can there be any guarantee that FTY720 will achieve any particular levels of revenue in the future. In particular, management's expectations regarding FTY720 could be affected by, among other things, unexpected clinical trial results, including unexpected new clinical data (including the upcoming results of the FREEDOMS and FREEDOMS II trials) and unexpected additional analysis of existing clinical data (including the results of the ongoing additional analyses of the TRANSFORMS clinical data); unexpected regulatory actions or delays or government regulation generally; competition in general; the company's ability to obtain or maintain patent or other proprietary intellectual property protection; government, industry and general public pricing pressures; the impact that the foregoing factors could have on the values attributed to the Novartis Group's assets and liabilities as recorded in the Group's consolidated balance sheet, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those anticipated, believed, estimated or expected. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis AG provides healthcare solutions that address the evolving needs of patients and societies. Focused solely on healthcare, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic pharmaceuticals, preventive vaccines, diagnostic tools and consumer health products. Novartis is the only company with leading positions in these areas. In 2007, the Group's continuing operations (excluding divestments in 2007) achieved net sales of USD 38.1 billion and net income of USD 6.5 billion. Approximately USD 6.4 billion was invested in R&D activities throughout the Group. Headquartered in Basel, Switzerland, Novartis Group companies employ approximately 97,000 full-time associates and operate in over 140 countries around the world. For more information, please visit http://www.novartis.com.

References

[1.] Novartis. Data on file.
[2.] World Health Organization. Neurology atlas, 2004. http://www.who.int/mental_health/neurology/neurogy_atlas_review_references.pdf (Accessed 30 November 2008).
[3.] Confavreux C, Vukusic S. Accumulation of irreversible disability in multiple sclerosis: from epidemiology to treatment. Clin Neurol Neurosurg 2006;108:327-32.
[4.] Confavreux C, Aimard G, Devic M. Course and prognosis of multiple sclerosis assessed by the computerized data processing of 349 patients. Brain 1980;103:281-300.
[5.] Cohen J, et al. Oral fingolimod (FTY720) versus interferon beta-1a in relapsing-remitting multiple sclerosis: baseline patient demographics and disease characteristics from a Phase III trial (TRANSFORMS). Abstract at WCTRIMS, April 2008.
[*] Avonex® is a registered trademark of Biogen Idec.

# # #

Novartis Media Relations
Central media line: +41 61 324 2200

Eric Althoff
Novartis Global Media Relations
+41 61 324 7999 (direct)
+41 79 593 4202 (mobile)
eric.althoff@novartis.com

John Taylor
Novartis Pharma Communications
+41 61 324 6715 (direct)
+41 79 593 4279 (mobile)
john.taylor@novartis.com
e-mail: media.relations@novartis.com
Novartis Investor Relations
Central phone:

+41 61 324 7944

Ruth Metzler-Arnold

+41 61 324 9980

North America:

Pierre-Michel Bringer

+41 61 324 1065

Richard Jarvis

+1 212 830 2433
John Gilardi

+41 61 324 3018

Jill Pozarek

+1 212 830 2445
Thomas Hungerbuehler

+41 61 324 8425

Edwin Valeriano

+1 212 830 2456
Isabella Zinck

+41 61 324 7188

e-mail: investor.relations@novartis.com

e-mail: investor.relations@novartis.com

Elan seeks deals to raise cash

2008-12-11T09:08:00.001-07:00

Tue Nov 18, 2008 6:11pm EST

NEW YORK (Reuters) - Elan Corp Plc (ELN.N: Quote, Profile, Research, Stock Buzz)(ELN.I: Quote, Profile, Research, Stock Buzz) aims to cut costs, close locations and raise up to $500 million as it seeks to strengthen its balance sheet and offset slower growth of its multiple sclerosis drug Tysabri.

Elan, which expects to generate revenue of about $1 billion in 2008, is based in Ireland, but also has facilities in San Francisco, New York, Boston, Pennsylvania, Georgia and Tokyo.

Chief Executive Officer Kelly Martin said on Tuesday at the Reuters Health Summit in New York, that Elan is considering closing two of those locations or reducing its presence there, though the company has not yet decided which ones.

"We are currently going through a process where we are evaluating where we can take costs down and reallocate and reduce," he said. "There are a couple of locations we can potentially exit entirely."

In the nine months ended September 2008, Elan posted a net loss of $240.5 million. It is burning cash at a rate of more than $300 million a year, and it has $1.7 billion in debt that comes due over the next five years.

Elan's U.S. stock has fallen over 80 percent since early July to $6.06 late Tuesday afternoon, hurt by safety concerns over Tysabri and disappointing results from a mid-stage trial of its experimental Alzheimer's disease vaccine.

The company is on track to run out of money in less than two years if it doesn't take firm action.

To that end, Martin said he wants to raise between $300 million and $500 million within the next six to eight months, and he expects to do that by selling the rights to some of its experimental products.

Elan recently tried to sell its drug technology business, worth about $1 billion, but the credit crisis killed off a hoped-for sale to private equity and a sale is now unlikely for at least a year, Martin said.

"I'm not optimistic the markets will get back to normalcy any time soon," he said. "Our plans are not to wait around for a transaction but to run the business."

Kelly said the business is profitable but is not central to its portfolio of neurology drugs, and the company still hopes to sell it once the market improves.

For now, the company will focus on raising money by selling rights to experimental drugs in areas such as cancer and rheumatoid arthritis, products that are in early stages of development.

"By the middle of 2009 you can expect us to do something with our pipeline," he said.

Martin said the company is determined to keep its pipeline of drugs for neurological disorders, including experimental products to treat Alzheimer's disease, Parkinson's disease and Parkinson's disease.

Elan and its U.S. partner Biogen Idec Inc (BIIB.O: Quote, Profile, Research, Stock Buzz) have said they expect 100,000 patients to be taking Tysabri by the end of 2010, a figure Kelly said would represent around 20 percent of the market, but some analysts consider that over-optimistic.

Martin said it will begin to become clear over the next few quarters whether the companies can reach that figure, as they will reflect physician responses to the latest cases of PML, a potentially deadly brain infection that caused the drug to be temporarily withdrawn in 2005.

In the third quarter, Biogen said sales of Tysabri had slowed, though it maintained it can still meet the patient target of 100,000. For that to happen, growth will have to accelerate.

Tysabri was reintroduced in 2006, with stricter warnings, and physicians had started to become more comfortable with the drug. Then, at the end of July, two more cases of PML were reported. And another was reported in October.

That has once again cast doubt on the ultimate sales potential for the drug, but Martin sounded optimistic.

"We are hoping the emotion and rumoring around PML is beginning to fade away a little bit," he said. "I think a 20 percent market share for the drug is a very achievable number over time."

(For summit blog: summitnotebook.reuters.com/))

(Reporting by Toni Clarke, Ben Hirschler; Editing by Richard Chang)

© Thomson Reuters 2008. All rights reserved. Users may download and print extracts of content from this website for their own personal and non-commercial use only. Republication or redistribution of Thomson Reuters content, including by framing or similar means, is expressly prohibited without the prior written consent of Thomson Reuters. Thomson Reuters and its logo are registered trademarks or trademarks of the Thomson Reuters group of companies around the world.

MS services are poor

2008-12-11T09:04:00.002-07:00

Greg Baxter

greg.baxter@imt.ie

Ireland has scored poorly, relative to the rest of Europe, on care for multiple sclerosis from the EU body Multiple Sclerosis — the Information Dividend (MS-ID).

According to the Dividend’s MS Barometer 2008, which compares MS services around Europe, Ireland scored 24th out of 32 countries, just behind Croatia and ahead of Spain. Germany scored the best, and Romania finished last.

The countries were measured on access to treatments and therapies; MS research agenda; employment of people with MS; empowerment of people with MS; reimbursement of MS costs; accurate MS data collection; and MS medication coming to the market.

Ireland scored particularly poorly on empowerment of people with MS, scoring zero out of a possible 45 points. Romania also scored zero.

A country’s empowerment scores take into account the presence of a consultation group on MS that advises Government on MS policies; whether that MS society is a member of the body that decides on the reimbursement of a new MS therapy; whether the MS society has consultative status with your national administration; whether a self-management course exists for people with MS to empower them to co-manage their own health as far as possible; and other services.

Ireland also scored zero points in the area of data collection. Fourteen countries registered a score in this area, but 18 scored zero.

In all categories but reimbursement of costs related to MS, Ireland scored below average.

Environment Causes Increase In Multiple Sclerosis Among Women Only

2008-12-11T08:59:00.001-07:00

17 Nov 2008

Gender has become a dominant factor in Multiple Sclerosis (MS) during the last decades. Already with a ratio of 3.2 to 1 MS is gradually changing into a disease predominantly among women. Since genetic factors can be ruled out as a cause of this gender related increase, scientific attention is on environmental factors that may increase MS risk in women exclusively. Most likely environmental factors include smoking, viral infections, Vitamin D deficiency, hygiene changes and dietary factors.

Almost 400 MS scientists and clinicians from around the world gathered this week during a medical scientific conference on 'Multiple Sclerosis and Gender', organized by the independent European Charcot Foundation, to share and discuss their scientific views on the backgrounds of this major shift in gender ratio.

"In due course the raised attention on gender related topics will undoubtedly lead to better results and questions regarding individualized MS treatment, both in women and men", professor O.R. Hommes, chairman of the European Charcot Foundation stated. "This conference has raised the simple question whether females with MS should be treated differently than males".

One of the main focal points in the gender related approach is the effect of pregnancy on disease progression in MS. The disease practically disappears during the last trimester of pregnancy. Why is that and can we use our vast knowledge of natural female sex hormones, such as estriol, progesterone and prolactin, to develop new ways of treating women with MS? Several phase III clinical trials are already underway that will provide answers to this question by the end of 2009.

Multiple sclerosis (MS) is an inflammatory and degenerative disease of the central nervous system leading in time to severe disability. This chronic disease is affecting 70 to 200 per 100.000 persons in Europe.

European Charcot Foundation

Article URL: http://www.medicalnewstoday.com/articles/129672.php

Multiple Sclerosis Progression Can Be Predicted With MRI

2008-11-10T08:59:00.001-07:00

ScienceDaily (Nov. 6, 2008) — A new study published in Journal of Neuroimaging shows that MRI scans used on multiple sclerosis (MS) patients to determine if the disease has affected gray matter in the brain can identify those at-risk for progression of disability.

MS affects approximately 400,000 people in the United States and as many as 2.5 million worldwide. It is the most common cause of progressive disability in young adults. While the cause of the disease remains unknown, it is characterized by damage to the covering over the nerve fibers in the brain and spinal cord, or to the nerve fiber itself.

In an attempt to understand the causes of disease progression, researchers at the Partners MS Center, led by Dr. Rohit Bakshi and his team, have developed new ways to detect gray matter damage.

Dr. Bakshi, Director of the Laboratory for Neuroimaging Research and an Associate Professor of Neurology and Radiology at the Brigham and Women's Hospital and Harvard Medical School, led a four year follow-up study, which found that patients with unnatural darkness of gray matter structures as seen on MRI pictures carried a higher risk for progression of physical disability. This abnormal darkness is referred to as T2 hypointensity, and is suggestive of excessive iron deposits. In addition, the researchers found that the new marker of gray matter damage showed closer correlations with patients' clinical status than other established MRI markers of disease severity, including lesions, also known as "plaques," and shrinkage of the brain, also know as "atrophy."

"MRI scans obtained from patients with MS are being used to develop measures and techniques that can accurately measure the visible and hidden damage to the brain, especially in gray matter areas and can more accurately predict the course of the disease," says Bakshi.

As a result of the findings, MRI-based measurement of gray matter damage may be used as a surrogate marker of disease progression. Physicians may therefore be able to more accurately identify patients at risk for developing this progressive disease.

MS has been traditionally viewed as a disease affecting the white matter of the brain, where messages are transferred between the brains gray matter sections, which control the processing of information. While prior research has shown that the brain's gray matter is also affected, studies detailing its effects have been limited. In addition, current therapies for MS are incomplete, raising the need to better understand disease mechanisms and the biomarkers of disease progression. If excessive iron in gray matter contributes to damage, this would open a new avenue for developing better therapies.

Journal reference:

1. Neema et al. Deep Gray Matter Involvement on Brain MRI Scans Is Associated with Clinical Progression in Multiple Sclerosis. Journal of Neuroimaging, 2008; DOI: 10.1111/j.1552-6569.2008.00296.x

Adapted from materials provided by Wiley-Blackwell, via EurekAlert!, a service of AAAS.
Need to cite this story in your essay, paper, or report? Use one of the following formats:
APA

MLA
Wiley-Blackwell (2008, November 6). Multiple Sclerosis Progression Can Be Predicted With MRI. ScienceDaily. Retrieved November 10, 2008, from http://www.sciencedaily.com /releases/2008/11/081105164308.htm

Avigen Announces Restructuring and Strategic Direction

2008-11-10T08:53:00.002-07:00

ALAMEDA, Calif., Nov 3, 2008 (GlobeNewswire via COMTEX News Network) -- Avigen, Inc. (Nasdaq:AVGN), a biopharmaceutical company innovating therapeutics for neurological care, today announced a significant restructuring of the company aimed at preserving cash and reassessing its strategic opportunities. As a result of the restructuring, which will involve staff reductions of over 70 percent of the company's total workforce, Avigen expects to have sufficient cash to fund operations for over four years.

"Our deepest gratitude goes out to all the talented and dedicated employees who have worked so hard to complete a timely and well executed AV650 clinical program, as well as enhance the value of our other clinical-stage drug candidates from our internal research and development," said Kenneth Chahine, Ph.D., J.D., Avigen's President and Chief Executive Officer. "By reducing to only a core team, however, we can significantly lower our infrastructure costs, while retaining the know-how and expertise necessary to monetize our current assets and identify opportunities to acquire new assets. We believe our strong cash position and management team will make Avigen an attractive partner in this challenging financial climate."

Highlights:

-- Avigen expects to end 2008 with top-line cash and securities of
approximately $49 million and believes this restructuring
will extend its resources to support at least four years of
operations, including providing funds to develop one asset to a
meaningful value inflection point without additional equity
financings.

-- Avigen's contract with Sanochemia Pharmaceutica AG regarding
AV650 has been terminated to avoid further payment obligations
on the part of Avigen.

-- Avigen believes the breadth and value of AV411 can be best
realized with the support of a partner; therefore, Avigen
intends to seek a partner and does not currently plan to
initiate the Phase 2b development program for neuropathic
pain. Current and future National Institute on Drug Abuse-funded
Phase 2a trials in opioid withdrawal and methamphetamine relapse
will continue.

-- Proceeds from the sale or partnering of Avigen's current assets
(AV411 and AV513) could significantly increase its cash position
and its ability to develop additional assets to meaningful value
inflection points without additional equity financings.

"Our experienced team has faced similar challenges, and has demonstrated the ability to take decisive action to create valuable opportunities," said Dr. Chahine. "Given the current economic environment, we believe this strategy represents a sound plan that will not only withstand, but capitalize on, the current market conditions."

About AV411

AV411 is a first-in-class orally bioavailable small molecule, a glial attenuator that suppresses pro-inflammatory cytokines IL-1 beta, TNF alpha, and IL-6, and may upregulate the anti-inflammatory cytokine IL-10. While considered a New Molecular Entity (NME) in the United States and Europe, the drug was first approved in Japan more than 15 years ago. The drug has been prescribed to over one million patients for a different indication and has a good post-marketing safety profile in nearly 15,000 patients studied at the prescribed doses.

Glial activation in the brain and spinal cord contribute to the establishment and amplification of the chronic pain state. As part of its program investigating glial attenuation as a novel approach to the treatment of neuropathic pain, Avigen discovered that AV411 (ibudilast) was efficacious in standard preclinical models of opioid withdrawal. While ibudilast was initially developed as a non-selective phosphodiesterase (PDE) inhibitor for the treatment of bronchial asthma, its efficacy in neuropathic pain models appears to be independent of this activity. AV411 has advanced through Phase 1 and 2a clinical trials for neuropathic pain and is currently in a NIDA-funded trial with Columbia University addiction research specialists for opioid withdrawal. Additional preclinical research has revealed that AV411 can attenuate opioid-induced glial activation and both behavioral and neurochemical markers of opioid-induced reward and withdrawal. Moreover, collaborative studies with NIDA have revealed utility in methamphetamine relapse in animals which is being translated to a NIDA-funded exploratory clinical trial with UCLA investigators in 2009. Based on its research, Avigen has filed for patents protecting the use of AV411, as well as for patents on AV411 analogs which the company believes have the potential to be effective second generation molecules. Additional information on AV411 can be found on Avigen's website at www.avigen.com.

About Avigen

Avigen is a biopharmaceutical company focused on developing and commercializing unique small molecule therapeutics to treat serious neurological disorders, including neuropathic pain and opioid addiction and withdrawal. Avigen's strategy is to complete the requirements of clinical development for each of the candidates in its product pipeline, and continue to look for opportunities to expand its pipeline through a combination of internal research, acquisitions and in-licensing, with the goal of becoming a fully integrated commercial biopharmaceutical company that remains committed to its neurology products. Avigen is currently developing AV411 for neuropathic pain, as well as opioid withdrawal and addiction in collaboration with NIDA. Additionally, the company is advancing AV513, a novel therapy for the treatment of multiple bleeding disorders, including hemophilia A and B, toward clinical trials. For more information about Avigen, consult the company's website at www.avigen.com.

The Avigen, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=2981

Statement under the Private Securities Litigation Reform Act

This press release contains forward-looking statements, which include, among others, statements relating to Avigen's expectations that the current restructuring and staff reduction will reduce its infrastructure costs and extend its financial resources to support at least four years of operations without additional equity financings; that it will retain the know-how and expertise necessary to monetize its current assets; that it will be able to identify and acquire new assets; that it will be able to secure a partner to initiate the AV411 Phase 2b development program for neuropathic pain; that current and future National Institute on Drug Abuse-funded trials in opioid withdrawal or methamphetamine relapse will continue; that Avigen can avoid further payment obligations to Sanochemia beyond those already paid; that it will be able to take any asset to a meaningful value inflection point with or without additional equity financings; that it can generate any proceeds from the sale or partnering of current assets; and that the Company's current strategy will allow it to withstand or capitalize on the current market conditions. These risks and uncertainties include: unexpected expenses incurred with respect to the restructuring may occur; savings from the restructuring may not be as much as Avigen expects due to unexpected impediments to reducing expenses; in this economic environment, Avigen may not be able to find a partner for AV411 on terms favorable to Avigen; that costs necessary to bring products to a meaningful inflection point may be more than Avigen expects; and those detailed in reports filed by Avigen with the Securities and Exchange Commission, including Avigen's quarterly report on Form 10-Q for the period ended June 30, 2008, under the caption "Risks Related to Our Business" in Item 2 of Part I of that report, which was filed with the SEC on August 11, 2008.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Avigen, Inc.

Avigen, Inc.
Michael Coffee, Chief Business Officer
510-748-7372
IR@avigen.com
1301 Harbor Bay Parkway
Alameda, CA 94502

Source MDx, Brigham and Women's Partner on Multiple Sclerosis Biomarker Discovery

2008-11-10T08:49:00.003-07:00

[November 3, 2008]

By a GenomeWeb staff reporter

NEW YORK (GenomeWeb News) - Source MDx has formed a partnership with Boston’s Brigham and Women’s Hospital to study RNA-based biomarkers for multiple sclerosis, the company announced today.

The duo plans to use expression profiling to find diagnostic markers, markers for active or stable MS, and response markers for currently available MS therapies.

Multiple sclerosis, a chronic central nervous system inflammatory disease, has unpredictable and highly variable progression. For instance, many individuals with the disease experience partial or complete remission punctuated by MS relapses whereas others experience progressively worse symptoms. Current methods for determining the stage of disease are imprecise, but some research suggests that gene expression profiles could help map disease progression and guide treatment, the companies said.

“Our objective is to evaluate RNA-based markers in the broader context of each patient’s genetics, protein markers, family history, and clinical information in order to determine markers that can help in making a diagnosis of MS and prognosticate on drug response in MS,” Brigham and Women’s Hospital neurology researcher Phil De Jager, co-leader of the study, said in a statement. “By doing so, we hope to be better able to identify makers that could lead to improved diagnostic tools, therapies, or treatment regimen.”

The research ties into ongoing work by De Jager and Brigham and Women’s molecular immunologist David Hafler and others in the International Multiple Sclerosis Genetics Consortium and Partners Healthcare MS Center.

Source MDx said that it has patented the use of gene expression data for identifying, monitoring, and treating MS. The company added that it has identified and patented MS gene expression biomarkers linked to MS in independent studies by De Jager and Hafler.

Virtual Reality Helps MS Patients Regain Stability While Walking

2008-11-10T08:40:00.003-07:00

Medigait LLC announces the release of a new device that combines virtual reality programming and real-time motion detection into a cell-phone sized device that helps MS patients restore their ability to walk more normally. The neuroplastic brain response stimulated by this device helps MS patients jump-start the rewiring process inside the patient's brain. Their brain literally rewires itself creating new healthy walking circuits bypassing diseased areas sometimes in as little as two weeks.
When I saw these positive results I was amazed, very excited, and gratified.

Haifa, Israel (PRWEB) November 3, 2008 -- Studies performed over the last few years show many Multiple Sclerosis (MS) patients no longer have to live with a meandering or unsteady gait caused by their disease. According to a study published in the highly acclaimed medical journal "Neurology", closed-loop visual feedback helps MS patients improve their walking. Both stride length and walking speed improved dramatically helping MS patients walk safely.

Better yet, a study published in the "Journal of Neurological Sciences" found that when closed-loop auditory feedback was combined with visual cues, MS patients showed even more improvement and confidence while walking.

Based on concepts discovered while working on a NASA project helping helicopter pilots to safely navigate around tall objects, Professor Yoram Baram, CEO of Medigait LLC, created the GaitAid Virtual Walker. The Virtual Walker is a highly sophisticated device about the size of a cell phone paired with a set of high-tech eyeglasses. Professor Baram designed this device specifically to help patients with MS and other movement disorders regain their stability while walking.

GaitAid

GaitAid

Medigait announces the re-introduction of the GaitAid Virtual Walker to the market after locating a reliable high quality US-based medical device company to build the device for them.

The GaitAid Virtual Walker combines proven visual aids and audio feedback helping patients with MS and other movement disorders improve their stability while walking through a process called neuroplasticity. This process essentially rewires the patient's brain by creating new healthy circuits to bypass disease-damaged areas.

The National Multiple Sclerosis Society estimates there are approximately 400,000 people in the United States with MS. World-wide about 2.5 million people suffer from MS.

Because MS afflicts both the central and the peripheral nervous systems, not all MS patients show the same symptoms. However, a large percentage of MS patients exhibit loss of balance and muscle coordination affecting their ability to walk safely. Although a cure for MS hasn't been found, the GaitAid Virtual Walker offers a safe, effective, and non-pharmacological method of helping MS patients walk better leading to more productive and safer lives.

Clinical studies have shown major improvements to walking speed and stride length in 70% to 85% of MS and other movement disorder patients. Some patients have been helped to the point of no longer needing the device except occasionally to maintain their improvement.

Clinical studies appearing in medical journals, Neurology, Neural Processing Letters, and Journal of Neurological Sciences described the device as being effective but without the risk or possible adverse side effects caused by surgery and medication. Links to this information can be found at http://www.gaitaidmedical.net/studies.html

"When I saw these positive results I was amazed, very excited, and gratified." -- Professor Yoram Baram Phd.

"The results clearly indicate that the device helps patients with MS control their gait. The degree of improvement is proportional to the degree of impairment. The results support the potential role of the device as a rehabilitation modality in MS, and substantiate their specific implementation in efforts to alleviate, improve, and restore mobility in patients with gait disturbances due to neurological disorders in general." -- Ariel Miller, MD, Multiple Sclerosis Center, Carmel Medical Center and Rappaport Faculty of Medicine & Research Institute

For more information on the GaitAid Virtual Walker device:

Visit our website: http://www.medigait.com or
contact Amir Baram by calling 1-888-777-9906 (U.S) or email him at
support (at) medigait (dot) com

About MediGait LLC
MediGait was founded by Yoram Baram, who received his PhD degree from MIT and is presently a Professor of Computer Science and incumbent of the Roy Matas / Winnipeg Chair in Biomedical Engineering at the Technion, Israel Institute of Technology. The idea for this project was sparked 12 years ago while Professor Baram was designing a mechanism for NASA to navigate low-flying helicopters around obstacles. The concept of the design, which Baram later applied to the medical device, is that the optical images of objects help the observer navigate, stabilize and pace movement in space.

About the Technion Institute of Technology
The Technion-Israel Institute of Technology is Israel's leading science and technology university. Home to the winners of the Nobel Prize in science, it commands a worldwide reputation for its pioneering work in nanotechnology, electrical engineering, computer science, biotechnology, water-resource management, materials engineering, aerospace and medicine. The majority of the founders and managers of Israel's high-tech companies are alumni.

Interferon Could Be A Key To Preventing Or Treating Multiple Sclerosis

2008-10-31T10:00:00.000-06:00

31 Oct 2008

Multiple sclerosis (MS) results when the body's own defense system attacks nerve fibers in the brain and spinal cord. Now scientists led by John Russell, Ph.D., at Washington University School of Medicine in St. Louis have shown that interferon-gamma plays a deciding role in whether immune cells attack and injure the central nervous system (brain and spinal cord) in mice.

Interferon-gamma is an immune system protein that helps the body defend itself from invaders. In their latest research, which appeared in the October issue of the Journal of Experimental Medicine, the researchers show that interferon-gamma determined whether activated immune cells previously primed to go after nerve cells would actually cause nerve damage in experimental mice.

The researchers found that in the cerebellums and brainstems of the mice, interferon-gamma was protective. However, in the spinal cord, interferon-gamma had the opposite effect, permitting nerve cell damage.

"Some studies show that the most serious cases of MS in people occur when the immune system specifically targets the cerebellum, a part of the brain responsible for sensory perception, coordination and movement control," says Russell, professor of developmental biology. "Our study suggests that researchers need to look at the amount of interferon-gamma produced in the cerebellum and other brain regions in people with MS."

The researchers studied mice genetically engineered to be physiologically "blind" to interferon-gamma the mice had none of the usual receptors on their cells that recognize and respond to interferon-gamma. So in these mice it was as though interferon-gamma didn't exist.

In the interferon-insensitive mice, immune cells primed to attack nerves and then injected into the mice's veins were able to get into the cerebellum and brain stem and initiate nerve cell damage leading to MS-like disease.

In comparison, in mice with normal interferon-gamma recognition, immune cells were prevented from entering the brain and causing problems. The exact mechanism to account for this is still under study.

"Down the road, we would like to investigate whether we can prevent disease in the cerebellum in mice if we promote interferon production in that brain region," Russell says. "One way to do that would be to use gene therapy to insert a gene that would increase interferon in the mice's brains. Then we would test the mice to see if they gained protection against MS-like disease."

In contrast to its protective role in the brain, in the spinal cord interferon-gamma helped instigate nerve damage. In mice with intact interferon-gamma recognition, activated and injected immune cells were able to enter the spinal cord and cause injury. In mice without interferon recognition, the immune cells were unable to initiate spinal cord inflammation, and no damage occurred.

"Our research shows that certain characteristics inherent in different regions of the brain and spinal cord can provoke immune attacks on nerve cells," Russell says. "An understanding of the mechanisms involved in immune system invasion of the nervous system may allow development of better models for determining prognosis and treating many neurological diseases such as multiple sclerosis."

This latest research bolsters Russell's central hypothesis about MS and related disorders, which goes against some widely held assumptions. He holds that in physiological circumstances that ultimately lead to MS, the central nervous system itself allows or even aids immune system attacks.

"A scientifically popular view of how MS occurs is that the immune system somehow gets armed against normal brain antigens and attacks neurons," Russell says. "In that view, brain cells have a passive role. But in this and previous research, we've shown that there's a 'conversation' between the immune system and the central nervous system and that molecular signals passed between them are involved in the development of MS-like disease in mice."

Lees JR, Golumbek PT, Sim J, Dorsey D, Russell JH. Regional CNS responses to IFN-γ determine lesion localization patterns during EAE pathogenesis. Journal of Experimental Medicine. 2008 Oct 27;205(11):2633-2642.

Funding from the National Multiple Sclerosis Society and the National Institutes of Health supported this research.

Washington University School of Medicine's 2,100 employed and volunteer faculty physicians also are the medical staff of Barnes-Jewish and St. Louis Children's hospitals. The School of Medicine is one of the leading medical research, teaching and patient care institutions in the nation, currently ranked third in the nation by U.S. News & World Report. Through its affiliations with Barnes-Jewish and St. Louis Children's hospitals, the School of Medicine is linked to BJC HealthCare.

Washington University in St. Louis
1 Brookings Dr., Campus Box 1070
St. Louis
MO 63130
United States
http://www.wustl.edu

Biogen, Elan shares fall as third Tysabri patient suffers brain infection

2008-10-31T09:50:00.001-06:00

30 October 2008

Biogen Idec and partner Elan Corp have suffered a slide in their respective share prices after a new case of progressive multifocal leukoencephalopathy in a multiple sclerosis patient being treated with Tysabri, was disclosed.

In a filing to the US Securities and Exchange Commission, Biogen said that the relevant regulatory agencies had been notified of a confirmed case of PML, a potentially deadly brain infection, in a patient suffering from MS in the USA who had been on Tysabri (natalizumab). The disclosure comes after Biogen and Elan reported in July that two cases of PML had emerged in Europe.

In the latest case, the filing reveals that the patient received 14 injections of Tysabri. The patient has a history of prior disease-modifying therapies including beta-interferons and Texa’s Copaxone (glatiramer acetate) and he/she had also been treated with methotrexate for a rheumatology condition. The individual is now under the care of their treating physician, Biogen said.

PML has played a major part in the history of Tysabri. These recent cases are the first to be recorded since the reintroduction of natalizumab in the USA and approval in Europe two years ago. Biogen and Elan had voluntarily withdrawn the drug a year earlier after three patients developed the brain infection.

Since reintroduction, sales of Tysabri have soared – by the end of September there were 35,500 patients using the drug worldwide and in the third quarter of this year, 3,700 new patients were added. It has also now available in the USA as a treatment for Crohn's disease and Biogen and Elan expect that 100,000 MS patients will be taking the drug by 2010.
.
Biogen and Elan have a strict monitoring programme in place for the therapy and the reintroduction of Tysabri was based in part on the number of PML cases not exceeding the level acceptable to the US Food and Drug Administration (one in every 1,000). As only three cases have been reported, the treatment is well within that limit but the news has certainly spooked investors.

In after-hours trading last night, Biogen shares had fallen by as much as 15%, while this morning Elan stock has been hit. At 9.40 UK time, the Irish firm’s shares were down 18% to 4.80 euros.

Ian Hunter, an analyst at Goodbodys in Dublin, has issued a research note this morning saying “we believe that this new case should do little to projected numbers of patients on Tysabri, given that the considerable pull back in momentum after the first two cases…will have accounted for the majority of those patients and physicians wary enough of the PML risk” to consider not taking or prescribing the drug. However, the fact that this patient was on the drug for 13 months and the other two were on it for 14 and 17 months “may suggest that there is a timing issue and that it takes over a year on Tysabri for the interaction between drug and patient to lead to the development of PML”.

By Kevin Grogan

Novel Oral Agent for MS May Reduce Brain Lesions

2008-10-29T11:07:00.002-06:00

By Crystal Phend, Staff Writer, MedPage Today
Published: October 23, 2008
Reviewed by Robert Jasmer, MD; Associate Clinical Professor of Medicine, University of California, San Francisco

Patients given oral fumarate at the highest dose had 69% fewer new inflammatory lesions enhanced by gadolinium on brain MRI scans at 12 to 24 weeks of treatment compared with placebo (P<0.0001), reported Ludwig Kappos, M.D., of University Hospital Basel, and colleagues in the Oct. 25 issue of The Lancet.

In the phase IIb dose-ranging trial, the experimental drug also reduced the annualized relapse rate by 32% compared with placebo, although the difference was not significant (P=0.272).

Fumarate, also known as BG00012, may have dual neuroprotective and anti-inflammatory effects by activating a pathway that defends against neuronal death from oxidative stress, protects the blood-brain barrier, and supports myelin integrity in the central nervous system, the researchers said.

If the longer-term phase III studies now underway show the agent to be as effective as the currently available injection- or infusion-only drugs, fumarate may have an advantage as initial treatment based on convenience alone, Dr. Kappos' group said.

"BG00012 could also be an alternative for patients who cannot tolerate or choose not to initiate injectable therapies because of injection-related effects or anxiety," they said.

However, fumarate will have to compete with a number of other oral agents being developed, commented Per Soelberg Sorensen, M.D., and Finn Sellebjerg, M.D., Ph.D., both of Rigshospitalet in Copenhagen, in an accompanying commentary.

Phase III studies of at least four other oral agents -- cladribine (Leustatin), fingolimod, teriflunomide, and laquinimod -- are in progress for relapsing-remitting multiple sclerosis.

"Within the next four to five years neurologists may be able to choose between up to five new oral drugs for relapsing-remitting multiple sclerosis," Drs. Sorensen and Sellebjerg wrote.

The decisive factor in which drug wins out will be the balance between efficacy, safety, and convenience, they said.

But the findings of this study suggest fumarate may have a better benefit-to-risk profile than these oral competitors or approved first-line injectable drugs, they suggested.

The trial included 257 patients ages 18 to 55 with relapsing-remitting multiple sclerosis treated at 43 centers across Europe and Russia.

Participants were randomized to receive double-blind treatment for 24 weeks with oral fumarate at a dose of 120 mg once daily, 120 mg three times daily, or 240 mg three times daily, or to placebo.

The highest dose group had significantly fewer total new gadolinium-enhancing lesions on MRI at weeks 12, 16, 20, and 24 combined -- the primary endpoint -- compared with the placebo group (1.4 versus 4.5, P<0.0001).

This type of lesion indicates blood-brain barrier breakdown and continued inflammatory activity within the central nervous system, the researchers noted.

Gadolinium-enhancing lesions have been linked to acute clinical relapses and cerebral atrophy, although the short follow-up precluded atrophy as an outcome in the study, they said.

The highest-dose group also had a 48% reduction in the mean number of new or enlarging T2-hyperintense lesions over 24 weeks (P=0.0006) and a 53% reduction in new T1-hypointense lesions compared with placebo (P=0.014).

Lower doses of the experimental agent were also associated with fewer total new enhancing lesions, although there were no significant differences compared with placebo (3.3 with 120 mg once daily and 3.1 with three times daily versus 4.5).

Although the study was not powered for relapse endpoints, annualized relapse rates were reduced by 32% with the highest dose of fumarate over the first 24 weeks (0.44 versus 0.65 with placebo, P=0.272).

In an extension period of 24 weeks for safety assessment, during which patients continued on their assigned dose and the placebo group switched to the highest fumarate dose, relapse rates dropped further.

The reductions compared with the first 24 weeks were 43% for the lowest dose group, 40% for the intermediate dose group, and 64% for the highest dose group, and 60% for those who switched to fumarate after placebo.

"This result could indicate a delayed and increasing effect of BG00012 over time," Dr. Kappos' group suggested.

The researchers said the experimental drug was safe and generally well tolerated.

The most common adverse events included flushing, multiple sclerosis relapse, and headache. Abdominal pain was also significantly more common with the highest dose than with placebo.

The editorialists noted, however, that it was worrying that 25% of patients on the highest dose of fumarate withdrew from the study, with 13% of patients in this group discontinuing because of adverse events.

The study was funded by Biogen Idec.

Dr. Kappos reported participating as principal investigator, member, or chair of planning and steering committees or advisory boards in multiple sclerosis clinical trials sponsored by Allozyne, Amgen, Bayer HealthCare Pharmaceuticals, Bayer-Schering Pharma, Bayhill, Biogen Idec Inc, Eisai, Elan Pharmaceuticals Inc, Genmab, Genzyme, Merck-Serono, Medicinova, Novartis, sanofi-aventis, Shire, Roche, Teva, UCB, and Wyeth.

He also reported giving lectures on the diagnosis and management of multiple sclerosis sponsored by non-restricted educational grants from one of the sponsors and receiving research and clinical operations funding at his center supported by non-restricted grants from one or more of the sponsors and by grants from the Swiss MS Society, the Swiss National Research Foundation, the European Union, and the Gianni Rubatto Foundation.

Co-authors reported conflicts of interest for Biogen Idec, GlaxoSmithKline, Schering AG, Novartis, Bayer Schering, and the US National Institutes of Health, Serono, Teva, Octapharma, sanofi-aventis, Teva, Antisense Pharmaceuticals, Janssen Cilag, Johnson & Johnson, Orion, UCB, AstraZeneca, and Roche.

Dr. Sorensen reported receiving honoraria for lecturing and advisory councils, trial steering committees, travel expenses for attending meetings, or research grants from Biogen Idec, Bayer Schering, Merck Serono, TEVA, Biopartners, sanofi-aventis, and Genmab. Dr. Sellebjerg has received honoraria for lecturing and advisory councils, travel expenses for attending meetings, or research grants from Biogen Idec, Bayer Schering, Merck Serono, and TEVA.

Primary source: Lancet
Source reference:
Kappos L, et al "Efficacy and safety of oral fumarate in patients with relapsing-remitting multiple sclerosis: a multicentre, randomised, double-blind, placebo-controlled phase IIb study" Lancet 2008; 372: 1463-72.

Additional source: Lancet
Source reference:
Sorensen PS, Sellebjerg F "Oral fumarate for relapsing-remitting multiple sclerosis" Lancet 2008; 372: 1447-48.

Opexa Provides Additional Promising Data Including Statistically Significant Reduction in Disability With Tovaxin® for the Treatment of Multiple Scle

2008-10-29T10:58:00.002-06:00

Tovaxin Also Demonstrates Reduction in Relapse Risk and Myelin T-cell Reactivity in Patients With More Active Disease

THE WOODLANDS, Texas--(BUSINESS WIRE)--Opexa Therapeutics, Inc. (NASDAQ:OPXA), a company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes, today announced additional positive data from the company’s Phase IIb TERMS clinical trial (Tovaxin® for Early Relapsing Multiple Sclerosis). The latest analysis focused on a prospective group of patients (n=50) with an annualized relapse rate (ARR) of greater than 1 at study entry which is comparable to ARR baselines of patients in previous Tovaxin studies. These findings demonstrate a statistically significant improvement in disability as measured by the Expanded Disability Status Scale (EDSS) (p=0.045) for patients treated with Tovaxin as compared to those receiving placebo. In this group, 28.1 percent of patients treated with Tovaxin showed an improvement in EDSS as compared to only 5.6 percent in the placebo group. Additionally, there was an 88 percent reduction in the level of brain atrophy and a more than 20 percent reduction in the number of gadolinium (Gd) lesions progressing to black holes in the Tovaxin group, which may suggest a beneficial neuroprotective effect. Overall, the analysis shows that patients treated with Tovaxin demonstrated a benefit across all clinical and magnetic resonance imaging (MRI) endpoints (primary, secondary and tertiary) in this patient population. Immunology data also appears to support Tovaxin’s mechanism of action, indicating that patients with less myelin T-cell reactivity have a lower risk of relapse. Additional quality of life measurements, such as the Timed 25 foot Walk, also showed a benefit for Tovaxin over placebo (0.14 vs. -0.02, as measured by respective Z scores).

The data shows that these patients when treated with Tovaxin demonstrate an ARR of 0.28 which represents a 55 percent reduction compared to those patients on placebo. This relapse rate is on par with the lowest relapse rates observed with currently available MS treatments which range from 0.2 to 0.9. Study findings also show Tovaxin possesses an impressive safety and tolerability profile. Opexa expects to conduct a Phase II close-out meeting with the United States Food and Drug Administration during the first half of 2009 to discuss next steps for the further clinical development of Tovaxin. Opexa is actively engaged in discussions with potential strategic partners for the Tovaxin program.

The TERMS study provided Opexa the opportunity to create a comprehensive database of clinical immunology and epitope data in MS patients that may significantly advance the understanding and treatment of the disease. This type of information is particularly important for Opexa as Tovaxin’s dual mechanism of action involves the depletion of myelin-reactive T-cells in the peripheral blood and the regulation of anti-inflammatory T-cells to rebalance an MS patient’s immune system. Previous studies show that if the myelin-peptide reactivity in a patient’s peripheral blood is reduced, the clinical symptoms associated with MS will also be reduced. The initial analysis of the study’s immunology and epitope database has shown that patients treated with Tovaxin appeared to have less myelin-peptide reactivity over the course of the study than those on placebo. This was measured using Opexa’s proprietary Epitope Analysis Assay (EAA) which assessed reactivity across all three key myelin proteins at several time points throughout the 52-week study. Additionally, over the full course of treatment, more patients on Tovaxin remained in the lower quartile of peptide reactivity (56 percent) than those on placebo (39 percent). This epitope data appears to correlate with the study’s MRI and clinical endpoints which, among other findings, showed that following the full course of treatment (weeks 28-52), the ARR in the Tovaxin group dropped to 0.065 (0.749 for placebo).

The pattern emerging from the analysis of the immunology and epitope database suggests that those patients exhibiting a higher T-cell reactivity often demonstrated a worsening of at least one MRI endpoint (Gd lesion, Gd volume, new T2 count or progression to black holes) and one clinical endpoint (either relapse or EDSS). The converse also appears true with less T-cell reactivity implying less or no worsening of certain MRI and clinical endpoints.

Additionally, the immunology data shows that reactivity to all three of the key myelin proteins (MOG, PLP and MBP) was broadly present in study patients, which may be an important finding for the future treatment of MS. Opexa is also assessing which specific peptides from each of the proteins may provide the most relevant targets for the company to enhance its manufacturing process and further strengthen its intellectual property portfolio.

“Tovaxin seems to be demonstrating a benefit to these patients as indicated by immunology, MRI and clinical parameters,” stated Dr. Clyde Markowitz, associate professor of neurology and director of the MS center at the University of Pennsylvania School of Medicine. “This is interesting data and additional trials are certainly warranted to further explore the potential of this novel treatment.”

Previously reported top-line results from the TERMS study demonstrated that Tovaxin was safe and well tolerated with no serious adverse events related to treatment. The most common adverse event related to Tovaxin was mild injection site reaction. Continued analysis of safety and tolerability data has confirmed these top-line results with no serious adverse events observed in any Tovaxin-treated patients during the entire study.

“This landmark first-in-class study has yielded impressive results for Tovaxin in MS patients with high disease burden, including a marked reduction in disability, relapse risk and levels of T-cell reactivity. These findings are very encouraging and we are eager to continue the clinical development of this novel therapeutic,” commented Neil K. Warma, president and chief executive officer of Opexa Therapeutics. “There remains a tremendous unmet medical need for MS patients, and we believe a safe and effective patient-specific treatment may generate considerable interest among regulatory authorities, patients, physicians and potential strategic partners. With this in mind, we remain committed to aggressively pursuing our two primary objectives: the continued development of Tovaxin towards a pivotal Phase III trial and a high-value partnership for the Tovaxin program. With the MS market exceeding $6 billion and growing, we believe Tovaxin is very competitively positioned from a safety, efficacy and patient compliance perspective.”

About Tovaxin

Tovaxin is an individualized T-cell therapeutic vaccine that consists of attenuated patient-specific myelin-reactive T-cells (MRTCs) against peptides of proteins from Myelin basic protein (MBP), Myelin oligodendrocyte glycoprotein (MOG) and Proteolipid protein (PLP) or combinations thereof. Tovaxin’s dual mechanism of action combats the demyelination of the nerve fibers in the central nervous system, the underlying cause of MS. Clinical results have demonstrated that Tovaxin produces the following therapeutic effects:

* Anti-idiotypic effect – Induces an immune response that depletes and regulates the circulating pathogenic myelin-reactive T-cells that attack the myelin sheath of nerve fibers.
* Anti-ergotypic effect – Rebalances the overall immune system by causing a shift from pathogenic inflammatory T-cells to anti-inflammatory T-cells.

Tovaxin is manufactured in Opexa’s in-house cGMP facility.

About the TERMS Study

The TERMS study was a Phase IIb multi-center, randomized, double blind, placebo-controlled trial in 150 patients with Relapsing-Remitting Multiple Sclerosis or high risk Clinically Isolated Syndrome (CIS). The study involved 2:1 randomization with 100 patients receiving Tovaxin and 50 receiving placebo. According to the study protocol, patients received a total of five subcutaneous injections at weeks 0, 4, 8, 12 and 24. The primary efficacy endpoint of the TERMS trial was the cumulative number of gadolinium-enhanced brain lesions (CELs) using MRI scans summed over weeks 28, 36, 44 and 52. The trial’s secondary efficacy endpoints included annualized relapse rate (ARR), new CELs at weeks 28 through 52 and T2-weighted lesion volume compared to baseline.

About Opexa

Opexa Therapeutics develops and commercializes cell therapies to treat autoimmune diseases such as multiple sclerosis, rheumatoid arthritis and diabetes. The Company is focused on autologous cellular therapy applications of its proprietary T-cell and stem cell therapies. The Company's lead product is Tovaxin, a T-cell therapy for multiple sclerosis is in Phase IIb trials. The Company holds the exclusive worldwide license for adult multipotent stem cells derived from mononuclear cells of peripheral blood. The technology allows large quantities of monocyte-derived stem cells to be produced efficiently for use in autologous therapy, thus circumventing the threat of rejection. The Company is in preclinical development for diabetes mellitus. For more information visit the Opexa Therapeutics website at www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of collaborative relationships, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. We assume no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

Permalink: http://www.businesswire.com/news/opexa/20081023005419/en

Study Results: Multiple Sclerosis Patients Have Significant and Sustained Reduction in Disability and Risk Of Relapse On Alemtuzumab Versus Approved T

2008-10-29T10:49:00.004-06:00

Date: October 22, 2008

Final Phase 2 Data Published in New England Journal of Medicine
International Phase 3 Alemtuzumab Studies Enrolling

Genzyme Corporation (Nasdaq: GENZ) and Bayer HealthCare Pharmaceuticals Inc. today announced study results showing that patients with early relapsing-remitting multiple sclerosis (RRMS) taking once-yearly cycles of alemtuzumab reduced their risk of relapse by 74 percent and the risk of sustained accumulation of disability by 71 percent compared to patients treated with the active comparator Rebif® (high-dose interferon beta-1a). Importantly, the mean disability of patients on alemtuzumab improved from baseline, whereas the mean disability of those on Rebif worsened. The treatment benefits of alemtuzumab were sustained for at least three years, even though the majority of alemtuzumab-treated patients were last dosed two years earlier. These results come from the final three-year analysis of a Phase 2 clinical study (CAMMS223) reported in the Oct. 23 issue of the New England Journal of Medicine. The study involved 334 patients who had not previously been treated for their disease.

“The alemtuzumab trial data continue to suggest a potentially new and exciting treatment for patients with early, active multiple sclerosis,” says Alastair Compston, Professor of Neurology and the head of the Department of Clinical Neurosciences at the University of Cambridge, United Kingdom, and the study’s principal investigator. “This randomized study confirms findings from prior studies demonstrating that treatment with alemtuzumab can have a profound and durable impact on patients with relapsing-remitting multiple sclerosis, including restoring some lost function in many patients.”

“Symptoms of multiple sclerosis result from an immune system attack on the protective insulation surrounding nerve fibers of the central nervous system. We believe alemtuzumab shuts down this immune system attack, treating the disease at its root cause,” says Alasdair Coles, Senior Lecturer, Department of Clinical Neurosciences, University of Cambridge and a lead investigator in the study.

Common non-serious adverse events in the trial included infusion-associated reactions in the alemtuzumab patients and flu-like symptoms in patients using Rebif. Alemtuzumab-treated patients were more likely than Rebif patients to experience infections, which were predominantly mild to moderate in severity, as well as autoimmune thyroid disease. Three percent of alemtuzumab-treated patients developed the potentially serious autoimmune adverse event immune thrombocytopenic purpura (ITP), a disorder characterized by a low platelet count and corresponding increased risk of uncontrolled bleeding. Additional study and trial safety information is below.

“The trial was larger and follow-up was longer than the typical Phase 2 trial in multiple sclerosis. It is important to note that we compared the investigative drug directly against a widely used therapy rather than against placebo. The trial did not show an increased risk of life-threatening or opportunistic infections, but a proportion of alemtuzumab patients experienced new autoimmune disease. We have been able to create a robust patient monitoring program that allows us to proceed into our two international Phase 3 trials with greater assurance on safety associated with patient management,” says Dr. Richard Moscicki, chief medical officer for Genzyme.

According to the National Multiple Sclerosis Society, approximately 400,000 Americans acknowledge having MS, and every week about 200 people are diagnosed. Worldwide, multiple sclerosis may affect 2.5 million individuals. The disease causes a wide range of symptoms including difficulty with walking, numbness, fatigue and impairment of vision, and progresses to permanent, severe disability in the majority of patients. Relapsing-remitting MS is the most common presenting form of the disease.

“We are pleased to see potential new treatment options move positively through the MS pipeline,” says Dr. John Richert, executive vice president for research and clinical programs at the National Multiple Sclerosis Society, “and as alemtuzumab is moved into Phase 3 studies, we hope that individuals with MS will consult with their physicians to assess whether they are appropriate patients and if so will consider the pros and cons of participating in these important clinical trials.”

Additional New Top-Line Data

New and previously unreported top-line data from secondary analyses of the CAMMS223 Phase 2 clinical trial, presented last month at the World Congress on Treatment and Research in Multiple Sclerosis (WCTRIMS), revealed that the proportion of clinically disease-free patients was significantly higher in the alemtuzumab group than in the Rebif group at year one (86 percent vs. 63 percent), year two (81 percent vs. 48 percent), and year three (71 percent vs. 39 percent, p-values <0.0001). “Clinically disease-free” was defined as the absence of both relapses and sustained accumulation of disability during the time-period assessed.

The top-line data from WCTRIMS also showed that the proportion of patients free of sustained accumulation of disability over a six-month period was also significantly greater in the alemtuzumab group than in the Rebif group at year one (97.2 percent vs. 87.0 percent), year two (94.3 percent vs. 82.4 percent), and year three (91.0 percent vs. 73.8 percent, p-values <0.005).

Further, the proportion of relapse-free patients over time was significantly greater in the alemtuzumab group than in the Rebif group at year one (91.1 percent vs. 69.3 percent), year two (88.2 percent vs. 58.5), and year three (80.2 percent vs. 51.6 percent, p-values <0.0001).

Phase 2 Extension Trial

Genzyme with Bayer support has launched an extension of the CAMMS223 trial to examine safety and efficacy outcomes beyond three years, and to compare two distinct retreatment strategies. The results should provide an understanding of the long-term effects of prior alemtuzumab treatment as well as the safety and sustained efficacy of additional alemtuzumab retreatment delivered in fixed annual cycles or as needed for resumed MS disease activity. In the fixed arm, patients will receive two annual cycles of alemtuzumab (12 mg/day for three consecutive days/cycle). In the as-needed arm, retreatment is deferred until a patient relapses or develops two or more new/active brain lesions on MRI.

Phase 3 Trials

Genzyme with Bayer support is sponsoring two Phase 3 trials in which patients are now enrolling. The CARE-MS I Phase 3 study (Comparison of Alemtuzumab and Rebif Efficacy in Multiple Sclerosis), is a randomized, rater-blinded study that will again compare alemtuzumab to Rebif in patients with relapsing-remitting MS who have not been previously treated for their disease. The CARE-MS II trial is studying patients who have continued to relapse while using approved MS therapies.

Physicians or patients in the United States seeking additional information about the CARE-MS Phase 3 trials should go to www.care-ms.com or call Genzyme Medical Information at 800-745-4447.

About Study CAMMS223

In the Phase 2 trial, 334 patients with active relapsing-remitting multiple sclerosis were enrolled at 49 medical centers in Europe and the United States. Patients in the trial were randomized to treatment with alemtuzumab at one of two dose levels (12 or 24 mg per day intravenously) for five days during the first cycle and three days 12 months later during the second cycle of therapy, or Rebif (44 mcg administered by subcutaneous injection three times per week, as indicated in its product label). A third cycle of alemtuzumab therapy was received by 46 patients at month 24.

The trial compared the efficacy of alemtuzumab with Rebif using two primary outcome measures: the Relapse Rate and the time to Sustained Accumulation of Disability as evidenced by an increase in the Expanded Disability Status Scale (EDSS) score for six consecutive months. Efficacy assessments were made by independent neurologists blinded to patients’ treatment assignments. The EDSS is a 10-point scale in which every 0.5-point step marks a notable deterioration in neurological capabilities.

The mean disability score of patients after alemtuzumab actually improved (by 0.39 EDSS points) indicating a recovery of neurologic functions. The median disability score improved to a similar extent after alemtuzumab. In contrast, mean disability worsened in the Rebif group (by 0.38 EDSS points) resulting in a difference of nearly a full EDSS point (0.77 difference, p<0.0001) at three years.

Safety Data

A total of six alemtuzumab-treated patients, and one Rebif-treated patient, in this study developed a serious adverse event, immune thrombocytopenic purpura (ITP). ITP is a disorder characterized by a low platelet count and corresponding increased risk of uncontrolled bleeding. The Rebif patient with ITP was asymptomatic but ITP persisted at the time of study completion. In the previously reported alemtuzumab-related fatal case, symptoms of ITP were experienced but were not recognized in time, thus delaying medical attention. Of the remaining alemtuzumab cases, four patients were diagnosed promptly, responded well to medical treatment, and have been stable without a need for ongoing treatment. The other alemtuzumab-treated case experienced spontaneous remission of ITP. A patient monitoring program was instituted in the trial, and there have been no new cases of ITP reported in CAMMS223 in approximately two years.

Common non-serious adverse events in the trial included infusion-associated reactions in the alemtuzumab patients and flu-like symptoms in patients using Rebif. Alemtuzumab-treated patients were more likely than Rebif patients to experience infections, particularly of the upper respiratory tract; infections were predominantly mild to moderate in severity and there were no life-threatening or fatal infections. Though alemtuzumab transiently lowers white blood cell counts, the trial did not show an increased risk of opportunistic infections. Serious infections were infrequent in the alemtuzumab-treated patients. Approximately 23 percent of alemtuzumab-treated patients developed autoimmune thyroid-related adverse events, including Graves’ disease, and were managed using conventional therapies.

Alemtuzumab is an investigational drug for the treatment of MS and must not be used in MS patients outside of a formal, regulated clinical trial setting in which appropriate patient monitoring measures are in place.

About Alemtuzumab

Alemtuzumab is licensed in the United States as a single agent for the treatment of B-cell chronic lymphocytic leukemia (B-CLL), and outside of the U.S. for the treatment of B-CLL in patients who have been treated with alkylating agents and for whom fludarabine combination therapy is not appropriate. The product was launched in its oncology indication in 2001 in the US, where it is marketed by Bayer HealthCare Pharmaceuticals Inc. as Campath®, and in Europe, where it is named MabCampath®.

Alemtuzumab is a humanized monoclonal antibody that binds to a specific target, CD52, on cell surfaces and directs the body’s immune system to destroy those cells. It is the first and only monoclonal antibody approved by the FDA for the treatment of patients with B-CLL.

Genzyme and Bayer are co-developing alemtuzumab in oncology and multiple sclerosis. Bayer holds exclusive worldwide marketing and distribution rights to alemtuzumab.

Campath for B-CLL has a boxed warning that includes information on cytopenias, infusion reactions, and infections. The most commonly reported adverse reactions in patients with B-CLL were infusion reactions (fever, chills, hypotension, urticaria, nausea, rash, tachycardia, dyspnea), cytopenias (neutropenia, lymphopenia, thrombocytopenia, anemia), and infections (CMV viremia, CMV infection, other infections). Other commonly reported adverse reactions include vomiting, abdominal pain, insomnia and anxiety. The most commonly reported serious adverse reactions are cytopenias, infusion reactions, and immunosuppression/infections.

About Genzyme

One of the world's leading biotechnology companies, Genzyme is dedicated to making a major positive impact on the lives of people with serious diseases. Since 1981, the company has grown from a small start-up to a diversified enterprise with more than 10,000 employees in locations spanning the globe and 2007 revenues of $3.8 billion. In 2007, Genzyme was chosen to receive the National Medal of Technology, the highest honor awarded by the President of the United States for technological innovation.

With many established products and services helping patients in nearly 90 countries, Genzyme is a leader in the effort to develop and apply the most advanced technologies in the life sciences. The company's products and services are focused on rare inherited disorders, kidney disease, orthopaedics, cancer, transplant and immune disease, and diagnostic testing. Genzyme's commitment to innovation continues today with a substantial development program focused on these fields, as well as cardiovascular disease, neurodegenerative diseases, and other areas of unmet medical need.

About Bayer HealthCare Pharmaceuticals Inc.

Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions. Bayer HealthCare Pharmaceuticals comprises the following business units: Women's Healthcare, Diagnostic Imaging, General Medicine, which includes Cardiology and Primary Care and Specialty Medicine, which includes Hematology, Oncology and Multiple Sclerosis. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

This press release contains forward-looking statements regarding Genzyme’s future plans and business strategies, including: its expectations for the success of alemtuzumab to treat MS, and its expectations regarding the information to be gained from the phase 2 extension trial. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements, including: that the phase 3 trials are not successful; that the phase 2 extension trial fails to produce the anticipated results regarding longer-term effects; and the risks and uncertainties described in reports filed by Genzyme with the Securities and Exchange Commission under the Securities Exchange Act of 1934, as amended, including without limitation the information under the heading "Risk Factors" in Genzyme’s Quarterly Report on Form 10-Q for the quarter ending June 30, 2008. Genzyme cautions investors not to place substantial reliance on the forward-looking statements contained in this press release. These statements speak only as of the date of this press release, and Genzyme undertakes no obligation to update or revise these statements.

This news release contains forward-looking statements based on current assumptions and forecasts made by Bayer Group management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in our public reports filed with the Frankfurt Stock Exchange and with the U.S. Securities and Exchange Commission (including Form 20-F). The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

Genzyme®, Campath®, and MabCampath® are registered trademarks of Genzyme Corporation. All rights reserved. Rebif® is a registered trademark of EMD Serono, Inc.

Genzyme’s press releases and other company information are available at www.genzyme.com and by calling Genzyme’s investor information line at 1-800-905-4369 within the United States or 1-678-999-4572 outside the United States.

Bayer HealthCare Pharmaceuticals Inc. press releases and other information are available at www.bayerus.com.

Editor’s Note: This study appears in the October 23, 2008 issue of the New England Journal of Medicine. The citation is N Engl J Med 2008;359:1786-1801.

Terms and Conditions of Use | Privacy Policy | © 2002-2008 Genzyme Corporation. All rights reserved.

Avigen Announces AV650 Did Not Meet Efficacy Endpoints in Phase 2b Clinical Trial for Spasticity in Patients With Multiple Sclerosis

2008-10-29T10:43:00.002-06:00

ALAMEDA, Calif., Oct 21, 2008 (GlobeNewswire via COMTEX News Network) -- Avigen, Inc. (Nasdaq:AVGN), a biopharmaceutical company innovating therapeutics for neurological care, today announced that the top-line data from its Phase 2b study of AV650 (tolperisone HCl) for the treatment of spasticity associated with multiple sclerosis (MS) did not achieve statistical significance on its primary endpoint of a reduction from baseline of patients' Ashworth scores as compared to placebo. The reduction of muscle spasm, a secondary endpoint, also failed to achieve statistical significance. There were no safety issues. The trial was adequately powered, and all statistical parameters were in line with expectations.

"We are disappointed with the result of this trial," said Kenneth Chahine, Ph.D., J.D., Avigen's President and Chief Executive Officer. "We had hoped AV650 would become an important new treatment option for people in the United States who currently suffer from spasticity. While we will continue to review the additional data from the trial and consider further options, we are confident in the trial design and the quality of the top-line results.

"We will now shift our focus and resources toward the clinical development of our AV411 program. At the end of 2008, we will have approximately $50 million of cash and securities. This represents approximately 2 years of cash and provides a strong foundation for advancing the development of AV411 for neuropathic pain and opioid addiction and withdrawal."

About the Trial

This double-blind, randomized clinical trial was conducted in 27 MS centers in Germany and other European countries to evaluate safety, tolerability, pharmacokinetics and efficacy in up to 150 MS patients suffering from spasticity. This trial was being conducted to Good Clinical Practice standards by a leading international clinical research organization. Following a five-week double blind assessment, patients were offered the opportunity to continue for up to twelve months in an open-label safety phase. The primary efficacy endpoint was the Ashworth scale, a standard tool used in the clinic to measure increased resistance to passive limb movement. Secondary endpoints included Brief Pain Index (BPI), painful spasm diaries, and clinical impression of change.

Avigen is a biopharmaceutical company focused on developing and commercializing small molecule therapeutics to treat serious neurological disorders, including neuropathic pain and opioid addiction and withdrawal. Avigen's strategy is to complete the requirements of clinical development for each of the candidates in its product pipeline, and continue to look for opportunities to expand its pipeline through a combination of internal research, acquisitions, and in-licensing, with the goal of becoming a fully integrated commercial biopharmaceutical company that remains committed to its neurology products. Avigen is currently developing AV411 for neuropathic pain, as well as opioid withdrawal and addiction in collaboration with the National Institute on Drug Abuse. Additionally, the company is advancing AV513, a novel therapy for the treatment of multiple bleeding disorders, including hemophilia A and B, toward clinical trials. For more information about Avigen, consult the company's website at www.avigen.com.

The Avigen, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=2981

Statement under the Private Securities Litigation Reform Act

The statements in this press release relating to Avigen's expectations regarding the quality of the top-line results from the AV650 Phase 2b spasticity trial and its consideration of further options based on a review of additional data from the trial, its expectation regarding the level of its financial resources at December 31, 2008 and how long its financial resources will last, its expectation regarding shifting resources toward the clinical development of AV411, and its goal of becoming a fully integrated commercial biopharmaceutical company remaining committed to its neurology products, are forward-looking statements. These risks and uncertainties include, among others, the fact that development of small molecule therapeutics and other therapeutic discovery and development is a time- and resource-intensive process, which may result in the expenditure of a significant amount of time and resources with no progress towards clinical trials or marketable product resulting from the effort; the risk that Avigen will not be able to obtain regulatory approvals for its drug products, which is required prior to marketing drug products; and the risk that early positive preclinical and clinical results will not guarantee that the potential products will ultimately be effective in treating the indications for which they are developed, or exhibit the unique properties they appear to possess. In addition, there are many other risks and uncertainties inherent in the development of drug products. Other risks and uncertainties relating to Avigen are detailed in reports filed by Avigen with the Securities and Exchange Commission, including Avigen's Quarterly Report on Form 10-Q for the period ended June 30, 2008, under the caption "Risks Related to Our Business" in Item 2 of Part I of that report, which was filed with the SEC on August 11, 2008.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Avigen, Inc.

Avigen, Inc.
Michael Coffee, Chief Business Officer
510-748-7372
IR@avigen.com

Bugs In The Gut Trigger Production Of Important Immune Cells, Study Finds

2008-10-29T10:40:00.002-06:00

ScienceDaily (Oct. 17, 2008) — A new study reveals that specific types of bacteria in the intestine trigger the generation of pro-inflammatory immune cells, a finding that could eventually lead to novel treatments for inflammatory bowel disease and other diseases.

The study by NYU Langone Medical Center researchers is published in the October 16 issue of the journal Cell Host and Microbe. The new finding adds to the growing body of research showing that the kinds of bacteria in our intestine, and in our stomach, have an impact on our health.

"There is more and more evidence that gut flora have a tremendously important influence on human health," says Yasmine Belkaid, Ph.D., chief of the mucosal immunology unit in the laboratory of parasitic diseases at the National Institutes of Health "If some set of microbes induces a specific immune response, this points to a way to manipulate the immune system," says Dr. Belkaid. "This new study is the first report that has associated a defined set of gut flora with the induction of specific immune cells."

The new research is from the laboratory of Dan Littman, M.D., Ph.D., the Helen L. and Martin S. Kimmel Professor of Molecular Immunology at NYU School of Medicine and a Howard Hughes Medical Institute Investigator. "It's not the amount of microbial flora but the kind of microbial flora that seems to count," says Dr. Littman.

The new study found that cytophaga-flavobacter-bacteroidetes (CFB) bacteria were associated with the creation of Th17 cells in mice. Typically, in both mice and humans, most of the bacteria found in the gut fall into the CFB phylum or another phylum called Firmicutes. These bacteria play many roles, such as aiding in digestion and protecting against pathogens by outcompeting harmful bacteria.

Inflammatory bowel disease (IBD) affects as many as 700,000 people each year and is one of the most prevalent gastrointestinal diseases in the United States. Treatment with antibiotics has had limited success. But pinpointing the specific species of bacteria that influence the balance of inflammatory cells, says Dr. Littman, could lead to more sophisticated treatments that fine-tune bacteria in the intestine and, in turn, dampen the production of inflammatory cells.

The Yin and Yang of Immunity

A healthy immune system is a balancing act between two opposing yet intimately connected forces, one calming, the other inflammatory. Sometimes called the yin and yang of adaptive immunity, pro-inflammatory cells (the "yang") dominate when the body needs protection, and regulatory cells (the "yin") soothe the immune system when it doesn't.

When this balance is disrupted and there is an overload of fiery yang cells, inflammatory disease results. In recent years, scientists have linked a striking number of autoimmune disorders to excess pro-inflammatory cells, including psoriasis, inflammatory bowel disease, and multiple sclerosis. "The number of inflammatory diseases known to involve T helper 17 (Th17) cells," – the fiery yang cells – "seems to be growing every week," says Dr. Littman.

For this reason, Dr. Littman has been studying the molecular pathways that stimulate the production of these cells. Recently, his team reported on a promising potential therapeutic target that may help ameliorate diseases associated with overproduction of Th17 cells.

In the new study, Dr. Littman's team observed that newborn mice that remain isolated from bacteria never generate any of these cells. Normally, newborn mice are born without any bacteria or Th17 cells in their intestines. They begin to generate the cells only after they begin to eat food and ingest bacteria. These observations suggested that the introduction of bacteria in the gut is associated with the creation of Th17 cells.

To determine if the bacteria actually cause the generation of Th17 cells, the team gave normal, bacteria-filled mice antibiotics that selectively killed some of the bacteria in their small intestine. Some of these antibiotics also depleted their Th17 cells, indicating for the first time a causal link between specific bacteria and the generation of inflammatory cells.

Littman's team then found a colony of mice that have intestinal bacteria but do not have Th17 cells. This colony, it turned out, had different bacteria in their guts than other colonies. "The same way people from different countries have different bacteria in their guts, mice from different colonies will have different bacteria," explains Dr. Ivaylo Ivanov, an author of the study and a post-doctoral fellow in Dr. Littman's laboratory. In this case, "one colony has the bacterial species associated with Th17 cells and the other doesn't."

By comparing the intestinal bacteria in mice, the team discovered that cytophaga-flavobacter-bacteroidetes (CFB) bacteria were associated with the creation of Th17 cells. Dr. Littman's team is now working to determine the specific bacteria that induce pro-inflammatory immune cells in mice. They will use this information to help determine the bacterial species in the intestines of humans that trigger the overproduction of these cells.

Dr. Littman also is interested in identifying the signals emitted by bacteria that influence the innate immune system, which responds to immediate threats from foreign pathogens and produces substances that spur naive or unspecialized T cells to develop into Th17 cells. Manipulation of the bacteria or their products, says Dr. Littman, could then be used to shift the balance of pro-inflammatory and regulatory immune cells.

Adapted from materials provided by NYU Langone Medical Center / New York University School of Medicine.

Improving Therapeutic Options for Multiple Sclerosis

2008-10-29T10:35:00.003-06:00

Improving Therapeutic Options for Multiple Sclerosis: An Interview With Edward Fox, MD, PhD

Elizabeth Samander, PhD

Medscape Neurology & Neurosurgery. 2008; ©2008 Medscape
Posted 10/16/2008

Editor's Note

New data from key trials in multiple sclerosis (MS) regarding novel therapeutic approaches were presented at the World Congress on Treatment and Research in Multiple Sclerosis held from September 17-20, 2008, in Montreal, Canada. Medscape Scientific Director Elizabeth Samander, PhD, interviewed Edward Fox, MD, PhD, about the results of these trials and what implications these results may have on clinical practice and the future management of patients with MS.

Medscape: Dr. Fox, in your opinion, what were the most compelling research findings presented at the World Congress on Treatment and Research in Multiple Sclerosis (WCTRIMS) meeting this year?

Dr. Fox: The overall message from the meeting this year was that a number of medications are currently being investigated that hold great promise for the treatment of multiple sclerosis (MS). Research was presented from numerous trials involving novel therapies, including monoclonal antibody and oral products. There was a substantial amount of evidence presented on both of those categories, including further data from completed phase 2 trials and descriptions of ongoing phase 3 trials. The number of medications that we have under current investigation is really quite large, so it is difficult to see the timeline for these medicines actually being brought to market. We had hoped that these studies would fully enroll quickly, so that we would have finalized data coming within the next few years. However, it is likely that there will be some delays because of the large number of competing trials with slower-than-expected enrollment.

Monoclonal antibody agents discussed at the meeting included alemtuzumab, rituximab, and daclizumab.[1-6] Those antibodies have certainly been well-tested in phase 2 trials, and plans are ongoing for phase 3 trials in relapsing-remitting MS. There was also a phase 3 trial regarding rituximab[1,5,7] for the treatment of primary progressive MS. The primary endpoint of time to confirmed disease progression did not show a statistically significant difference between rituximab and placebo. However, subcohort analysis suggested a benefit for those patients with active magnetic resonance imaging (MRI) at entry into the study. I think that the overall feeling about B-cell depletion in the treatment of MS is very positive. We are looking forward to other studies, including ones on ocrelizumab,[8] another monoclonal antibody, which can selectively deplete B cells and thereby reduce MS disease activity.

The data presented on the alemtuzumab trial was further information from the phase 2 trial, which showed a highly significant decrease in the number of relapses seen in patients on alemtuzumab compared with patients receiving interferon beta-1a subcutaneously 3 times weekly.[4] The data presented were on a 3 year follow-up trial designed to evaluate the percentage of patients who were clinically disease-free. These were patients who did not have relapses and did not have a progression of disability during the course of the trial. The superiority of alemtuzumab compared with beta-interferon was sustained throughout the 3 years on study. Within the third year it was found that 71% of the patients who were on alemtuzumab were clinically disease-free compared with 38% of those who were receiving interferon beta-1a. These findings have led to the development of 2 phase 3 trials currently enrolling patients.

Medscape: What other novel therapeutic approaches are undergoing clinical trials?

Dr. Fox: The oral medications discussed at this meeting included cladribine,[9-11] fingolimod,[12-14] BG-00012,[15,16] teriflunomide,[1,13,17] and laquinimod.[18] All of these oral agents, as well as several others, are showing promise for the treatment of MS. It may be that any or all of these products will have success in the treatment of MS, but at this time we're still in the discovery phase for the safety as well as for the efficacy of these medications.

The phase 2 trials that have been performed have primarily been based on looking for a reduction in new MRI activity in patients with multiple sclerosis. They all have shown various degrees of benefit in reducing gadolinium-enhancing lesions on MRIs during the course of the phase 2 trials. However, these trials were rather short and generally were not powered to show significant clinical outcomes. An exception was the phase 2 trial on fingolimod, which did show a substantial reduction in relapse rate as well.

The phase 3 trials are going to be looking not only at reduction in MRI activity, but will also be looking at the important clinical measures that will be required for these medications to be licensed. Those include a reduction in relapse rate and prevention of sustained accumulation of disability compared with placebo. These clinical outcome measures may or may not be found despite the MRI evidence that has been seen in the past, because there has been a disconnect between MRI activity and clinical activity seen in several previous agents tested for MS.

Medscape: There is evidence that disease-modifying treatments, although partially effective, are associated with injection-related side effects and suboptimal patient adherence. These findings may suggest that novel therapeutic strategies, including oral therapies, monoclonal antibodies, symptomatic treatments, and combination regimens, are warranted. What is the role of these novel agents in the therapeutic armamentarium with respect to first-line use, add-on treatment for failed first-line therapy, or as an effective induction agent?

Dr. Fox: Certainly our hope is that there's going to be an increased number of agents available for the treatment of MS, but it's going to be necessary to determine at what stage of disease we would use these medications.[19] The strategy of phase 3 trials is typically in first-line treatment of relapsing-remitting MS, in a placebo-controlled trial evaluating medication over a period of 2 years. This certainly gives us an idea as to what its overall effectiveness can be, and the newer treatments will be evaluated for the ability to reduce relapses and accumulation of disability. Phase 3 trials will also have to show an acceptable safety profile and risk management plan. The first-line agents that we currently have on the market, glatiramer acetate and the beta interferons, will likely continue to have a role in first-line therapy when we look at long-term efficacy and safety measures of the newer medicines. It will likely take a number of years to determine the true safety of the monoclonal and oral agents, and their overall use as first-line therapies will depend on these data.

The concern that we have about using any of the new agents as an add-on treatment for failed first-line therapy is again, a safety issue. If it's being used in a combination with other immunomodulatory or immunosuppressive therapy, we cannot currently define the safety of such an approach. This has been a major concern lately with the findings of opportunistic infections such as progressive multifocal leukoencephalopathy in patients who are immunosuppressed. Switching from ineffective first-line therapy to a new therapy is going to be the most likely use in clinical practice soon after these agents reach the market.

Using any of these agents as an effective induction agent is going to require more data from trials where this has been specifically tried. Older medications such as mitoxantrone have been tested as induction therapies with some very favorable results, but the novel agents that are being investigated right now have not been similarly tested.[20-24] Therefore, I believe that the novel agents are likely to be used as first-line or second-line agents rather than initially as induction agents.

Medscape: What is the evidence for agents optimized for neuroprotection and neurorepair?

Dr. Fox: Ideally, any medication that we use as a preventive medicine for MS would have neuroprotective and neurorepair abilities. Research has shown a frequent association of better clinical outcomes if there's prevention of atrophy. The concept of neuroprotection is that we are preventing axonal loss and neuronal loss during the early stages of MS prior to the onset of disability. All of the trials that are looking at atrophy measures are addressing this issue to some degree, but we would like to have advanced imaging metrics to look more directly at the preservation of axonal and neuronal function.

The World Congress and Treatment for Research of Multiple Sclerosis Meeting had a substantial amount of data that were presented regarding these advanced imaging metrics, including magnetic resonance spectroscopy (MRS) and magnetization transfer imaging (MTR). Measurement of neuronal loss by optical coherence tomography (OCT) is also a very promising method of evaluating the degree of atrophy seen in MS.[25] It is likely that the novel therapies currently being investigated will also be tested in a similar manner. It is reassuring to know that we are seeing some positive data on neuroprotection. Improving repair mechanisms may require a new type of medication that would stimulate the production of new myelin growth rather than working as an immunosuppressant medication.

Medscape: What are the limitations and benefits of these novel therapeutic agents, with respect to safety and efficacy, compared with long-approved therapies such as interferon beta and glatiramer acetate?

Dr. Fox: The-first line therapies that we are currently using for the treatment of MS have been on the market for a number of years. The users of these medicines have a certain comfort level with these medications now and the understanding that the long-term data have indicated a relatively good safety profile.

Immunomodulating therapies do not appear to cause the degree of risk that the immunosuppressant therapies may cause. We understand the risk-benefit ratios of the medicines that have been on the market for a number of years, but the novel therapies that are currently under investigation will undoubtedly have a higher degree of uncertainty as to their safety for many years to come. It is quite possible that the novel therapies are going to have immunosuppressant activity that could lead to increased risk for opportunistic infections, limiting their use as first-line therapies.

The efficacy measures, on the other hand, may show direct or indirect evidence of superiority to the first-line therapies. If indeed the therapeutic abilities of these novel therapies are great enough, then we may accept a higher risk potential with these medications and use them as first-line therapy. However, I don't think that the true measure of a medication's safety will be known at the time it enters the market.

Medscape: Does the increase of potential therapeutic agents complicate how to choose first-line therapy?

Dr. Fox: I think with the advent of more choices, we're going to have a greater divide among practicing neurologists as to how they approach the disease. Right now there's fairly good uniformity among neurologists in terms of how to treat early MS. There are differences in opinion about the optimal medication strategies for people with aggressive or chronic disease. But early active disease is currently treated by a limited number of agents that have relatively similar outcome measures in terms of their ability to control the disease. If we have new medicines available to us over the next several years that have a much different risk-benefit ratio with greater promise of disease control but increased risk, I feel that there's likely to be a group of neurologists who will use these medications first line. But there will be a number of other neurologists who are much more conservative in their acceptance of the new medications and will limit the new medicines only to those patients who have failed existing therapies.

Medscape: The fields of genetics and proteomics are beginning to play a large part in the discovery of novel therapeutic targets. How do these newly identified targets impact the development of individualized therapeutic strategies?

Dr. Fox: As we understand the pathology of MS better, we may find that certain medications are tailored for some patients more than others. For example, the biomarkers that may eventually be used for MS would determine whether we would want to use therapies that were depleters of B cells, T cells, monocytes, or a wide-spectrum immunosuppressant.[26,27] Critical to this line of thinking is the understanding of the mechanism of action of our current medications, which remains somewhat murky to this day, because MS is a highly variable and unpredictable disease with different pathologies among different patients. If we understood the pharmacogenomics of MS better, we might be able to tailor appropriate therapies much more accurately.

Medscape: What are some of the outstanding challenges in the treatment of MS that need to be addressed in the future?

Dr. Fox: Balancing the risks and benefits of medications in the treatment of MS has become increasingly important in the last few years. The major challenge in understanding the nature of this disease is elucidating the prognostic factors for an individual patient, so that we may prevent the development of permanent disability that at this point cannot be alleviated. If we can determine which patients have early, aggressive MS before the development of axonal loss and atrophy, we stand a much greater chance of being able to appropriately determine who might require stronger immunosuppressant. Our challenge going forward is to address both the inflammatory and the degenerative nature of MS with medications that effectively control the disease. Data presented at the World Congress on Treatment and Research in Multiple Sclerosis suggests we are now closer to that goal.

Medscape: Thank you Dr. Fox for sharing this information and your insights with us today.

This activity is supported by an independent educational grant from Teva.
References

1. Linker RA, Kieseier BC, Gold R. Identification and development of new therapeutics for multiple sclerosis. Trends Pharmacol Sci. 2008 Sep 17. [Epub ahead of print].
2. Gasperini C, Cefaro LA, Borriello G, et al. Emerging oral drugs for multiple sclerosis. Expert Opin Emerg Drugs. 2008;13:465-477. Abstract
3. Burton JM, O'Connor P. Novel oral agents for multiple sclerosis. Curr Neurol Neurosci Rep. 2007;7:223-230. Abstract
4. Brinar V for the CAMMS223 Study Group. Alemtuzumab Phase 2 Extension Study Design (CAMMS223): assessing long-term outcomes and potential benefits of additional alemtuzumab treatment in patients with relapsing-remitting multiple sclerosis. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P17.
5. Naismith R, Trinkaus K, Fairbairn M, Lauber J, Piccio L, Cross A. Rituximab as add-on therapy for breakthrough disease: clinical effects over 24 weeks. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P476.
6. Neyer L, Singer R, Wang H, Caras I. Daclizumab exhibits efficacy in multiple sclerosis subjects positive for interferon-beta neutralizing antibodies. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P479.
7. Hawker K. Efficacy and safety of rituximab in patients with primary progressive multiple sclerosis: results of a randomized, double-blind, placebo-controlled, multicenter trial. Parallel Session 10 of Late Breaking News of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Scientific Program S78.
8. Dörner T, Burmester GR. New approaches of B-cell-directed therapy beyond rituximab. Curr Opin Rheumatol. 2008;20:263-268. Abstract
9. Guarnaccia J, Rinder H, Smith B. Preferential effects of cladribine on lymphocyte subpopulations. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P55.
10. Laugel B, Challier J, Siegfried C, Chvatchko Y, Weissert R, Galibert L. Cladribine exerts a modulatory effect on T-cell activation. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P80.
11. Rieckmann P, Giovannoni G, Cook S, et al, for the CLARITY Study Group. Cladribine tablets in relapsing-remitting multiple sclerosis: study design of the 2-year, phase III b CLA RITY (CLA dRibine tablets Treating multiple sclerosis orallY) extension study. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P453.
12. Kappos L, Radue E, O'Connor P, et al., for the FREEDOMS Study Group. Oral fingolimod (FTY 720) in patients with relapsing multiple sclerosis: 3-year results from a phase II study extension. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P72.
13. Kappos L, Polman C, Radue E, et al. Oral fingolimod (FTY 720) in relapsing-remitting multiple sclerosis: baseline patient demographics and disease characteristics from a 2-year phase III trial (FREEDOMS). Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P73.
14. Montalban X, Comi G, Anel J, et al. Oral fingolimod (FTY 720) in relapsing multiple sclerosis: impact on health-related quality of life in a phase II study Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P400.
15. MacManus D, Miller D, Kappos L, R. Gold, et al. The effect of BG00012 on conversion of gadolinium-enhancing lesions to T1-hypointense lesions. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P459.
16. Gold R, Kappos L, Miller D, et al. Safety profile of BG00012, an oral formulation of dimethyl fumarate for patients with relapsing MS. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P50.
17. Tallantyre E, Evangelou N, Constantinescu CS. Spotlight on terfilunomide. Int MS J. 2008;15:62-68. Abstract
18. Comi G, Abramsky O, Arbizu T, et al., for the LAQ/5062 Clinical Advisory Board and Study Group. Oral laquinimod in patients with relapsing-remitting multiple sclerosis: 9-month double-blind active extension of the multi-center, randomized, double-blind, parallel-group placebo-controlled study. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P31.
19. Gold R. Combination therapies in multiple sclerosis. Neurology. 2008;255 Suppl 1:51-60.
20. Vollmer T, Panitch H, Bar-Or A, et al. Glatiramer acetate after induction therapy with mitoxantrone in relapsing multiple sclerosis. Mult Scler. 2008;14:663-670. Abstract
21. Le Page E, Leray E, Taurin G, et al. Mitoxantrone as induction treatment in aggressive relapsing remitting multiple sclerosis: treatment response factors in a 5 year follow-up observational study of 100 consecutive patients. J Neurol Neurosurg Psychiatry. 2008;79:52-56. Abstract
22. Filippi M, Le Page E, Leray E, Edan G, Comi G. Magnetic resonance imaging results of a 3-year randomized trial comparing two therapeutic strategies in aggressive relapsing-remitting multiple sclerosis: mitoxantrone as induction for 6 months followed by interferon-b-1b versus interferon-b-1b. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P254.
23. Perumal J, Hreha S, Caon C, et al. Intense immunosuppression as the initial disease-modifying therapy in clinically active relapsing multiple sclerosis. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P490.
24. Ramathal J, Boggild M. Glatiramer acetate following mitoxantrone induction in relapsing-remitting multiple sclerosis: extended experience. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P498.
25. Balcer L. OCT: window on multiple sclerosis. Parallel Session 5 of Advances in Imaging Techniques of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Scientific Program S43.
26. Garcia-Montojo M, Alvarez-Lafuente R, Dominguez-Mozo M, De las Hera V, Bartolome F, Arroyo R. MxA and MMP-9/TI MP-1 ratio as biomarkers of treatment efficacy and disease activity in multiple sclerosis patients. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster 452.
27. Melnikov A, Scholtens D, Reder A, Balabanov R, Stefoski D, Levenson V. Composite methylation profiles of blood DNA as biomarkers for multiple sclerosis. Disease-modifying therapy-part 2. Program and abstracts of the World Congress on Treatment and Research in Multiple Sclerosis; September 17-20, 2008; Montreal, Quebec, Canada. Poster P869.

Interviewer: Elizabeth Samander, PhD, Scientific Director, Medscape LLC

Interviewee: Edward J. Fox, MD, PhD, Clinical Assistant Professor, University of Texas Medical Branch, Round Rock, Texas

Disclosure for Interviewer: Elizabeth Samander, PhD, is a member of the professional editorial group at Medscape and has reported no relevant financial relationships.

Disclosure for Interviewee: Edward J. Fox, MD, PhD, has disclosed that he has received grants for clinical research from Biogen-Idec, BioMS, EMD-Serono, Genzyme, Opexa Therapeutics, Sanofi-Aventis, and Teva Neuroscience. Dr. Fox has also disclosed that he has received grants for educational activities from Bayer, Biogen-Idec, EMD-Serono, Pfizer, and Teva Neuroscience. Dr. Fox has also disclosed that he has served as an advisor or consultant to Bayer, Biogen-Idec, EMD-Serono, Genzyme, Opexa Therapeutics, and Teva Neuroscience.

Pipex Pharmaceuticals, Inc. Completes Corporate Name Change to Adeona Pharmaceuticals

2008-10-29T10:27:00.002-06:00

ANN ARBOR, Mich., Oct 16, 2008 (GlobeNewswire via COMTEX) -- Pipex Pharmaceuticals, Inc. (the "Company" or "Pipex"), a pharmaceutical company developing innovative late-stage oral drug candidates for the treatment of autoimmune and central nervous system diseases, announced today that it has completed a corporate name change from Pipex Pharmaceuticals, Inc. to Adeona Pharmaceuticals, Inc.

As a result of the name change, the American Stock Exchange (AMEX) will begin to trade the Company's shares of common stock as of today, under the new ticker symbol 'AEN.'
Nicholas Stergis, Chief Executive Officer of Adeona, commented, "As previously announced, we are pleased to have completed our corporate name and ticker symbol change. Our primary focus is toward the continued development of new disease-modifying treatments for autoimmune and central nervous system diseases. This strategy and direction is evident by our recent in-licensing of oral dnaJP1 for the treatment of rheumatoid arthritis (RA) which has completed a 160-patient, double-blind, placebo-controlled phase II clinical trial in RA patients. Oral dnaJP1 complements our existing immunology pipeline which includes, TRIMESTA, an oral treatment for multiple sclerosis (MS) which is currently in a 150-patient double-blind, placebo-controlled phase IIb clinical trial is being funded by a $5 million grant."

About Adeona
Adeona Pharmaceuticals, Inc. ("Adeona") is a specialty pharmaceutical company that is developing proprietary, late-stage drug candidates for the treatment of central nervous system and autoimmune diseases. Adeona's strategy is to exclusively in-license clinical-stage drug candidates for the treatment of unmet medical diseases. Adeona is focused on developing products to treat, rheumatoid arthritis, multiple sclerosis, dry age-related macular degeneration (AMD), and fibromyalgia.

This press release contains forward-looking statements, within the meaning of Section 21E of the Securities Exchange Act of 1934, that reflect Adeona Pharmaceuticals, Inc.'s current expectations about its future results, performance, prospects and opportunities, including statements regarding our late stage pipeline of products and its focus on development of new disease modifying treatments for autoimmune diseases. Where possible, the Company has tried to identify these forward-looking statements by using words such as "anticipates," "believes," "intends," or similar expressions. These statements are subject to a number of risks, uncertainties and other factors that could cause actual events or results in future periods to differ materially from what is expressed in, or implied by, these statements including the risks set forth in our recent Form 10-Q and other filings with the Securities and Exchange Commission. We cannot assure you that we will be able to successfully develop or commercialize products based on our technologies, including, dnaJP1, TRIMESTA, Zinthionein, oral flupirtine, SOLOVAX, oral TTM, or CD4 inhibitors, particularly in light of the significant uncertainty inherent in developing, manufacturing and conducting preclinical and clinical trials of new pharmaceuticals, and obtaining regulatory approvals, that our technologies will prove to be safe and effective, that our cash expenditures will not exceed projected levels, that we will be able to obtain future financing or funds when needed, that product development and commercialization efforts will not be reduced or discontinued due to difficulties or delays in clinical trials or due to lack of progress or positive results from research and development efforts, that we will be able to successfully obtain any further grants and awards, maintain our existing grants which are subject to performance, that we will be able to patent, register or protect our technology from challenge and products from competition or maintain or expand our license agreements with our current licensors, or that our business strategy will be successful. All forward-looking statements made in this press release are made as of the date hereof, and the Company assumes no obligation to update the forward-looking statements included in this news release whether as a result of new information, future events, or otherwise.

This news release was distributed by GlobeNewswire, www.globenewswire.com
SOURCE: Adeona Pharmaceuticals, Inc.

Adeona Pharmaceuticals, Inc.
Nicholas Stergis, Chief Executive Officer
(734) 332-7800

Redington, Inc.
Investor Relations
Thomas Redington
(203) 222-7399

(C) Copyright 2008 GlobeNewswire, Inc. All rights reserved. End of Story

Former Immune Response Corp. files for liquidation

2008-10-29T10:18:00.002-06:00

By BRADLEY J. FIKES - Staff Writer | Wednesday, October 15, 2008 8:07 PM PDT ∞

CARLSBAD ---- The battered biotech company formerly known as The Immune Response Corp. finally has thrown in the towel.

Orchestra Therapeutics filed for Chapter 7 bankruptcy Monday in U.S. Bankruptcy Court for the Southern District of California. The company listed assets of $1.64 million and liabilities of $32.61 million.

Co-founded by the late Jonas Salk in 1986 to develop a vaccine to treat AIDS called Remune, Immune Response was one of North County's most famous biotech companies.

In the early '90s, as its vaccine showed signs of activity against HIV, the virus that causes AIDS, Immune Response became a Wall Street darling.

But the company failed in repeated attempts to prove Remune actually helped those infected with HIV. While its stock plummeted and investors lost interest, Immune Response kept searching for other products.

In April 2007, the company changed its name to Orchestra Therapeutics. The company's main hope was to develop a vaccine for multiple sclerosis, which it called NeuroVax.

But failing to make a profit through Remune, NeuroVax or any of its other drug candidates, Orchestra barely kept going, raising a few hundred thousand dollars at a time from various investors.

In its filing, Orchestra included a long list of creditor claims, including the city of Carlsbad, seeking $335; Delaware's Division of Corporations, seeking $288,000; Gazzard-St. Stephen's Research Ltd. in the U.K., seeking $195,000; Levit Zacks Accounting, seeking $59,000; the Nasdaq stock market, seeking $31,000; and Shred It in Vista, seeking an amount described as "unknown."

Contact staff writer Bradley J. Fikes at (760) 739-6641 or bfikes@nctimes.com.

Link Between Vaccine and MS Unproven

2008-10-01T10:37:00.002-06:00

TUESDAY, Sept. 30 (HealthDay News) -- Children vaccinated against hepatitis B probably are not at an increased risk of developing multiple sclerosis (MS) unless they were inoculated with a particular brand of the vaccine, according to a new study.

The French study found that children with MS were almost twice as likely to have received the vaccine called Engerix B three or more years before the disease's onset. Further studies will need to be done to determine whether the vaccine is a direct cause of the development of MS.

The study, which involved 349 children with MS and 2,941 children without the disease, is to be published in the Oct. 8 online issue of Neurology.

The U.S. Centers for Disease Control and Prevention has more about vaccinations.

SOURCE: American Academy of Neurology, news release, September 2008

Tysabri PML Cases Cause European Regulators to Recommend Stronger Warnings

2008-10-01T10:34:00.002-06:00

September 29th, 2008

The European Medicines Agency, an advisory body for drug regulators in Europe, recommended last week that the warning label for the multiple-sclerosis drug Tyasabri be updated following new reports of users developing a rare brain disease known as PML, or progressive multifocal leukoencephalopathy.

Tysabri (natalizumab) is an intravenous injection given every 28 days to treat Crohn’s Disease or multiple sclerosis (MS). It has been shown to prevent relapse, cognitive decline and vision loss which could be associated with multiple sclerosis.

Shortly after Tysabri was introduced in 2004, it was associated with side effects that could lead to PML, which is a potentially fatal brain infection. After three cases of PML resulted in two deaths, a Tysabri recall was issued in 2005, and the drug was re-introduced the next year with stricter guidelines for usage and more prominent warnings about the possible Tysabri PML side effects.

European and American drug regulators have been re-examing the risk of progressive multifocal leukoencephalopathy in recent months, after new reports of PML among Tysabri users surfaced in July 2008. According to filings with the SEC, the drugs makers, Biogen Idec and Elan Corporation, identified two PML cases in Europe, which were the first new diagnosed Tysabria PML cases since 2006.

On September 25, 2008, the European Medicines Agency’s Committee for Medical Products for Human Use (CHMP) called for drug regulators to strengthen the Tysabri progressive multifocal leukoencephalopathy warnings in Europe. They recommended that the drug’s prescription information be updated and strengthened to highlight the potential Tysabri side effects for patients with relapsing-remitting multiple sclerosis (MS).

The European regulatory committee did not call for a Tysabri recall, as they indicated that the benefits of the drug still do outweigh the risks. However, in light of these new PML cases, they felt that the current warnings in Europe need to be strengthened to raise awareness about the risk of the brain disease from Tysabri.

In August 2008, the FDA issued a MedWatch Alert regarding the new reports of PML associate with Tysabri. They indicated that the prescribing information in the United States would be revised to include information for prescribers and patients about the cases of PML that occurred among patients taking Tysabri as a monotherapy. The U.S. drug regulators also recommended that doctors should monitor their patients for signs and symptoms of PML.

that they are investigating the reports to determine what, if any, regulatory actions are necessary regarding the Tysabri side effects.

Progressive multifocal leukoencephalopathy is a rare viral infection which causes inflammation at multiple locations in the brain, leading to progressive brain damage. Symptoms could include loss of vision, impaired speech, paralysis, cognitive decline and weakness. There is no known cure for PML, but the disease can sometimes be slowed or stopped by reducing immunosuppression. In many cases the brain infection is fatal.

MS Patients Have Higher Spinal Fluid Levels Of Suspicious Immune Molecule

2008-10-01T10:30:00.000-06:00

ScienceDaily (Sep. 30, 2008) — A protein that helps keep immune cells quiet is more abundant in the spinal fluid of patients with multiple sclerosis (MS), further boosting suspicion that the protein, TREM-2, may be an important contributor to the disease.

More of an immune-control protein might seem like a boon to MS sufferers, whose symptoms are caused by misdirected immune attacks on the protective lining that coats nerve cell branches. But researchers at Washington University School of Medicine in St. Louis found the extra TREM-2 was not in the right place to reduce aggression in immune cells, a revelation that could eventually lead scientists to new pharmaceutical targets for MS prevention.

"Previously, TREM-2 had only been seen on the surface of immune cells; in the new study, we found it floating freely in spinal fluid," says lead author Laura Piccio, M.D., Ph.D., postdoctoral fellow. "This is only speculation for now, but these 'free agent' copies of TREM-2 could be making it harder for the TREM-2 that is attached to immune cells to keep the cells' aggressiveness under control."

Piccio explains that TREM-2 is a receptor protein, which means that another molecule activates it. Scientists don't currently know what that other molecule is, but the "free agent" TREM-2 in the spinal fluid could be binding to the molecule, reducing the chances that it will bind to and activate TREM-2 attached to immune cells. If Piccio and her colleagues can confirm their theory, the TREM-2 in the spinal fluid or its unknown partner could become targets for new MS treatments. The findings appear in the journal Brain.

Epidemiologists estimate that 400,000 people in the United States have MS. Symptoms, which often strike in episodic bursts, include bladder and bowel dysfunction, memory problems, fatigue, dizziness, depression, difficulty walking, numbness, pain and vision problems. The disease is more common among Caucasians than any other group and affects two to three times as many women as men.

TREM-2 first came to MS researchers' attention because of Nasu-Hakola disease, a rare genetic disorder that involves a mutation in the gene for TREM-2. Among other symptoms, Nasu-Hakola causes loss of the same protective sheath around nerve cell branches that is damaged by MS.

One place where the TREM-2 protein commonly appears is the macrophage, an immune cell that performs a variety of functions, including cleaning up debris and emitting inflammatory signals that escalate immune attacks. Macrophages come in two classes: one that promotes inflammation and one that suppresses it. TREM-2 is present only on the anti-inflammatory macrophages.

Prior experiments had shown that activation of the TREM-2 receptor can help reduce immune inflammation and promote phagocytosis, a process that lets cells consume things. In the context of the central nervous system, researchers think this allows macrophages to consume dying nerve cells and to perform "housekeeping functions," such as shutting down inflammatory processes.

"The main thing we knew about MS and the function of TREM-2 before this study was that blocking TREM-2 in a mouse model of MS made their conditions worse," says senior author Anne Cross, M.D., professor of neurology and head of the neuroimmunology section.

After Piccio identified TREM-2 in the spinal fluid, she compared that form of the protein in patients with various types of MS, patients with other inflammatory diseases of the central nervous system, and patients with non-inflammatory central nervous system diseases. To ensure that the soluble TREM-2 wasn't seeping into the patients' spinal fluid from the bloodstream, they also analyzed TREM-2 levels in blood.

While there were no differences in blood levels, the soluble form of TREM-2 was significantly higher in the spinal fluid of MS patients.

Scientists are trying to develop a mouse line where the TREM-2 gene has been disabled to learn more about the protein's contributions to the immune system.

Journal reference:

1. Piccio L, Buonsanti C, Cella M, Tassi I, Schmidt RE, Rinker II J, Naismith RT, Panina-Bordignon P, Passini N, Fenoglio C, Galimberti D, Scarpini E, Colonna M, Cross AH. Identification of soluble TREM-2 in the cerebrospinal fluid and its association with multiple sclerosis and CNS inflammation. Brain, 2008; DOI: 10.1093/brain/awn217

Adapted from materials provided by Washington University School of Medicine.

Acute Multiple-Sclerosis Relapses Rarely Result in Permanent Disability: Presented at ANA

2008-10-01T10:23:00.002-06:00

By Andrew Wilner, MD

SALT LAKE CITY, Utah -- September 24, 2008 -- Acute relapses in patients with relapsing-remitting multiple sclerosis (MS) rarely lead to disability, according to a retrospective chart review presented here at the American Neurological Association (ANA) 133rd Annual Meeting.

Loren Rolak, MD, Marshfield Clinic, Marshfield, Wisconsin, explained that many patients with MS fear they "might wake up paralyzed" from an acute relapse.

To assess the likelihood of severe disability from an acute relapse, Dr. Rolak examined the clinical course of 1,078 patients with relapsing-remitting MS over the last 14 years from his own database of patients with MS treated at Marshfield Clinic.

The patients in the database had a total of 2,587 attacks (mean of 2.4 attacks per patient, range: 1-14 attacks over 1-34 years). Only 7 of 1,078 patients (0.6%) had an attack that resulted in severe disability, defined as an Expanded Disability Status Scale (EDSS) score of 6 or more sustained for longer than 6 months.

Two of the 7 patients who had an attack that resulted in severe disability presented with an acute severe attack at the time of their diagnosis with MS. Of the remaining 5 patients, 2 were taking interferon beta-1b, which did not prevent the severe disability. The other 3 were not taking interferon or glatiramer-acetate therapy.

Genetic analysis failed to reveal any association between major histocompatibility complex class II DR beta 1 (HLA-DRB1) or nitric oxide synthase (NOS2A) genotypes and severe disability from an acute relapse.

Dr. Rolak concluded, "These results allow me to reassure my patients that a relapse with severe disability is extremely unlikely -- whether or not they are treated with disease-modifying therapy. Consequently, the fear of irreversible disability should not influence their decision to take interferons or other treatment."

[Presentation title: What Is the Risk of Permanent Disability From an MS Attack? Abstract T-91]

Magnetic Resonance Imaging Can Predict Who Will Develop Multiple Sclerosis: Presented at WCTRMS

2008-10-01T10:19:00.002-06:00

By Louise Gagnon

MONTREAL -- September 24, 2008 -- Specific magnetic resonance imaging (MRI) scans can predict which patients will develop multiple sclerosis (MS), according to retrospective research presented here at the World Congress on Treatment and Research in Multiple Sclerosis (WCTRMS).

The Betaferon/Betaseron in Newly Emerging MS for Initial Treatment (BENEFIT) study is a randomised, double-blind, placebo-controlled, parallel-group clinical trial that was carried out among 468 patients whose first clinical event suggestive of MS happened within 60 days of trial entry. The study found that treatment with interferon (INF) beta-1b 250 mcg prevented the onset of clinically definite multiple sclerosis (CDMS) by 1 year.

Patients in the BENEFIT study were assigned to either early treatment, which was IFN beta-1b from the start of the trial, or delayed treatment, which was initial placebo followed by IFN beta-1b therapy after conversion to CDMS or upon completing 2-year follow-up.

Principal investigator Bastiaan Moraal, MD, VU Medical Center, Amsterdam, Netherlands, speaking at an oral session here on September 19, said that this analysis examined radiological rather than clinical endpoints.

"We were looking at which type of lesions measured at baseline would predict conversion to clinically definite multiple sclerosis or McDonald multiple sclerosis," said Dr. Moraal.

In this analysis, blinded raters assessed baseline MRI parameters using T2-weighted and postcontrast T1-weighted sequences. Statistical analysis was employed to assess the predictive value of each baseline MRI parameter and treatment interaction.

Investigators found overall conversion to CDMS was 42%, with factors such as the presence of =>9 T2 lesions and =>3 periventricular lesions demonstrating predictive value. They found that conversion rose with the cumulative number of positive criteria. No specific advantage was demonstrated for a threshold of =>3 Barkhof criteria.

"Patients whose treatment was delayed and had 4 positive Barkhof criteria had a higher chance of conversion to CDMS and McDonald MS compared to those with early treatment and fewer positive Barkhof criteria at baseline," explained Dr. Moraal.

Investigators found that prognostic value was affected by treatment (P = .002) for 4 positive Barkhof criteria. The prognostic value was not influenced by therapy for CDMS.

"We saw that, with one exception, the predictive value of MRI values was not affected by treatment," said Dr. Moraal.

Future analysis will examine the predictive value of MRI variables at 3, 6, and 9 months to assess the impact of those variables on conversion to either CDMS or McDonald MS.

[Presentation title: Baseline Magnetic Resonance Imaging Predictors for Conversion to Clinically Definite Multiple Sclerosis and McDonald Multiple Sclerosis, Based on Integrated 3-Year Data From the BENEFIT Study. Abstract 51]

BIOMS MEDICAL ANNOUNCES RECEIPT OF MILESTONE PAYMENT FROM ELI LILLY AND COMPANY

2008-10-01T10:15:00.001-06:00

~ US$10 million received based on the positive review of interim analysis for international pivotal multiple sclerosis trial ~

Edmonton, Alberta, September 22, 2008 – BioMS Medical Corp. (TSX: MS), a leading developer in the treatment of multiple sclerosis (MS), today announced that it has received the US$10 million milestone payment from its partner, Eli Lilly and Company.

The milestone payment is based on the previously announced positive review of the scheduled interim analysis of the Company’s pivotal phase II/III Canadian and European trial (MAESTRO-01) of dirucotide (MBP8298) in patients with secondary progressive MS. The independent Data Safety Monitoring Board (DSMB) for the MAESTRO-01 trial recommended that the trial proceed to completion based on the interim analysis which included efficacy and safety data from the first 200 patients to complete the trial.

To date, BioMS has received a total of US$97 million in payments from Eli Lilly and Company, based on the licensing and development agreement for dirucotide (MBP8298). An additional $400 million in milestone payments are possible under the terms of the licensing agreement, in addition to escalating royalties on sales.

“We are pleased that our partner has recognized the outcome of the interim analysis as a significant positive event,” said Kevin Giese, President and CEO of BioMS Medical. “We look forward to completing the MAESTRO-01 trial and reviewing the complete data set in the second half of 2009.”

About BioMS Medical Corp.

BioMS Medical is a biotechnology company engaged in the development and commercialization of novel therapeutic technologies. BioMS Medical’s lead technology, dirucotide (MBP8298), is for the treatment of multiple sclerosis and is being evaluated in two pivotal phase III clinical trials for secondary progressive MS patients, MAESTRO-01 in Canada and Europe and MAESTRO-03 in the United States. It additionally is being evaluated for relapsing remitting MS patients in a Phase II trial in Europe entitled MINDSET-01. In December 2007, BioMS entered into a licensing and development agreement granting Eli Lilly and Company exclusive worldwide rights to dirucotide (MBP8298) in exchange for an $87 million upfront payment, milestone payments and escalating royalties on sales. For further information please visit our website at www.biomsmedical.com.

This press release may contain forward-looking statements, which reflect the Corporation’s current expectation regarding future events. These forward-looking statements involve risks and uncertainties that may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, changing market conditions, the successful and timely completion of clinical studies, the establishment of corporate alliances, the impact of competitive products and pricing, new product development, uncertainties related to the regulatory approval process and other risks detailed from time to time in the Corporation’s ongoing quarterly and annual reporting. Certain of the assumptions made in preparing forward-looking statements include but are not limited to the following: that dirucotide (MBP8298) will continue to demonstrate a satisfactory safety profile in ongoing and future clinical trials; and that BioMS Medical Corp. will complete the respective clinical trials within the timelines communicated in this release. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Ryan Giese
VP Corporate Communications
Phone: 780-413-7152
rgiese@biomsmedical.com

Tony Hesby
Executive VP Corporate Affairs
Phone: 780-413-7152
tony.hesby@biomsmedical.com

Amanda Stadel
Investor Relations Manager
Phone: 780-413-7152
astadel@biomsmedical.com

TYSABRI® DEMONSTRATES SUSTAINED IMPROVEMENT IN FUNCTIONAL OUTCOMES IN MULTIPLE SCLEROSIS PATIENTS ACCORDING TO NEW POST-HOC ANALYSIS

2008-10-01T10:13:00.001-06:00

TYSABRI is the Only Marketed MS Treatment to Show Both Significant Slowing in Disability Progression and Sustained Improvement in Physical Disability

Montreal, Canada - September 19, 2008 - Biogen Idec (NASDAQ: BIIB) and Elan Corporation, plc (NYSE: ELN) announced that a post-hoc analysis showed TYSABRI® (natalizumab) treatment increases the probability of achieving sustained improvement in physical disability over two years when compared to placebo. This post-hoc analysis provides the first evidence that TYSABRI is associated with a significant improvement in functional outcome, rather than only slowing or preventing progression of disability, in those living with relapsing multiple sclerosis (MS). These findings were derived from a subset analysis of the Phase III AFFIRM trial and were presented today as a poster presentation at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada. This is the first joint meeting of the Americas Committee on Treatment and Research in Multiple Sclerosis (ACTRIMS) and its counterparts in Europe and Latin America: ECTRIMS and LACTRIMS.

"These results show that TYSABRI treated patients are significantly more likely to experience a sustained improvement in disability compared to placebo patients. This finding from a post-hoc analysis of the pivotal AFFIRM trial supports both the earlier findings from the AFFIRM trial that TYSABRI is associated with an improvement in quality of life as well as anecdotal evidence of recovery of function in some patients." said Frederick E. Munschauer, MD, Smith Professor and Chair, Department of Neurology, State University of New York at Buffalo. "While, like TYSABRI, other therapies have shown a slowing of progression in disability, this analysis represents the first evidence supporting a sustained improvement in function associated with an approved disease modifying therapy."

Post-hoc Disability Analysis of Phase III AFFIRM Study

The proportion of patients exhibiting sustained improvements in physical disability in the AFFIRM study was determined based upon the Expanded Disability Status Scale (EDSS) over two years in patients with relapsing MS. EDSS is one of the oldest and most widely utilized methods of quantifying disability in MS.

Post-hoc analysis of AFFIRM patients assessed sustained improvement in disability among patients with a baseline EDSS score ≥ 2.0. Improvement in disability was defined as a one-point decrease in EDSS score sustained for 12 weeks. The cumulative probabilities of 12-week sustained improvement in disability at two years were estimated using the Kaplan-Meier method. Treatment effects were analyzed using the Cox proportional hazards model adjusted for baseline EDSS score. The distribution of sustained improvement by baseline EDSS score for each treatment group was also examined.

TYSABRI produced significant results on the cumulative probability of sustained improvement in disability in those treated over two years compared with placebo. In patients with a baseline EDSS score ≥ 2.0, the probability of achieving sustained improvement was 29.6% with TYSABRI (n=417) compared with 18.7% with placebo (n=203) (p=0.006). In patients with an EDSS score ≥ 2.0 and highly active disease at baseline, the difference between groups was even greater, 35.5% for TYSABRI (n=103) and 15.4% for placebo (n=40) (p=0.045).

The submitted abstract for this study, entitled "Natalizumab significantly increases the cumulative probability of sustained improvement in physical disability" (ID #P474), is available on the World Congressâ€™ website.
About TYSABRI

TYSABRI is a treatment approved for relapsing forms of MS in the US and relapsing-remitting MS in the European Union. According to data that have been published in the New England Journal of Medicine, after two years, TYSABRI treatment led to a 68% relative reduction (p<0.001) in the annualized relapse rate compared to placebo and reduced the relative risk of disability progression by 42-54% (p<0.001).

TYSABRI was approved in early 2008 to induce and maintain clinical response and remission in adult patients with moderately to severely active Crohn's disease (CD) with evidence of inflammation who have had an inadequate response to, or are unable to tolerate, conventional CD therapies and inhibitors of TNF-alpha. According to the US full prescribing information, among patients who responded to TYSABRI, 54% sustain their response through every visit for one year compared to 20% of patients receiving placebo (p<0.001), for a treatment difference of 34%.

TYSABRI increases the risk of progressive multifocal leukoencephalopathy (PML), an opportunistic viral infection of the brain that usually leads to death or severe disability. Cases of PML have been reported in patients taking TYSABRI who were recently or concomitantly treated with immunomodulators or immunosuppressants, as well as in patients receiving TYSABRI as monotherapy. Other serious adverse events that have occurred in TYSABRI-treated patients included hypersensitivity reactions (e.g., anaphylaxis) and infections. Serious opportunistic and other atypical infections have been observed in TYSABRI-treated patients, some of whom were receiving concurrent immunosuppressants. Herpes infections were slightly more common in patients treated with TYSABRI. In MS and CD clinical trials, the incidence and rate of other serious adverse events, including serious infections, were similar in patients receiving TYSABRI and those receiving placebo. Common adverse events reported in TYSABRI-treated MS patients include headache, fatigue, infusion reactions, urinary tract infections, joint and limb pain and rash. Other common adverse events reported in TYSABRI-treated CD patients include respiratory tract infections and nausea. Clinically significant liver injury has been reported in patients treated with TYSABRI in the post-marketing setting.

TYSABRI is approved in more than 35 countries. At the end of June 2008, more than 31,800 patients were on commercial and clinical TYSABRI therapy worldwide. Patients on TYSABRI therapy have continued to increase. An update will be provided in October in conjunction with Biogen Idec's third quarter earnings release.

For more information about TYSABRI please visit www.tysabri.com, www.biogenidec.com or www.elan.com or call 1-800-456-2255.
About Elan

Elan Corporation, plc is a neuroscience-based biotechnology company committed to making a difference in the lives of patients and their families by dedicating itself to bringing innovations in science to fill significant unmet medical needs that continue to exist around the world. Elan shares trade on the New York, London and Dublin Stock Exchanges. For additional information about the company, please visit www.elan.com.
About Biogen Idec

Biogen Idec creates new standards of care in therapeutic areas with high unmet medical needs. Founded in 1978, Biogen Idec is a global leader in the discovery, development, manufacturing, and commercialization of innovative therapies. Patients in more than 90 countries benefit from Biogen Idec's significant products that address diseases such as lymphoma, multiple sclerosis, and rheumatoid arthritis. For product labeling, press releases and additional information about the company, please visit www.biogenidec.com.

For more information contact:

Media Contacts:

Biogen Idec
Shannon Altimari
617-914-6524

Elan
Jonathan Birt
212-850-5664
or
+44 20 7269 7205

Niamh Lyons
Ph: +353 1 663 3602

Investor Contacts:

Biogen Idec
Eric Hoffman
617-679-2812

Elan
Chris Burns
+ 353 1 709 4444
800-252-3526

David Marshall
Ph: +353 1 709 4444

Results from IMPROVE Study Show Therapeutic Effect of New Formulation of Rebif® at 16 Weeks in Patients with Multiple Sclerosis

2008-10-01T10:04:00.004-06:00

• Study meets primary endpoint by demonstrating significant effect of new formulation of Rebif® on disease activity as measured by MRI after 16 weeks of treatment

• Data presented at late-breaking session of the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada

Geneva, Switzerland, September 22, 2008 - Merck Serono, a division of Merck KGaA, Darmstadt, Germany, announced today that the ongoing IMPROVE (Investigating MRI Parameters with Rebif imprOVEd formulation) study met its primary endpoint. The primary objective of the study was to evaluate the efficacy of the new formulation of Rebif®, compared to placebo, in patients with relapsing-remitting multiple sclerosis (RRMS) and active disease by means of magnetic resonance imaging (MRI) at the end of 16 weeks of treatment. The 16-week study results show that the mean number of combined unique active brain MRI lesions per patient was reduced by 69% in patients treated with the new formulation of Rebif® compared with those receiving placebo, a statistically significant result (p<0.001). These data were presented at the late-breaking session of the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada.

"Patients who received Rebif® experienced far fewer new active brain MRI lesions than the placebo group after 16 weeks of treatment,” said Dr. Mark Freedman, Professor of Neurology at the University of Ottawa, Director of the MS Research Clinic at the Ottawa Hospital, and an investigator of the IMPROVE trial. “These data demonstrate a
significant effect of the new formulation of Rebif® on disease activity and provide further evidence of its benefit in treating patients with relapsing-remitting multiple sclerosis.”

The IMPROVE study is a two-arm, randomized, double-blind, controlled, multicenter, international Phase IIIb study to evaluate the efficacy, safety and tolerability of the new formulation of Rebif® in patients with RRMS according to the revised McDonald criteria and evidence of active disease. A total of 180 patients were randomized in a 2:1 ratio to receive either the new formulation of Rebif® 44 micrograms three times a week subcutaneously, or placebo for an initial period of 16 weeks. At the end of this initial 16-week treatment period, patients from the placebo group have been switched in a single-blinded fashion to treatment with the new formulation of Rebif® 44 micrograms three times a week subcutaneously for a period of 24 weeks (the physician assessing treatment response and side effects is blinded). Patients who were initially assigned to the new formulation of Rebif® group continue to receive active treatment for an additional period of 24 weeks. The duration of the whole treatment period is 40 weeks.

The primary endpoint of the study is the difference between the number of combined unique active MRI lesions at week 16 in the group treated with the new formulation of Rebif® versus the placebo group. Combined unique active MRI lesions are defined as an active lesion on T1 sequence with gadolinium or T2 sequence, or both, avoiding double counting. The primary endpoint mainly reflects inflammatory activity (gadolinium-enhancing T1 lesions), but also reflects disease progression (T2 lesions).

The primary efficacy analysis showed that, at week 16, the number of combined unique active brain lesions was significantly lower in patients treated with the new formulation of Rebif® than in patients who received a placebo (p<0.001). The mean number of combined unique active brain lesions per patient was reduced by 69% in patients treated with the new formulation of Rebif® compared with those receiving placebo (0.7 versus 2.2). The median number of combined unique active brain lesions at week 16 was 0.0 in the group treated with the new formulation of Rebif® and 1.0 in the placebo group. Over half (53%) of patients treated with the new formulation of Rebif® had zero combined unique active brain lesions at week 16, compared to only 16.7% in the placebo group.

Results for the secondary and tertiary endpoints of the IMPROVE study will be available at the end of the 40-week treatment period.

The safety profile of the new formulation of Rebif® reported in this study is consistent with the known safety profile of Rebif®. No unexpected safety concerns were identified in this study.

The new formulation of Rebif® was approved in the European Union in August 2007 and in Canada in September 2007. It is now marketed in all EU countries and in Canada. The new formulation of Rebif® is not available in the United States.

About Rebif®

Rebif® (interferon beta-1a) is a disease-modifying drug used to treat relapsing forms of multiple sclerosis (MS) and is similar to the interferon beta protein produced by the human body. The efficacy of Rebif® in chronic progressive MS has not been established. Interferons are thought to help modulate the body’s immune system and reduce inflammation. The exact mechanism is unknown.

Rebif®, which was approved in Europe in 1998 and in the US in 2002, is registered in more than 80 countries worldwide. Rebif® has been proven to delay the progression of disability, reduce the frequency of relapses and reduce MRI lesion activity and area.* Rebif® is available in a 22 micrograms and 44 micrograms ready-to-use pre-filled syringe and a titration pack (8.8 micrograms).

Rebif® should be used with caution in patients with a history of depression, liver disease and seizures. Most commonly reported side effects are flu-like symptoms, injection site disorders, elevation of liver enzymes and blood cell abnormalities. Patients, especially those with depression, seizure disorders, or liver problems, should discuss treatment with Rebif® with their doctors. For more information about Rebif®, please visit www.mslifelines.com for prescribing information.

* The exact correlation between MRI findings and the current or future clinical status of patients, including disability progression, is unknown.

About Merck Serono and multiple sclerosis

Merck Serono is a leader in multiple sclerosis (MS) with Rebif® (interferon beta-1a), a disease-modifying drug used to treat relapsing forms of MS, which is registered in more than 80 countries worldwide. Full prescribing information for Rebif® can be obtained by contacting the Company or visiting its website. Additional therapeutic options are currently under development at Merck Serono, including oral cladribine, currently in Phase III and potentially the first oral therapy for MS, as well as several products in early stage development. Merck Serono also is taking a leading role in developing an understanding of the role of genetics in MS.

About multiple sclerosis

Multiple sclerosis (MS) is a chronic, inflammatory condition of the nervous system and is the most common, non-traumatic, disabling, neurological disease in young adults. The World Health Organization estimates that up to 2.5 million people suffer from MS worldwide. While symptoms can vary, the most common symptoms of MS include blurred vision, numbness or tingling in the limbs and problems with strength and coordination. The relapsing forms of MS are the most common.

About Merck Serono

Merck Serono is the division for innovative prescription pharmaceuticals of Merck, a global pharmaceutical and chemical group. Headquartered in Geneva, Switzerland, Merck Serono discovers, develops, manufactures and markets innovative small molecules and biopharmaceuticals to help patients with unmet medical needs. Its North American business operates in the United States and Canada as EMD Serono.

Merck Serono has leading brands serving patients with cancer (Erbitux®), multiple sclerosis (Rebif®), infertility (Gonal-f®), endocrine and cardiometabolic disorders (Glucophage®, Concor®, Euthyrox®, Saizen®, Serostim®), as well as psoriasis (Raptiva®).
With an annual R&D expenditure of around € 1bn, Merck Serono is committed to growing its business in specialist-focused therapeutic areas including neurodegenerative diseases, oncology, fertility and endocrinology, as well as new areas potentially arising out of research and development in autoimmune and inflammatory diseases.

About Merck

Merck is a global pharmaceutical and chemical company with total revenues of € 7.1 billion in 2007, a history that began in 1668, and a future shaped by 31,946 employees in 60 countries. Its success is characterized by innovations from entrepreneurial employees. Merck's operating activities come under the umbrella of Merck KGaA, in which the Merck family holds an approximately 70% interest and free shareholders own the remaining approximately 30%. In 1917 the U.S. subsidiary Merck & Co. was expropriated and has been an independent company ever since.

For more information, please visit www.merckserono.net or www.merck.de

Data From MediciNova's Two-Year Phase II Clinical Trial of MN-166 in Multiple Sclerosis Presented At the World Congress for Treatment and Research in

2008-10-01T10:01:00.002-06:00

SAN DIEGO, Sep 18, 2008 (GlobeNewswire via COMTEX News Network) -- MediciNova, Inc., a biopharmaceutical company that is publicly traded on the Nasdaq Global Market (Nasdaq:MNOV) and the Hercules Market of the Osaka Securities Exchange (Code Number: 4875), today announced that data from the completed two-year Phase II clinical trial of orally administered MN-166 for the treatment of multiple sclerosis (MS) was presented today in two poster presentations at the World Congress for Treatment and Research in MS being held in Montreal, Canada.

The first presentation entitled "Clinical Effect of the Neuroprotectant MN-166 in Relapsing Forms of MS: Year 2 Data" (Poster 48), given by Dr. Richard Gammans, Chief Development Officer of MediciNova, reported that MN-166 treatment resulted in positive findings on three independent measures indicative of a potential disease-progression modifying effect. The poster can be accessed at: http://www.medicinova.com/MN-166-Relapsing-MS.html. The findings include:

* Sustained disability progression was significantly less likely (by approximately 50 percent) in those patients receiving MN-166 at either 30 or 60 mg per day for 24 months than in those patients receiving the drug for 12 months (p=0.026). Sustained disability progression was measured as a greater than or equal to 1.0 point increase from baseline in the Expanded Disability Status Scale (EDSS) score for four consecutive months. This positive clinical finding was corroborated by positive findings on two separate radiologic measures.

* The Phase II clinical trial demonstrated that the significant reduction in brain volume loss (p=0.035), as measured by cranial magnetic resonance imaging (MRI) scans, observed after 12 months in patients treated with 60 mg per day of MN-166 compared to placebo was again demonstrated in year two of the study. Brain volume loss was significantly less (p=0.030) in patients who received 60 mg per day of MN-166 for 24 months compared to the other treatment groups.

The second presentation entitled "Ibudilast Reduces Conversion of New Inflammatory Lesions to Persistent Black Holes in Active Relapsing Multiple Sclerosis" (Poster 271), given by Dr. Hanneke Hulst of VU University Medical Center, Amsterdam, The Netherlands, reported that the Phase II data demonstrated that MN-166 treatment resulted in positive findings on independent MRI measures indicative of a neuroprotective effect. The findings include:

* MN-166 treatment at 60 mg per day significantly reduced the relative risk for conversion of new inflammatory lesions identified at month two to Persistent Black Holes (PBH), an MRI indicator of neuronal loss, eight months later at month ten by 37 percent (p=0.011); such lesions that remain unchanged for eight months are considered PBHs as compared to transient inflammatory lesions that are more closely associated with relapses. MN-166 treatment at 30 mg per day resulted in a trend toward reducing evolution to PBH (p=0.074). Loss of brain volume and development of PBHs on MRI have been shown to correlate with clinical progression and disability in MS patients.

"We are pleased that the WCTRIMS Scientific Program Committee accepted two presentations on different aspects of the results from our recently completed Phase II clinical trial of MN-166," said Yuichi Iwaki, M.D., Ph.D., President and Chief Executive Officer of MediciNova, Inc. "The significant favorable effects on measures of disability progression and reduced neuronal damage observed in the study, as described in these presentations, are quite exciting and representative of the type of new treatment being sought by the MS scientific community according to our advisors. We are excited to be part of advancing MS treatment in a new direction and look forward to confirming these findings in future clinical trials with the assistance of a corporate partner."

The two-year randomized, double-blind, placebo-controlled Phase II clinical trial included 297 patients with relapsing MS. In the second year of the study, all patients were on drug. Patients who received 30 or 60 mg of MN-166 per day during the first 12 months of the study remained on the assigned dose for the second 12 months of the study; patients who received placebo during the first 12 months of the study were randomized to receive either 30 or 60 mg of MN-166 per day (double-blind maintained) during the second 12 months of the study. Clinical and radiological outcomes were evaluated.

MN-166 was well tolerated at all doses over the 24 months of this Phase II clinical trial. Of the 297 patients enrolled in the study, 82.5 percent, or 245 patients, completed the full 24 months of the study. The most common adverse events possibly related to MN-166 included mild, transient gastrointestinal disturbances and depression.

First-year efficacy results of this clinical trial were announced in March 2007 and described more completely at the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in November 2007. Clinical results from the completed two-year clinical trial were announced in April 2008.

About MN-166

MN-166 is a novel, orally administered compound being evaluated for the treatment of MS. MN-166 increases the release of neuronal growth factors and inhibits leukotriene activity, phosphodiesterases and nitric oxide synthase. MN-166 may also suppress the production of pro-inflammatory cytokines (IL-1beta, TNF-alpha) and may enhance the production of the anti-inflammatory cytokines (IL-4, IL-10).

MediciNova acquired an exclusive, worldwide (excluding Japan, China, Taiwan and South Korea), sublicensable license to MN-166 for the treatment of MS, excluding ophthalmic solution formulations, from Kyorin Pharmaceutical Co. Ltd. For the past 18 years, MN-166 has been marketed in Japan and South Korea as Ketas(r) for the treatment of asthma and cerebrovascular disorders. Data from the existing clinical trial and post-marketing surveillance databases, which includes treatment of an estimated 3.2 million patients with these disorders, indicate that Ketas(r) is well tolerated.

About MediciNova

MediciNova, Inc. is a publicly-traded biopharmaceutical company focused on acquiring and developing novel, small-molecule therapeutics for the treatment of diseases with unmet need with a specific focus on the U.S. market. Through strategic alliances primarily with Japanese pharmaceutical companies, MediciNova holds rights to a diversified portfolio of clinical and preclinical product candidates, each of which MediciNova believes has a well-characterized and differentiated therapeutic profile, attractive commercial potential and patent assets having claims of commercially adequate scope. MediciNova's pipeline includes six clinical-stage compounds for the treatment of acute exacerbations of asthma, multiple sclerosis, asthma, interstitial cystitis, solid tumor cancers, Generalized Anxiety Disorder, preterm labor and urinary incontinence and two preclinical-stage compounds for the treatment of thrombotic disorders. MediciNova's current strategy is to focus its resources on the development and commercialization of two prioritized assets in its development pipeline: MN-221 for the treatment of acute exacerbations of asthma and MN-166 for the treatment of multiple sclerosis. MediciNova will seek to monetize its other product candidates at key value inflection points. For more information on MediciNova, Inc., please visit www.medicinova.com.

The MediciNova, Inc. logo is available at http://www.globenewswire.com/newsroom/prs/?pkgid=3135

Statements in this press release that are not historical in nature constitute forward-looking statements within the meaning of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, without limitation, statements regarding MediciNova's clinical trials supporting safety and efficacy of product candidates and the potential novelty of such product candidates as treatments for disease, plans and objectives for present and future clinical trials and product development, strategies, future performance, expectations, assumptions, financial condition, liquidity and capital resources. These forward-looking statements may be preceded by, followed by or otherwise include the words "believes," "expects," "anticipates," "intends," "estimates," "projects," "can," "could," "may," "would," or similar expressions. These forward-looking statements involve a number of risks and uncertainties that may cause actual results or events to differ materially from those expressed or implied by such forward-looking statements. Factors that may cause actual results or events to differ materially from those expressed or implied by these forward-looking statements, include, but are not limited to, the risks and uncertainties inherent in clinical trials and product development and commercialization, such as the uncertainty in results of clinical trials for product candidates, the uncertainty of whether the results of clinical trials will be predictive of results in later stages of product development, the risk of delays or failure to obtain or maintain regulatory approval, the risk of failure of the third parties upon whom MediciNova relies to conduct its clinical trials and manufacture its product candidates to perform as expected, the risk of increased cost and delays due to delays in the commencement, enrollment, completion or analysis of clinical trials or significant issues regarding the adequacy of clinical trial designs or the execution of clinical trials and the timing, cost and design of future clinical trials and research activities, the timing of expected filings with the FDA, MediciNova's failure to execute strategic plans or strategies successfully, MediciNova's collaborations with third parties, the availability of funds to complete product development plans and MediciNova's ability to raise sufficient capital when needed, intellectual property or contract rights, and the other risks and uncertainties described in MediciNova's filings with the Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2007 and its subsequent periodic reports on Forms 10-Q and 8-K. Undue reliance should not be placed on these forward-looking statements, which speak only as of the date hereof. MediciNova disclaims any intent or obligation to revise or update these forward-looking statements.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: MediciNova, Inc.

MediciNova, Inc.
Shintaro Asako, Chief Financial Officer
858-373-1500
info@medicinova.com

Rx Communications, LLC
Rhonda Chiger
(917) 322-2569
rchiger@rxir.com

ATL/TV1102 Trial Results Presented at World Congress on Treatment and Research in Multiple Sclerosis

2008-09-22T10:04:00.002-06:00

MELBOURNE, Australia and CARLSBAD, Calif., Sept 22, 2008 /PRNewswire-FirstCall via COMTEX News Network/ -- Antisense Therapeutics Ltd. (ASX: ANP) and Isis Pharmaceuticals, Inc. (Nasdaq: ISIS) are pleased to advise that the results from the ATL/TV1102 Phase IIa clinical trial in patients with multiple sclerosis (MS) were presented Saturday at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada by the Principal Investigator for the trial, Volker Limmroth, M.D., Ph.D., Chairman of the Department of Neurology, Cologne City Hospitals, Germany.

As previously reported, ATL/TV1102 significantly reduced disease activity in patients with Relapsing Remitting Multiple Sclerosis (RRMS) in a randomized, double-blind, placebo-controlled Phase II study designed to obtain evidence of ATL/TV1102's effectiveness in reducing MS-related brain lesions as detected by magnetic resonance images (MRI). The study met its primary endpoint showing a significant reduction by 54.4% (p=0.01) in cumulative number of new active lesions in the ATL/TV1102 treated patients compared to placebo. Further details on the study design and outcomes are provided in the conference abstract which follows this document.

Dr. Limmroth said, "As VLA-4 is a clinically validated target in MS, there is high interest in the potential application of treatment modalities directed against this target. In our study we were able to demonstrate, for the first time, that a second-generation antisense drug designed to specifically inhibit the production of VLA-4 could significantly reduce disease activity in RRMS patients as early as 8 weeks. The level of efficacy achieved by ATL/TV1102 is very promising particularly when considering the short duration of dosing within the trial. As an antisense drug ATL/TV1102 will have a different, potentially preferable, safety profile to other drugs that target VLA-4 and therefore presents an exciting future treatment option for RRMS patients."

Mark Diamond, Chief Executive Officer of Antisense Therapeutics, said, "We are delighted to have our ATL/TV1102 Phase IIa trial results presented at such an important scientific meeting on MS. The development of ATL/TV1102 as an MS treatment continues with our partner Teva Pharmaceutical Industries (Teva) and we look forward to the prospect of Teva reporting on ATL/TV1102's future clinical progress. Importantly, the demonstration of significant disease activity with ATL/TV1102 in MS points to the potential of ATL/TV1102 in other disease settings such as autoimmune, inflammation and cancer. It also provides important validation for the application of second-generation antisense drugs outside of cardiovascular and metabolic diseases such as high cholesterol and diabetes where there has been success to date. We believe our results provide important substantiation for the broader clinical application of the second-generation technology."

"We are very encouraged by the data. Not only do they represent promising results for patients with MS, but they also evidence the significant progress we are making in demonstrating the effectiveness, efficiency and the breadth of opportunity of our antisense platform. It's another disease and another tissue where we have great proof-of-concept for antisense drugs," said Stanley Crooke, M.D., Ph.D., Chairman and Chief Executive Officer of Isis. "ATL/TV1102 and ATL's strategic relationship with Teva to move the drug forward further validates our business strategy. ATL has successfully moved the drug forward and now has established a very experienced partner to complete development and commercialization of the drug. This strategy benefits patients as well as Isis' pipeline, platform and balance sheet."

The presentation has been webcasted and will be available on http://www.msmontreal.org/home/default_e.asp in the coming weeks. The Company will provide login details on its website http://www.antisense.com.au once these are confirmed.

Conference abstract follows:

VLA-4 Antisense - An Oligonucleotide targeting VLA-4 mRNA (ATL1102) significantly reduces new active lesions in patients with RR-MS

Volker Limmroth(1), Frederik Barkhof(2), Nuket Desem(3)

(1) Department of Neurology, Cologne City Hospitals, University of Cologne, Cologne, Germany, (2) Department of Radiology, MS Center Amsterdam, VU University Medical Center, Amsterdam, Netherlands, (3) Antisense Therapeutics Ltd, Melbourne, VIC, Australia.

Background: Antisense oligonucleotides (ASOs) are an innovative new class of drugs that inhibit the expression of proteins by sequence-specific binding to the protein's mRNA. ATL1102 is a 2nd generation antisense inhibitor of CD49d, a subunit of Very Late Antigen 4 (VLA-4) which plays a key role in cell adhesion to vessel walls. VLA-4 blockade, as shown by monoclonal antibodies such as natalizumab, prevents activated lymphocytes from migrating into the CNS and significantly reduces disease activity in MS.

Objective: To evaluate VLA-4 Antisense (ATL1102) in the treatment of RR-MS

Methods: Randomized, double-blind, placebo-controlled multicenter Phase-IIa trial. 77 patients with RR-MS were treated for 8 weeks with either 200mg of ATL1102 or placebo subcutaneously twice weekly and evaluated for 16 weeks. MRI scans were performed at screening, and then monthly over 16 weeks. Primary efficacy variable: cumulative number of new active lesions (CNNAL; new gadolinium-enhancing T1 lesions (T1-Gd), new or enlarging T2 lesions) on MRIs taken at weeks 4, 8 and 12. Secondary efficacy variable: cumulative volume of T1-Gd lesions (CVT1L) on MRIs taken at weeks 4, 8 and 12.

Results: ITT population: 74 patients with a valid baseline MRI and at least one post-baseline MRI scan after first injection of study medication (n=39 placebo, n=35 ATL1102). ATL1102 showed a significant reduction, 54.4%, in CNNAL (6.2 placebo, 3.0 ATL1102; p=0.01). A reduction of 66.7% (p=0.002) was observed in the cumulative number (weeks 4,8,12) of new T1-Gd lesions with ATL1102. A reduction in CVT1L was also observed under ATL1102 but did not reach significance (589.4 mm3 placebo, 358.0 mm3 ATL1102; p=0.1068). Adverse events that were more frequent under ATL1102 included mild to moderate injection site reactions and a tendency for decreased platelet counts which were reversible after treatment interruption.

Conclusions: This proof-of-concept study of a drug designed to inhibit VLA-4 mRNA showed a significant reduction of the cumulative number of new active lesions in RR-MS patients following 8 weeks of treatment. These promising results warrant further investigation.

Background Information

ATL/TV1102 Phase IIa trial is a randomised, double-blind, placebo-controlled clinical trial of ATL/TV1102. Patients received either ATL/TV1102 or placebo injections over 8 weeks, and were monitored with monthly MRI (magnetic resonance imaging) brain scans during treatment, and for the 8 weeks following treatment. 77 patients were enrolled in the trial, which was conducted at multiple trial sites across six European countries.

Multiple sclerosis is a lifelong chronic disease of the central nervous system which is believed to affect as many as 2.5 million people worldwide. Global drug sales for MS are in excess of $US 6 billion and are expected to grow with the introduction of novel treatment options. There remains a high demand for more effective and better tolerated treatments.

About ATL/TV1102 for MS: ATL/TV1102 is an antisense drug discovered by Isis Pharmaceuticals, Inc. and licensed to ANP. Antisense drugs specifically block disease-causing proteins from being produced by interacting with their intended target based on information in the genetic code. ATL/TV1102 is a second-generation antisense inhibitor of CD49d, a subunit of VLA-4 (Very Late Antigen-4). In inflammation, white blood cells (leukocytes) move out of the bloodstream into the inflamed tissue, for example, the CNS in MS, and the lung airways in asthma. The inhibition of VLA-4 may prevent white blood cells from entering sites of inflammation, thereby halting progression of the disease. VLA-4 is a clinically validated target in the treatment of MS. Antisense inhibition of VLA-4 has demonstrated positive effects in a number of animal models of inflammatory disease including MS (Myers et al. J Neuroimmunol 160, p12-24, 2005).

About Antisense Therapeutics Limited: Antisense Therapeutics Limited (ASX: ANP) is an Australian publicly listed biopharmaceutical drug discovery and development company. Its mission is to create, develop and commercialize antisense pharmaceuticals for large unmet markets. ANP has two drugs in development and two drugs in pre-clinical research. ATL/TV1102 (injection) has completed a Phase IIa trial as a potential treatment of multiple sclerosis. ATL1103 is a second-generation antisense drug designed to lower blood IGF-I levels and is entering pre-clinical development as a potential treatment for acromegaly and vision disorders. ATL/TV1102 (inhaled) is at the pre-clinical research stage as a potential treatment for asthma. ATL1101 is a second-generation antisense drug at the pre-clinical research stage being investigated as a potential treatment for prostate cancer. ATL/TV1102 has been licensed to Teva Pharmaceutical Industries Ltd.

About Isis Pharmaceuticals, Inc.

Isis is exploiting its expertise in RNA to discover and develop novel drugs for its product pipeline and for its partners. The Company has successfully commercialized the world's first antisense drug and has 19 drugs in development. Isis' drug development programs are focused on treating cardiovascular and metabolic diseases. Isis' partners are developing antisense drugs invented by Isis to treat a wide variety of diseases. Ibis Biosciences, Inc., Isis' majority-owned subsidiary, is developing and commercializing the Ibis T5000(TM) Biosensor System, a revolutionary system to identify infectious organisms. Isis is a joint owner of Regulus Therapeutics LLC, a joint venture focused on the discovery, development and commercialization of microRNA therapeutics. As an innovator in RNA-based drug discovery and development, Isis is the owner or exclusive licensee of over 1,500 issued patents worldwide. Additional information about Isis is available at http://www.isispharm.com.

Isis Forward-Looking Statement

This press release includes forward-looking statements regarding Isis' business, its drug discovery and development pipeline, and the therapeutic potential of ATL/TV1102 for the treatment of multiple sclerosis. Any statement describing Isis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement, including those statements that are described as Isis' goals. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such products. Isis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis' programs are described in additional detail in Isis' annual report on Form 10-K for the year ended December 31, 2007, and its most recent quarterly report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from Isis.

Isis Pharmaceuticals is a registered trademark of Isis Pharmaceuticals, Inc. Ibis Biosciences and Ibis T5000 are trademarks of Ibis Biosciences, Inc. Regulus Therapeutics is a trademark of Regulus Therapeutics LLC.

SOURCE Isis Pharmaceuticals, Inc.; Antisense Therapeutics Ltd.

New data presented at WCTRIMS* supports the importance of early and sustained treatment with Betaseron®

2008-09-22T09:59:00.002-06:00

Earlier treatment initiation and longer exposure to Betaseron was associated with improved long-term outcomes in multiple sclerosis

MONTREAL, CANADA, September 19, 2008 Data presented at the World Congress on Treatment and Research in Multiple Sclerosis (WCTRIMS) demonstrated that early initiation of Betaseron� (interferon beta-1b) treatment had a greater impact on long-term outcomes, when compared to delayed treatment.

The study, sponsored by Bayer HealthCare Pharmaceuticals, used data from the 16-Year Long-Term Follow-up Study of Betaseron to investigate the relationship between timing of drug initiation and length of exposure to treatment, and long-term outcomes. It demonstrated that initiating Betaseron treatment early in the disease reduced the risk of negative long-term outcomes, including conversion to secondary progressive multiple sclerosis (SPMS), reaching a confirmed EDSS of 6.0 or the use of a wheelchair. The study also found that the longer patients stayed on treatment, the better their long-term outcomes were.1

"The new analysis confirmed that in MS, timing of treatment is important. The findings showed that even if two patients are treated for an equivalent length of time, the one who started therapy earlier in the disease course had a better long-term outcome," said Douglas Goodin, MD, Director of the Multiple Sclerosis Center at UCSF Medical Center. "Patients and physicians should take these results into consideration when making treatment decisions."

A second study, called CogniMS, also presented at WCTRIMS, demonstrated that cognitive deficits can be measured early in the course of MS. The investigators suggested that such cognitive deficits may be clinically important for MS management decisions.

"Studies are now showing that apart from disease progression and relapse rates, cognition is another area that is impacted early in the course of the disease. In fact, there is a correlation between the level of disability at the start of treatment and cognitive function 16 years later. More research is needed to determine how treatment might benefit long-term cognitive outcome," said Dr. Goodin.

"The data presented here underscore the need to help patients start therapy earlier and stay on treatment for the long-term," said, Ludger Heeck, Ph.D. Vice President and General Manager, Specialty Medicine Bayer HealthCare Pharmaceuticals Inc. "Bayer is committed to helping provide both the medication and the support services that people need to help treat their MS. We pioneered the concept of customized MS support services, and our best-in-class BETAPLUSTM program goes far beyond treatment to offer a wide range of beneficial services for people with MS. From having dedicated MS nurses who can provide practical help and advice, to offering product enhancements like our new thinnest needle and optional autoinjector that can help make injection administration more comfortable, we continue to lead the way in helping people with MS start on and stay on treatment."

About the Trials
The 16-Year Long-term Follow-up Study is a multicenter observational study that collected data from patients with relapsing-remitting MS (RRMS) who participated in the pivotal North American trials for Betaseron. Several statistical methods were used to assess patient data and examine the relationship between timing of drug exposure and long-term outcomes. Drug exposure was measured as the medication possession ratio (MPR) defined as the actual time the patient received therapy divided by the total time possible before a negative outcome was reached (or at data censor). A statistical method called recursive partitioning was then used to divide treatment groups into "high" or "low" exposure and to determine the relationship between length of drug exposure and long-term outcomes. The use of MPR reduces the bias introduced in long-term trials by the tendency of patients who are doing well on therapy stay on therapy and for patients who are doing poorly to stop a particular therapy. Other statistical approaches including Propensity Scoring were used to control for other known sources of bias.1

CogniMS is a two-year observational study involving 1509 patients with early MS (diagnosed within two years) who were treated with Betaseron and assessed every six months using tests to measure cognition, fatigue and health-related quality of life. The trial includes patients from 32 countries2 and is currently ongoing.

About Betaseron
Betaseron is indicated for the treatment of relapsing forms of multiple sclerosis to reduce the frequency of clinical exacerbations. Patients with multiple sclerosis in whom efficacy has been demonstrated include patients who have experienced a first clinical episode and have MRI features consistent with multiple sclerosis.

The most commonly reported adverse reactions are lymphopenia, injection-site reaction, asthenia, flu-like symptom complex, headache and pain. Gradual dose titration and use of analgesics during treatment initiation may help reduce flu-like symptoms. Betaseron should be used with caution in patients with depression. Injection-site necrosis has been reported in four percent of patients in controlled trials. Patients should be advised of the importance of rotating injection sites. Female patients should be warned about the potential risk to pregnancy. Cases of anaphylaxis have been reported rarely. See "Warnings," "Precautions," and "Adverse Reactions" sections of full Prescribing Information. More information, including the full Prescribing Information, is available at www.betaseron.com.

About Bayer HealthCare Pharmaceuticals Inc.
Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare is one of the world's leading, innovative companies in the healthcare and medical products industry, and combines the activities of the Animal Health, Consumer Care, Diabetes Care, and Pharmaceuticals divisions. Bayer HealthCare Pharmaceuticals comprises the following business units: Women's Healthcare, Diagnostic Imaging, General Medicine, which includes Cardiology and Primary Care and Specialty Medicine, which includes Hematology, Oncology and Multiple Sclerosis. The company's aim is to discover and manufacture products that will improve human health worldwide by diagnosing, preventing and treating diseases.

Media Contact:
Marcy Funk
Bayer HealthCare Pharmaceuticals
973-305-5385

Forward-Looking Statements
This news release contains forward-looking statements based on current assumptions and forecasts made by Bayer Group management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in our annual and interim reports to the Frankfurt Stock Exchange and in our reports filed with the U.S. Securities and Exchange Commission (including our Form 20-F). The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

*WCTRIMS is the first joint meeting of ECTRIMS (the European Committee on Treatment and Research in Multiple Sclerosis) and its counterparts in North and Latin America: ACTRIMS and LACTRIMS

*DS Goodin, G Ebers, AT Reder, et al. Early Treatment with Interferon Beta-1b is Associated with Improved Long-Term Outcome in Multiple Sclerosis. World Congress on Treatment and Research in Multiple Sclerosis 2008.

*S Fredrikson, DW Langdon, K Kim, et al. Cognition, Fatigue, Depression and Health-Related Quality of Life in Early Multiple Sclerosis: Baseline Data from CogniMS, a Multinational Longitudinal Study. World Congress on Treatment and Research in Multiple Sclerosis 2008.

Glatiramer Acetate Decreases Severity of Relapsing-Remitting Multiple Sclerosis: Presented at WCTRMS

2008-09-22T09:55:00.002-06:00

By Louise Gagnon

MONTREAL -- September 20, 2008 -- Glatiramer acetate has been shown to be efficacious in several outcomes among patients with relapsing-remitting multiple sclerosis (RRMS) when a scale other than the Expanded Disability Status Scale (EDSS) was used, according to research presented here at the World Congress on Treatment and Research in Multiple Sclerosis (WCTRMS).

A study of 251 patients was presented by investigator Joseph Herbert, MD, New York University, New York, New York, who described the EDSS as not truly reflective of clinical patient outcomes.

In the pivotal study, glatiramer acetate resulted in a 29% decrease in relapse rates compared with placebo. While patients treated glatiramer acetate did show improvement in their EDSS score, Dr. Herbert described the scale as an insufficiently sensitive measure of disease progression.

He presented the study findings in a poster session here on September 19

In this study, Dr. Herbert employed a Multiple Sclerosis Severity Score (MSSS) algorithm that relates MS disability as measured by EDSS to disease duration from the time of first symptom.

"This measure is a more useful outcome measure, because it corrects for certain flaws in the EDSS," said Dr. Herbert in an interview. "It accounts for duration of disease. The EDSS needs to be considered in the context of duration of disease."

This study enrolled patients who had EDSS scores of between 0 and 5. The researchers randomised 125 patients to subcutaneous injections of glatiramer acetate at 20 mg/day and 126 to placebo. Patients were followed for approximately 35 months.

Subjects in this study were divided into 6 groups according to their disease severity, explained Dr. Herbert. At study entry, the median MSSS scores for patients treated with glatiramer acetate and those treated with placebo were similar (4.59 vs 4.29; P = .1). At the end of the study, the median MSSS change from study entry was significantly greater in patients treated with glatiramer acetate compared with those treated with placebo (-0.73 vs -0.19; P = .0019).

With the revised grading of patients according to the MSSS, fewer patients who received glatiramer acetate were categorised as having severe disease. Specifically, more patients treated with glatiramer acetate were reclassified as having less severe illness (49% vs 31%) and fewer patients were reclassified as having more severe illness (16% vs 26%; P < .0014).

"There was a significant drop in severity for the treated patients," said Dr. Herbert. "There was a reassignment toward the higher grades with placebo patients."

The data support the use of the MSSS scale to measure changes in disability in patients with RRMS.

"We are suggesting that, since EDSS and duration of disease must be collected to begin with, that [MSSS] should be used as an outcome measure," said Dr. Herbert. "There is a true change in the intrinsic severity of the disease over 3 years, which is quite dramatic."

Funding for this study was provided by Teva Pharmaceutical Industries Ltd.

[Presentation title: Glatiramer Acetate Reduces Multiple Sclerosis Severity: Analysis of Patients From the US Pivotal Study Using the Multiple Sclerosis Severity Score. Abstract P454]

Vitamin D Levels Might Predict Risk for MS

2008-09-22T09:52:00.001-06:00

FRIDAY, Sept. 19 (HealthDay News) -- Children with lower levels of vitamin D seem to be at a higher risk of being diagnosed with multiple sclerosis.

So say researchers who were expected to present the findings Friday at the World Congress on Treatment and Research in Multiple Sclerosis, in Montreal.

The idea fits nicely with previous research indicating that multiple sclerosis is more common the farther away you get from the equator, in other words, in areas where there is less sunlight.

Vitamin D synthesis is triggered when ultraviolet rays from the sun hit the skin. In addition, studies have also linked vitamin D with immune system function.

"In MS, the immune system is misregulated, and we do know that there's a susceptibility in the genes we inherit from our parents. We know that something triggers the disease," explained Patricia O'Looney, vice president of biomedical research at the National Multiple Sclerosis Society. "We know from epidemiological studies that there's a higher prevalence of MS the farther away you live from the equator and, more recently, we've learned that vitamin D does regulate the immune system."

"This is an interesting study of how environmental triggers and the immune system can be involved with MS, provided that one has these susceptibility genes," she added.

"Many studies have given us a good link between vitamin D status and immune function in MS," added study author Heather E. Hanwell, a doctoral candidate in nutritional sciences at the University of Toronto. "We wanted to see whether vitamin D status was lower in children who had their first demyelinating event and were subsequently diagnosed with MS."

A first demyelinating event is essentially an attack of symptoms that could indicate trouble with the central nervous system. One quarter of children who have such an attack go on to be diagnosed with MS.

The researchers measured levels of a vitamin D biomarker in children who had had a first event.

"The biomarker of vitamin D status was significantly lower in children diagnosed with MS to date," Hanwell said. "Children diagnosed with MS had lower vitamin D levels than those not diagnosed. Another way of looking at it, as vitamin D status increased, children had a lower risk of being diagnosed."

At this point, however, Hanwell believes the findings have more research than clinical implications.

"This type of work provides impetus for further research in this area, although, for a doctor, it would be important to look at vitamin D status in patients, particularly because 75 percent of our overall study group had vitamin D levels below what we considered to be optimal."

A second study also being presented at the World Congress found that the incidence of first demyelinating events increased by 9.2 percent for each higher degree of latitude up the eastern coast of Australia. The study was partially funded by the National Multiple Sclerosis Society, in the United States.

"There is growing evidence linking vitamin D and risk of MS," O'Looney said. "Further studies are certainly needed to see if vitamin supplementation could reduce the risk of MS. There is insufficient evidence that vitamin supplementation can influence the course of MS once it's begun."

More information

Visit the National Multiple Sclerosis Society for more on MS.

SOURCES: Patricia O'Looney, Ph.D., vice president, Biomedical Research, National Multiple Sclerosis Society, New York City; Heather E. Hanwell, doctoral candidate, department of nutritional sciences, University of Toronto

Tysabri PML patient deteriorates - researcher

2008-09-22T09:48:00.003-06:00

By Reuters
September 19, 2008

BOSTON—One of two European patients, who developed a potentially deadly brain infection after taking the multiple sclerosis drug Tysabri, is deteriorating and likely to suffer brain damage, according to a researcher involved in their care.

The other patient diagnosed with the infection in July is recovering, the researcher said Friday.

The drug, made by Biogen Idec Inc and Elan Corp , was temporarily withdrawn from the market in 2005 after being linked with three cases of an often lethal infection known as progressive multifocal leukoencephalopathy, or PML.

Due to its advantages over older MS treatments and patient willingness to take on the risks, Tysabri was reintroduced with stricter warnings in 2006. No new cases were reported until the end of July, when Biogen announced that two patients had developed the infection.

Biogen said on Friday that no further new PML cases have been confirmed and its shares closed up 6 percent. Elan shares closed up more than 1 percent.

Dr. Ralf Gold of Ruhr University Bochum, Germany, said in an interview that a German patient, hospitalized near the town of Freiburg, is close to a coma and being fed through tubes, though he is breathing on his own.

The other patient, a 37-year-old Swede, is in a rehabilitation center near Stockholm and suffering only from mild weakness on one side of his body.

Gold said the 52-year-old German patient was not diagnosed until at least three months after developing the disease, whereas the Swedish patient's condition was identified in less than a month. As a result, the German patient had a higher level of the virus in his brain.

The two were treated through plasmapheresis, a procedure in which a patient's blood is removed, cleared of the drug, and replaced.

Gold presented an update on the two cases at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal.

Investors have been watching the patients' progress to see whether PML can be reversed. They are also watching to see how many cases of the infection develop among patients taking the drug, which is considered a critically important growth driver for both companies.

Tysabri had second-quarter sales of $200 million, but its fortunes have been inextricably linked to PML concerns.

"The drug's strong efficacy profile should keep it as a preferred agent, in our opinion," Leerink Swann analyst William Tanner wrote in a research note.

In a report ahead of Gold's update, Geoff Porges, an analyst at Sanford Bernstein, said that if the disease becomes manageable through plasmapheresis then the impact of these cases will be blunted.

He said experts interviewed at the meeting have confirmed that in Europe and North America there are a number of patients with suspected PML whose physicians discontinued Tysabri use.

"We believe it is incumbent on Biogen to disclose this information to physicians in order for them to fully evaluate and disclose the risks associated with the product," he said.

Biogen spokeswoman Naomi Aoki said, "We have disclosed the only two cases of diagnosed PML that we have seen so far."

Gold added that while certain databases pick up cases in which a doctor suspects PML and discontinues Tysabri, the cases have not been confirmed either through an MRI or any other scientific measures.

Gold said the virus appears to be leaving the German patient's brain, but he is suffering from brain damage brought about by a condition known as immune reconstitution inflammatory syndrome, or IRIS. This occurs when the immune system, in eliminating an infection, produces an excessive inflammatory response that can worsen symptoms.

It is most likely the patient will be permanently brain damaged, Gold said. The only question is by how much.

The patient was started on a course of corticosteroids on Friday, the standard treatment for IRIS.

It typically becomes clear within a few days whether such a treatment is likely to work, Gold said. In the meantime, the patient is in a critical condition but not in intensive care.

© Copyright 2008 Reuters. Reuters content is the intellectual property of Reuters or its third-party content providers. Any copying, republication, or redistribution of Reuters content, including by caching, framing or similar means, is expressly prohibited without the prior written consent of Reuters.

Laquinimod Demonstrated Significant and Sustained Impact on Multiple Sclerosis Disease Activity

2008-09-22T09:46:00.002-06:00

New Crossover Data Presented at the World Congress on Treatment and Research in Multiple Sclerosis Shows Significant Reduction of Gadolinium-Enhancing Lesions

Jerusalem, Israel and Lund, Sweden, September 18, 2008 - New data from the extension phase of oral laquinimod in relapsing-remitting multiple sclerosis (RRMS) demonstrated a significant reduction in the mean number of gadolinium-enhancing (GdE) lesions in both patients who switched from placebo to laquinimod and patients who continued with their initial laquinimod dose.

In RRMS patients who switched from placebo to laquinimod, 52 percent reduction in the mean number of GdE lesions was observed (p<0.0007). The reduction was significant for both patients switching to high-dose (p<0.009) and low-dose laquinimod (p<0.03). In addition, the proportion of patients who switched to active treatment from placebo, and remained enhancing lesion-free, increased from 31 percent to 47 percent (p<0.012), further reinforcing the efficacy of laquinimod on magnetic resonance imaging (MRI) measured disease activity.

Patients initially treated with 0.6mg/day and 0.3mg/day during the double-blind trial remained on the same dose during the 36-week extension phase. An additional significant reduction in the mean number of GdE lesions was also observed in these patients (n=94, p=0.0062 and n=80, p=0.0013, respectively), a high proportion of which remained completely free of GdE lesions, demonstrating the sustained effect of laquinimod on MRI disease activity.

"These latest data show the rapid onset and sustainability of laquinimod efficacy in MS patients," said Giancarlo Comi, M.D., University Vita-Salute San Raffaele, Scientific Institute San Raffaele, Milan, Italy, principal investigator of the study. "Just as exciting is the fact that, with increased number of patients exposed to laquinimod, we found no new risks or safety issues. This reinforces earlier results demonstrating the laquinimod safety profile. The MS community looks forward to future data as we continue enrolling patients in the laquinimod Phase III clinical program."

These new data from the extension study build upon the initial 36-week, Phase IIb study results published in The Lancet*, which demonstrated that once-daily, oral 0.6mg laquinimod significantly reduced MRI disease activity by a median of 60 percent, compared to placebo, and was well tolerated.

Teva is currently enrolling patients for Allegro and Bravo, two pivotal Phase III clinical trials of laquinimod. For more information please visit www.TevaClinicalTrials.com.

*Lancet 2008; 371:2085-92

About the Study
In the study, "Oral laquinimod in patients with relapsing�remitting multiple sclerosis: 36 weeks double-blind active extension of the multi-center, randomized, double-blind, parallel-group placebo-controlled study," subjects originally assigned to placebo were equally randomized to receive either 0.3 or 0.6mg/day laquinimod, while others continued their original treatment for a 36-week, double-blind extension. Magnetic resonance imaging (MRI) was performed at the beginning and at the end of the active extension phase. The mean number of GdE lesions in patients who switched from placebo to laquinimod was reduced by 52 percent (from 4.46�6.55 to 2.12�3.73; p<0.0007) from the time patients began receiving active treatment.

Two hundred and fifty seven patients (91 percent) entered the extension phase to receive laquinimod 0.3mg/day or 0.6mg/day. Neither new safety signals nor an increase in the incidence rate of adverse events and laboratory abnormalities have emerged following new or prolonged exposure to laquinimod.

Patients on continuous 18 month laquinimod 0.6mg treatment continued to show low MRI disease activity, with a high proportion remaining free of GdE-lesions. A good tolerability profile was also observed. Treatment effects on MRI activity witnessed during the placebo-controlled phase were reproduced when placebo patients switched to laquinimod.

About Multiple Sclerosis
Multiple Sclerosis (MS) is the leading cause of neurological disability in young adults. It is estimated that more than 400,000 people in the United States are affected by the disease and that two million people may be affected worldwide. MS is a progressive, demyelinating disease of the central nervous system affecting the brain, spinal cord and optic nerves. Demyelination is the destructive breakdown of the fatty tissue that protects nerve endings.

About Laquinimod
Laquinimod is a novel once-daily, orally administered immunomodulatory compound that is being developed as a disease-modifying treatment for RRMS. Active Biotech developed laquinimod and licensed it to Teva Pharmaceutical Industries, Ltd. in June 2004. A Phase IIb study in 306 patients was recently published in The Lancet and demonstrated that an oral 0.6 mg dose of laquinimod, administered daily, significantly reduced MRI disease activity by a median of 60 percent versus placebo in RRMS patients. In addition, the study showed a favorable trend toward reducing annual relapse rates and the number of relapse-free patients compared with placebo. Treatment was well tolerated, with only some transient and dose-dependent increases in liver enzymes reported. Over 480 MS patients have received laquinimod in various clinical trials.

In addition to the efficacy that laquinimod has shown in Phase II RRMS clinical trials, laquinimod has demonstrated potent therapeutic efficacy in preclinical models of other autoimmune diseases such as rheumatoid arthritis, insulin-dependent diabetes mellitus, Guillain Barr� Syndrome, lupus and Inflammatory Bowel Disease. The broad profile of efficacy in animal models of inflammatory diseases suggests that laquinimod affects a pivotal pathway of inflammation and autoimmunity. Teva expects to initiate the clinical development of laquinimod for Crohn's disease and Lupus Nephritis in the near future.

About the Phase III Program
Allegro (assessment of oral laquinimod in preventing progression of MS) is a pivotal, global, 24/30-month, double-blind, Phase III study designed to evaluate the efficacy, safety and tolerability of laquinimod versus placebo in the treatment of RRMS. The enrollment goal is approximately 1,000 patients with RRMS.

Bravo (benefit-risk assessment of Avonex® and laquinimod) is a pivotal, multinational, multi-center, randomized, double-blind, parallel-group, placebo-controlled study designed to compare the safety and efficacy of laquinimod with placebo and to provide risk-benefit data for laquinimod versus a currently available injectable treatment. The enrollment goal is approximately 1,200 patients with RRMS.

The globally conducted clinical program will include centers throughout the United States as well as centers Canada, Europe, and Israel. To learn more about the research please visit www.TevaClinicalTrials.com or call 1-866-550-0614 (Allegro) or 1-800-840-5601 (Bravo).

About Active Biotech
Active Biotech AB (OMX NORDIC: ACTI), headquartered in Sweden, is a biotechnology company with R&D focus on autoimmune/inflammatory diseases and cancer. Projects in pivotal phase are laquinimod, an orally administered small molecule with unique immunomodulatory properties for the treatment of multiple sclerosis, as well as ANYARA for use in cancer targeted therapy, primarily renal cancer. Further key projects in clinical development comprise the three orally administered compounds TASQ for prostate cancer, 57-57 for SLE and RhuDex® for RA. Please visit www.activebiotech.com for more information.

About Teva
Teva Pharmaceutical Industries Ltd., headquartered in Israel, is among the top 20 pharmaceutical companies in the world and is the world's leading generic pharmaceutical company. The Company develops, manufactures and markets generic and innovative human pharmaceuticals and active pharmaceutical ingredients, as well as animal health pharmaceutical products. Over 80 percent of Teva's sales are in North America and Europe.

Teva's Safe Harbor Statement under the U. S. Private Securities Litigation Reform Act of 1995:
This release contains forward-looking statements, which express the current beliefs and expectations of management. Such statements are based on management's current beliefs and expectations and involve a number of known and unknown risks and uncertainties that could cause our future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to successfully develop and commercialize additional pharmaceutical products, the introduction of competing generic equivalents, the extent to which we may obtain U.S. market exclusivity for certain of our new generic products and regulatory changes that may prevent us from utilizing exclusivity periods, competition from brand-name companies that are under increased pressure to counter generic products, or competitors that seek to delay the introduction of generic products, the impact of consolidation of our distributors and customers, potential liability for sales of generic products prior to a final resolution of outstanding patent litigation, including that relating to the generic versions of Allegra®, Neurontin®, Lotrel® and Protonix®, the effects of competition on our innovative products, especially Copaxone® sales, the impact of pharmaceutical industry regulation and pending legislation that could affect the pharmaceutical industry, the difficulty of predicting U.S. Food and Drug Administration, European Medicines Agency and other regulatory authority approvals, the regulatory environment and changes in the health policies and structures of various countries, our ability to achieve expected results though our innovative R&D efforts, our ability to successfully identify, consummate and integrate acquisitions, including the pending acquisition of Barr Pharmaceuticals Inc., potential exposure to product liability claims to the extent not covered by insurance, dependence on the effectiveness of our patents and other protections for innovative products, significant operations worldwide that may be adversely affected by terrorism, political or economical instability or major hostilities, supply interruptions or delays that could result from the complex manufacturing of our products and our global supply chain, environmental risks, fluctuations in currency, exchange and interest rates, and other factors that are discussed in this report and in our other filings with the U.S. Securities and Exchange Commission ("SEC").

Company Contacts:
Elana Holzman
Kevin Mannix

Teva Pharmaceutical Industries Ltd.
Teva North America

972 (3) 926-7554
(215) 591-8912

Copaxone® Demonstrated Protective Effect in Patients with Clinically Isolated Syndrome Suggestive of Multiple Sclerosis

2008-09-22T09:41:00.002-06:00

PreCISe Data Presented at World Congress on Treatment and Research in Multiple Sclerosis

Jerusalem, Israel, September 19, 2008 - New data from PreCISe, in clinically isolated syndrome patients, have demonstrated that COPAXONE® (glatiramer acetate injection) significantly improved neuro-axonal integrity in patients presenting with a first clinical event suggestive of multiple sclerosis (MS) versus patients who received placebo (p=0.03), as measured by proton magnetic resonance spectroscopy (MRS). This effect was maintained over two years of treatment.

The data represent the first evidence of neuro-axonal protection by a disease modifying therapy in patients presenting with a first clinical event suggestive of MS.

Data were derived from an ancillary study from the Phase III, randomized, placebo-controlled PreCISe trial, which demonstrated that patients treated with COPAXONE® (n=243) had a 45 percent reduction in the risk of developing clinically definite multiple sclerosis (CDMS) compared to those on the placebo (n=238).

"These newly announced data, so far shown in RRMS patients treated with COPAXONE®, provide more evidence that treatment may control the neuronal damage associated with MS disease pathology," said Douglas Arnold, M.D., Professor of Neurology, McGill University and the primary investigator of this ancillary study. "The PreCISe trial demonstrated a significant benefit of COPAXONE® on both clinical and MRI disease activity, along with further reinforcing the excellent safety profile."

The data were presented along with two other presentations from the PreCISe study at the World Congress on Treatment and Research in Multiple Sclerosis, in Montreal, Canada. Additional data derived from the PreCISe study demonstrated that COPAXONE® significantly delayed time to conversion to CDMS and reduced magnetic resonance imaging (MRI) disease activity. The effect was robust among the PreCISe study population (n=481), as a whole and also in subgroups of patients (segmented by gender, age, type of unifocal manifestation as well as steroid treatment for the initial attack, and MRI findings at study baseline). Based on these data, applications for marketing authorization for the extension of its indication to include the treatment of patients with a first clinical event suggestive of MS were submitted in Europe and in the U.S. and are currently under review.

About the PreCISe Data
The study, "Treatment with glatiramer acetate protects axons in patients with clinically isolated syndromes: evidence from the PreCISe trial," determined that patients with clinically isolated syndrome (CIS) who received COPAXONE® showed improvement in their cerebral neuro-axonal integrity relative to patients treated with placebo.

Proton MRS was performed in a subgroup of patients in the PreCISe trial (n=34) to measure the concentration of N-acetulaspartate (NAA) levels. NAA/CR measurements were acquired each year from each patient enrolled at 10 clinical sites in seven countries. Patients terminated at the time of relapse.

Patients who received COPAXONE® showed significant improvement in their cerebral neuro-axonal integrity compared to patients treated with placebo, who showed the decline expected from natural history studies. Paired changes in NAA/CR ratio differed significantly between both patients treated with COPAXONE® (+0.14, n=11) compared to those treated with placebo (-0.33, n=9, p=0.03) at one year, and the change maintained at two years (COPAXONE® n=6, +0.17; placebo n=3, -0.23, p=0.15).

Previously announced PreCISe studies included:

"Treatment with glatiramer acetate reduces MRI-detectable disease activity in patients at presentation with CIS suggestive of MS"

* Demonstrated that early treatment with COPAXONE® is effective in both delaying time to conversion to CDMS and reducing MRI activity in patients presenting with CIS

"Treatment with glatiramer acetate delays conversion to CDMS in patients with CIS: subgroup analyses"

* Demonstrated that COPAXONE® treatment effect was robust among the PreCISe study population (n=481) as a whole ,as well as in subgroups of patients (broken down by gender, age and type of unifocal manifestation and steroid treatment for the initial attack, and MRI findings at study baseline)

About COPAXONE®
COPAXONE® is indicated for the reduction of the frequency of relapses in RRMS. The most common side effects of COPAXONE® are redness, pain, swelling, itching, a lump or an indentation at the site of injection, weakness, infection, pain, nausea, joint pain, anxiety, and muscle stiffness.

COPAXONE® is now approved in 51 countries worldwide, including the United States, Canada, Mexico, Australia, Israel, and all European countries. In North America, COPAXONE® is marketed by Teva Neuroscience, Inc., which is a subsidiary of Teva Pharmaceutical Industries Ltd. (NASDAQ:TEVA). In Europe, COPAXONE® is marketed by Teva Pharmaceutical Industries Ltd. and sanofi-aventis. COPAXONE® is a registered trademark of Teva Pharmaceutical Industries Ltd.

See additional important information at http://www.copaxone.com/pi/index.html or call 1-800-887-8100 for electronic releases. For hardcopy releases, please see enclosed full prescribing information.

About Teva
Teva Pharmaceutical Industries Ltd., headquartered in Israel, is among the top 20 pharmaceutical companies in the world and is the world's leading generic pharmaceutical company. The Company develops, manufactures and markets generic and innovative human pharmaceuticals and active pharmaceutical ingredients, as well as animal health pharmaceutical products. Over 80 percent of Teva's sales are in North America and Europe.

Teva's Safe Harbor Statement under the U. S. Private Securities Litigation Reform Act of 1995:

This release contains forward-looking statements, which express the current beliefs and expectations of management. Such statements are based on management's current beliefs and expectations and involve a number of known and unknown risks and uncertainties that could cause our future results, performance or achievements to differ significantly from the results, performance or achievements expressed or implied by such forward-looking statements. Important factors that could cause or contribute to such differences include risks relating to: our ability to successfully develop and commercialize additional pharmaceutical products, the introduction of competing generic equivalents, the extent to which we may obtain U.S. market exclusivity for certain of our new generic products and regulatory changes that may prevent us from utilizing exclusivity periods, competition from brand-name companies that are under increased pressure to counter generic products, or competitors that seek to delay the introduction of generic products, the impact of consolidation of our distributors and customers, potential liability for sales of generic products prior to a final resolution of outstanding patent litigation, including that relating to the generic versions of Allegra® , Neurontin®, Lotrel® and Protonix®, the effects of competition on our innovative products, especially Copaxone® sales, the impact of pharmaceutical industry regulation and pending legislation that could affect the pharmaceutical industry, the difficulty of predicting U.S. Food and Drug Administration, European Medicines Agency and other regulatory authority approvals, the regulatory environment and changes in the health policies and structures of various countries, our ability to achieve expected results though our innovative R&D efforts, our ability to successfully identify, consummate and integrate acquisitions, including the pending acquisition of Barr Pharmaceuticals Inc., potential exposure to product liability claims to the extent not covered by insurance, dependence on the effectiveness of our patents and other protections for innovative products, significant operations worldwide that may be adversely affected by terrorism, political or economical instability or major hostilities, supply interruptions or delays that could result from the complex manufacturing of our products and our global supply chain, environmental risks, fluctuations in currency, exchange and interest rates, and other factors that are discussed in this report and in our other filings with the U.S. Securities and Exchange Commission ("SEC").

Company Contacts:
Company Contacts:
Elana Holzman
Kevin Mannix

Teva Pharmaceutical Industries Ltd.
Teva North America

972 (3) 926-7554
(215) 591-8912

Biogen Idec MS pill found to slow damage

2008-09-22T09:35:00.003-06:00

Bloomberg News / September 19, 2008

NEW YORK - Biogen Idec Inc.'s experimental pill to treat multiple sclerosis prevented brain lesions associated with the disease from getting worse, a study found.

The pill, called BG-12, reduced the conversion of new spots of inflammation into permanent damage in a trial of 56 patients, Biogen said yesterday at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal.

In MS, neurons are stripped of an insulating coating known as myelin by the immune system, causing the cells to malfunction. That leads to MS symptoms such as muscle weakness and loss of coordination, according to the Mayo Clinic. Biogen has received approval from US regulators to speed the review process for its pill, the company said yesterday. If cleared for sale in the United States, BG-12 could be the first oral medication for MS patients to reach the market.

"There are two elements: You want to keep the lesions from forming in the first place and then, even if a lesion developed, you want to know the damage is reduced, and that's what you're seeing," said Mike Panzara, the chief medical officer for Cambridge, Mass.-based Biogen in a telephone interview yesterday. "Even if a lesion does develop on BG-12, injury is less because it's less often the lesions become permanent."

About 29 percent of the lesions in the brains of patients on BG-12 turned into signs of permanent damage, compared with 44 percent of those in the placebo group, the study showed.

The company began final-stage testing on BG-12 in January. The trials, on more than 2,000 patients with a recurring form of the disease, will last two years. The drug is being compared with a placebo and with Teva Pharmaceutical Industries' Copaxone, an approved treatment for the disease.

Biogen Idec sells the MS drugs Avonex, which is given as a once-a-week injection, and Tysabri, an infusion given once a month in a doctor's office or hospital clinic. About 1 million people worldwide suffer from MS.

© Copyright 2008 Globe Newspaper Company.

Merck Serono Announces Initiation of the ORACLE MS Trial to Evaluate Cladribine Tablets in Patients at Risk of Developing Multiple Sclerosis

2008-09-22T09:27:00.003-06:00

• ORACLE MS Phase III trial will assess effectiveness of cladribine tablets in preventing conversion to definite multiple sclerosis in addition to the fully enrolled Phase III pivotal trial – the CLARITY study – for treatment of relapsing forms of multiple sclerosis

Geneva, Switzerland, September 18, 2008 – Merck Serono, a division of Merck KGaA, Darmstadt, Germany, announced today the initiation of a Phase III trial to evaluate the therapeutic effects of its proprietary oral formulation of cladribine (cladribine tablets) in patients at risk of developing multiple sclerosis (MS).

The trial, called ORACLE MS (ORAl CLadribine in Early MS) will evaluate the safety and efficacy of two dosage regimens of cladribine tablets versus placebo in the treatment of patients who have experienced a first clinical event suggestive of MS. Cladribine tablets are currently also being evaluated in a fully enrolled Phase III pivotal trial – the CLARITY1 study – for treatment of relapsing forms of MS. As announced in January 2007, CLARITY was the first pivotal trial among all Phase III oral compounds in development for MS to complete enrollment. Cladribine tablets have been granted a fast track designation by the US Food and Drug Administration.

“There is increasing evidence supporting the initiation of treatment with a disease-modifying drug in patients who have experienced a first clinical event suggestive of multiple sclerosis, an initial stage of the disease when clinical manifestations are not necessarily pronounced but where the potential exists for irreversible neurological damage to take place,” said Dr. Thomas Leist, Associate Professor of Neurology and Director of the Comprehensive MS Center at Thomas Jefferson University, Philadelphia, PA, and an investigator in the ORACLE MS study. “The ORACLE MS study will evaluate the effectiveness and safety of cladribine tablets in preventing conversion to definite multiple sclerosis.”

“As a leader in multiple sclerosis treatments, we are committed to providing new options that can further improve the course of the disease and the quality of life for people living with this disease,” said Dr. Richard Douge, Executive Vice President, Global Marketing, Merck Serono. “The initiation of the ORACLE MS study further demonstrates our commitment to continue to build a solid portfolio of products for use in a broad multiple sclerosis patient population. We believe that our proprietary oral formulation of cladribine has the potential to address an important unmet medical need at a critical time of disease development.”

The ORACLE MS study is a randomized, double-blind, placebo-controlled, international trial. It will involve more than 600 patients considered at risk of developing MS due to a recently experienced isolated demyelinating event (e.g. optic neuritis, myelopathy or brainstem syndrome) and having MRI brain scans consistent with early signs of MS. Study participants will be randomized in one of the three arms of the study to receive one of two different dosage regimens of cladribine tablets or matching placebo tablets (1:1:1 ratio).

Patients will be treated for a period of two years (96 weeks), or up to the time when they experience a second attack leading to a diagnosis of clinically definite MS, in which case they would be offered open-label treatment with Rebif® 44 mcg three times a week for a 96-week maintenance treatment period. Patients who do not convert to clinically definite MS within the initial 96-week period of the study will be eligible to enroll in a 96-week long-term follow-up treatment period. These maintenance and long-term follow-up periods of the study are intended to assess the effect of early treatment with cladribine tablets on relapses and subsequent treatment response to disease-modifying therapy for relapsing-remitting MS and to evaluate the sustained effect of cladribine tablets in delaying the development of definite MS.

In the study, cladribine tablets are given in two or four treatment cycles in the first year, with each cycle consisting of once daily administration for four to five consecutive days, which means study patients take cladribine tablets for only 8 to 20 days during that year. In the second year, two treatment cycles are administered to all patient groups.

The primary endpoint of the ORACLE MS trial is time to conversion to MS, according to the McDonald criteria. Other endpoints include time to conversion to clinically definite MS according to the Poser criteria (the main secondary endpoint), assessments of MRI brain scans, and disability progression.

Approximately 200 medical centers globally are expected to participate in this trial.
1 CLARITY: CLAdRIbine Tablets Treating MS OrallY

About cladribine tablets

Merck Serono’s proprietary oral formulation of cladribine is currently being evaluated in Phase III as a treatment for patients with relapsing forms of multiple sclerosis (MS). Cladribine is a small molecule that may interfere with the behavior and the proliferation of certain white blood cells, particularly lymphocytes, which are thought to be involved in the pathological process of MS.

About Rebif®

Rebif® (interferon beta-1a) is a disease-modifying drug used to treat relapsing forms of multiple sclerosis (MS) and is similar to the interferon beta protein produced by the human body. The efficacy of Rebif® in chronic progressive MS has not been established. Interferons are thought to help modulate the body’s immune system and reduce inflammation. The exact mechanism is unknown.

Rebif®, which was approved in Europe in 1998 and in the US in 2002, is registered in more than 80 countries worldwide. Rebif® has been proven to delay the progression of disability, reduce the frequency of relapses and reduce MRI lesion activity and area*. Rebif® is available in a 22 microgram and 44 microgram ready-to-use pre-filled syringe and a titration pack (8.8 micrograms).

Rebif® should be used with caution in patients with a history of depression, liver disease and seizures. Most commonly reported side effects are flu-like symptoms, injection site disorders, elevation of liver enzymes and blood cell abnormalities. Patients, especially those with depression, seizure disorders, or liver problems, should discuss treatment with Rebif® with their doctors. For more information about Rebif®, please visit www.mslifelines.com for prescribing information.

* The exact correlation between MRI findings and the current or future clinical status of patients, including disability progression, is unknown.

About Merck Serono and multiple sclerosis

Merck Serono is a leader in multiple sclerosis (MS) with Rebif® (interferon beta-1a), a disease-modifying drug used to treat relapsing forms of MS, which is registered in more than 80 countries worldwide. Full prescribing information for this product can be obtained by contacting the Company or visiting its website. Additional therapeutic options are currently under development at Merck Serono, including cladribine tablets, currently in Phase III and potentially the first oral therapy for MS, as well as several products in early stage development. Merck Serono also is taking a leading role in developing an understanding of the role of genetics in MS.
About multiple sclerosis

Multiple sclerosis (MS) is a chronic, inflammatory condition of the nervous system and is the most common, non-traumatic, disabling neurological disease in young adults. The World Health Organization estimates that up to 2.5 million people suffer from MS worldwide. While symptoms can vary, the most common symptoms of MS include blurred vision, numbness or tingling in the limbs and problems with strength and coordination. The relapsing forms of MS are the most common.

About Merck Serono

Merck Serono is the division for innovative prescription pharmaceuticals of Merck, a global pharmaceutical and chemical group. Headquartered in Geneva, Switzerland, Merck Serono discovers, develops, manufactures and markets innovative small molecules and biopharmaceuticals to help patients with unmet medical needs. Its North American business operates in the United States and Canada as EMD Serono.

Merck Serono has leading brands serving patients with cancer (Erbitux®), multiple sclerosis (Rebif®), infertility (Gonal-f®), endocrine and cardiometabolic disorders (Glucophage®, Concor®, Euthyrox®, Saizen®, Serostim®), as well as psoriasis (Raptiva®).
With an annual R&D expenditure of around € 1bn, Merck Serono is committed to growing its business in specialist-focused therapeutic areas including neurodegenerative diseases, oncology, fertility and endocrinology, as well as new areas potentially arising out of research and development in autoimmune and inflammatory diseases.

About Merck

Merck is a global pharmaceutical and chemical company with total revenues of € 7.1 billion in 2007, a history that began in 1668, and a future shaped by 31,946 employees in 60 countries. Its success is characterized by innovations from entrepreneurial employees. Merck's operating activities come under the umbrella of Merck KGaA, in which the Merck family holds an approximately 70% interest and free shareholders own the remaining approximately 30%. In 1917 the U.S. subsidiary Merck & Co. was expropriated and has been an independent company ever since.

For more information, please visit www.merckserono.net or www.merck.de

Avigen Completes Multi-Center AV650 Phase 2b Spasticity Trial in Patients With Multiple Sclerosis

2008-09-22T09:23:00.002-06:00

Top Line Data Expected in Q4 2008

ALAMEDA, Calif., Sep 18, 2008 (GlobeNewswire via COMTEX News Network) -- Avigen, Inc. (Nasdaq:AVGN), a biopharmaceutical company innovating therapeutics for neurological care, today announced the completion of the double-blind portion of its Phase 2b trial for AV650 (tolperisone HCl) in the treatment of spasticity associated with multiple sclerosis (MS). This Phase 2b spasticity trial is evaluating the safety, tolerability and efficacy of AV650 in MS patients at doses up to 900mg/day for one month followed by an open-label safety extension. The trial is being conducted in top MS centers in Germany and several other European countries.

"The completion of this trial in less than a year marks a significant milestone for the AV650 program and Avigen," said Kenneth Chahine, Ph.D., J.D., Avigen's President and Chief Executive Officer. "The rapid enrollment further supports the need for new and non-sedating agents to treat spasticity, and we have confidence that our planned Phase 3 trials can be completed in a similar timeframe. While the blinded portion of the trial is complete and will report before the end of 2008, more than 90% of the patients have elected to participate in the on-going open-label extension and will continue to receive AV650 for up to an additional twelve months."

Avigen is developing AV650 for commercialization in the North American market for the treatment of disabling neuromuscular spasticity and spasm under a license and supply agreement with Sanochemia Pharmazeutika AG, Vienna, Austria. AV650 is considered a New Chemical Entity (NCE) in the United States.

Tolperisone is an orally administered, centrally acting small molecule marketed for the treatment of neuromuscular spasticity and spasm in Europe and Asia. Avigen's U.S. development program is designed to build on the extensive ex-U.S. safety and efficacy experience with this compound. Versions of tolperisone have been approved for marketing in Germany for over 10 years. Sanochemia and its European marketing partner, Orion Pharma, currently market a proprietary 150mg tablet formulation of tolperisone in Germany under the brand name Viveo(r).

Spasticity is considered one of the main symptoms of the more than 2.5 million people with MS worldwide, affecting up to 60% of this population. Randall Schapiro, M.D, Director of The Schapiro Center for Multiple Sclerosis at the Minneapolis Clinic of Neurology and Clinical Professor of Neurology at the University of Minnesota, commented, "MS patients must cope with significant side effects from currently available treatments for spasticity, including drowsiness, fatigue and cognitive impairment. These side effects do affect their quality of life. We recognize the need for better-tolerated, non-sedating therapies in the United States, and we are encouraged by the European experience of tolperisone."

About the Trial

This double-blind, randomized clinical trial is being conducted in 27 MS centers in Germany and other European countries. It will evaluate safety, tolerability, pharmacokinetics and efficacy in up to 150 MS patients suffering from spasticity. This trial is being conducted to Good Clinical Practice standards by a leading international contract research organization. Following a four-week double blind assessment, patients are offered the opportunity to continue for up to twelve months in an open-label safety phase. The primary efficacy endpoint is the Ashworth scale, a standard tool used in the clinic to measure increased movement. Secondary endpoints include Brief Pain Index (BPI), painful spasm diaries, and clinical impression of change.

About Neuromuscular Spasm and Spasticity

Chronic or recurrent muscle spasm is a sudden, violent, painful contraction of muscles typically associated with serious neurological disorders such as Lou Gehrig's disease (ALS), multiple sclerosis, stroke, spinal cord injury, and cerebral palsy. These painful muscle spasms are often, but not always, associated with spasticity, an abnormality in muscle "tone." Spastic limbs become stiff and rigid because the muscles fail to relax, lacking normal regulation by the damaged nervous system. Both spasticity and sudden, painful muscle spasms can occur as complications of the neurological disorders mentioned above. Spasticity is a common symptom in multiple sclerosis, which affects 2.5 million worldwide.

About Avigen

Avigen is a biopharmaceutical company focused on developing and commercializing small molecule therapeutics to treat serious neurological disorders, including neuropathic pain and neuromuscular spasm and spasticity. Avigen's strategy is to complete the requirements of clinical development for each of the candidates in its product pipeline, and continue to look for opportunities to expand its pipeline through a combination of internal research, acquisitions, and in-licensing, with the goal of becoming a fully integrated commercial biopharmaceutical company that remains committed to its neurology products. Avigen is currently developing AV650 for spasticity and neuromuscular spasm and AV411 for neuropathic pain, as well as opioid withdrawal and addiction in collaboration with the National Institute of Drug Abuse. Additionally, the company is advancing AV513, a novel therapy for the treatment of multiple bleeding disorders, including hemophilia A and B, toward clinical trials. For more information about Avigen, consult the company's website at www.avigen.com.
Statement under the Private Securities Litigation Reform Act

The statements in this press release relating to Avigen's expectations regarding the timely and reliable completion of its clinical trials, its expectations regarding reporting AV650 Phase 2 top-line data in the fourth quarter of 2008, its expectations regarding initiating and completing planned Phase 3 clinical trials in a similar timeframe to the Phase 2b spasticity trial in patients with MS, and its goal of becoming a fully integrated commercial biopharmaceutical company remaining committed to its neurology products are forward-looking statements. These statements are subject to risks and uncertainties that could cause actual results to differ materially from those projected in these forward-looking statements. These risks and uncertainties include, among others, the fact that development of small molecule therapeutics and other therapeutic discovery and development is a time- and resource-intensive process, which may result in the expenditure of a significant amount of time and resources with no progress towards clinical trials or marketable product resulting from the effort; the risk that Avigen will not be able to obtain regulatory approvals for its drug products, which is required prior to marketing drug products; and the risk that early positive preclinical and clinical results will not guarantee that the potential products will ultimately be effective in treating the indications for which they are developed, or exhibit the unique properties they appear to possess. In addition, there are many other risks and uncertainties inherent in the development of drug products. Other risks and uncertainties relating to Avigen are detailed in reports filed by Avigen with the Securities and Exchange Commission, including Avigen's Quarterly Report on Form 10-Q for the period ended June 30, 2008, under the caption "Risks Related to Our Business" in Item 2 of Part I of that report, which was filed with the SEC on August 11, 2008.

This news release was distributed by GlobeNewswire, www.globenewswire.com

SOURCE: Avigen, Inc.

Avigen, Inc.
Michael Coffee, Chief Business Officer
510-748-7372
Fax: 510-748-7155
1301 Harbor Bay Parkway
Alameda, CA 94502

MS rates higher farther from equator, atlas shows

2008-09-22T09:17:00.002-06:00

Multiple sclerosis is more common among people living in northern latitudes, but they aren't the only ones affected, according to a new report.

The World Health Organization and the London-based Multiple Sclerosis International Federation published the MS Atlas on Wednesday. It summarizes information on the disease in 112 countries, none of which were free of the disease.

MS is a neurodegenerative disease that attacks the brain and spinal cord, and can lead to paralysis and sometimes blindness.

Some people with MS experience little disability during their lifetime. But up to 60 per cent are no longer fully able to walk 20 years after onset, which has major implications for their quality of life and costs to society, the report said. Symptoms appear around 30 years of age on average.

“The Atlas of MS reveals how these implications impact women more than men, by at least two to one, at an age when they are starting a family and developing a career,” said Dr. Benedetto Saraceno, director of the WHO's department of mental health and substance dependence.

The study confirms that MS is a global disease, not solely of the more developed “northern” and “western” countries, the report said.

In Canada alone, the economic impact totals more than $1 billion annually, according to the Multiple Sclerosis Society of Canada.

Countries reporting the highest estimated prevalence, or total number of cases of MS at a particular point in time, were:

* Hungary at 176 per 100,000
* Slovenia 150 per 100,000.
* Germany 149 per 100,000.
* United States 135 per 100,000.
* Canada 132.5 per 100,000.
* Czech Republic 130 per 100,000.
* Norway 125 per 100,000.
* Denmark 122 per 100,000.
* Poland 120 per 100,000.
* Cyprus 110 per 100,000.

Countries reporting the highest estimated incidence or estimated number of new cases of MS were:

* Croatia 29 per 100,000.
* Iceland 10 per 100,000.
* Hungary 9.8 per 100,000.
* Slovakia 7.5 per 100,000.
* Costa Rica 7.5 per 100,000.
* United Kingdom 6.0 per 100,000.
* Lithuania 6.0 per 100,000.
* Denmark 5.9 per 100,000.
* Norway 5.5 per 100,000.
* Switzerland 5.0 per 100,000.

"Typically, our results confirmed the well established suggestion that there are strong geographical patterns to the disease and that the frequency of MS varies by geographical region throughout the world, increasing with distance from the equator in both hemispheres," the report said.

Low- and middle-income countries showed a lack of services and resources to care for people with MS. Poorer countries also had fewer diagnostic tools such as MS scanners, which means the disease is likely underrecorded in developing countries.

The atlas also compares how North and South America, Europe, Asia, Eastern Mediterranean and African countries offer resources to diagnose, treat, rehabilitate and support people with the MS.

NEW LONG-TERM DATA SHOW THAT PATIENTS TAKING AVONEX® FOR UP TO 15 YEARS EXPERIENCE REDUCED DISABILITY PROGRESSION AND IMPROVED QUALITY OF LIFE

2008-09-22T09:08:00.003-06:00

NEW LONG-TERM DATA SHOW THAT PATIENTS TAKING AVONEX® FOR UP TO 15 YEARS EXPERIENCE REDUCED DISABILITY PROGRESSION AND IMPROVED QUALITY OF LIFE

MONTREAL, CANADA - September 18, 2008 - Biogen Idec (NASDAQ: BIIB) today announced that data was presented from the ASSURANCE (ASSessment of Drug Utilization, EaRly TreAtmeNt, and Clinical OutcomEs) study, showing the long-term benefits of AVONEX® (interferon beta-1a IM) therapy in patients with relapsing multiple sclerosis (MS) for up to 15 years. The ASSURANCE study represents the long-term follow-up of patients who participated in the Multiple Sclerosis Collaborative Research Group (MSCRG), the original Phase III pivotal trial from which AVONEX was approved. Data from this study were presented today as a poster presentation at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada. This is the first joint meeting of the Americas Committee on Treatment and Research in Multiple Sclerosis (ACTRIMS) and its counterparts in Europe and Latin America: ECTRIMS and LACTRIMS.

MONTREAL, CANADA - September 18, 2008 - Biogen Idec (NASDAQ: BIIB) today announced that data was presented from the ASSURANCE (ASSessment of Drug Utilization, EaRly TreAtmeNt, and Clinical OutcomEs) study, showing the long-term benefits of AVONEX® (interferon beta-1a IM) therapy in patients with relapsing multiple sclerosis (MS) for up to 15 years. The ASSURANCE study represents the long-term follow-up of patients who participated in the Multiple Sclerosis Collaborative Research Group (MSCRG), the original Phase III pivotal trial from which AVONEX was approved. Data from this study were presented today as a poster presentation at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada. This is the first joint meeting of the Americas Committee on Treatment and Research in Multiple Sclerosis (ACTRIMS) and its counterparts in Europe and Latin America: ECTRIMS and LACTRIMS.

"As a physician, my goal in treating my MS patients is to delay disability progression and help them maintain their normal lifestyle for as long as possible," said Robert Bermel, MD, Mellen Center for Multiple Sclerosis Treatment and Research, Cleveland Clinic. "This follow-up study identifies a group of patients who achieved benefits from long-term treatment, and underscores the importance of starting on and continuing an effective therapy for MS."

The data from the study show that patients currently taking AVONEX for up to 15 years (range of 3 - 15 years) versus those not on AVONEX therapy reported:

* Significantly lower disability progression as measured by a mean change in Expanded Disability Scale Scores (EDSS) of 2.3 vs. 3.3 (p=0.011) from MSCRG baseline;
* Lower disability progression to EDSS milestones four (64% vs. 83%, p=0.06), six (32% vs. 62%, p=0.008) and seven (9% vs. 33%, p=0.008);
* Greater quality of life as measured by the physical component score of the SF-36 (p<0.0001);
* Significantly greater sense of independence in self care (p=0.0019); and
* Significantly more independent living (p=0.031).

ASSURANCE was an open-label, retrospective, patient-reported, multicenter, 15-year follow-up study that included patients with relapsing MS who received ≤ 2 years of treatment in the pivotal Phase III trial (n=172). One hundred thirty-six of a possible 172 patients enrolled in the study. Patients were categorized as current AVONEX users and non-AVONEX users. Forty-six percent of those patients were currently taking AVONEX, with median treatment duration of 13.3 years. Patients not currently on AVONEX were treated with a number of other disease-modifying therapies, with 24 percent of patients undergoing treatment with TYSABRI® (natalizumab).

"This level of impact on disability and quality of life over the course of 15 years reinforces the real-life benefits and proven clinical effectiveness of AVONEX," said Thorsten Eickenhorst, MD, Vice President of Global Medical Affairs, Biogen Idec. "We pride ourselves on providing a treatment that is not only efficacious now but will continue to offer patients the support they need for as long as possible."
About AVONEX

AVONEX is the most prescribed treatment for relapsing forms of MS worldwide, with more than 130,000 patients on therapy. It was launched in the U.S. in 1996 and in Europe in 1997 for the treatment of relapsing forms of MS to slow the progression of disability and reduce relapses. AVONEX has been proven effective in clinical trials for up to three years. AVONEX is marketed internationally in more than 90 countries. AVONEX was the first treatment approved for patients who have their first clinical MS attack and have a brain MRI scan consistent with MS; this use was approved in Europe in 2002 and in the U.S. in 2003.

The most common side effects associated with AVONEX multiple sclerosis treatment are flu-like symptoms, including myalgia, fever, fatigue, headache, chills, nausea, vomiting, pain, and asthenia.

AVONEX should be used with caution in patients with depression or other mood disorders and in patients with seizure disorders. AVONEX should not be used by pregnant women. Patients with cardiac disease should be closely monitored. Patients should also be monitored for signs of hepatic injury. Routine periodic blood chemistry and hematology tests are recommended during treatment with AVONEX. Rare cases of anaphylaxis have been reported. For more information, visit www.AVONEX.com
About Biogen Idec

Biogen Idec creates new standards of care in therapeutic areas with high unmet medical needs. Founded in 1978, Biogen Idec is a global leader in the discovery, development, manufacturing, and commercialization of innovative therapies. Patients in more than 90 countries benefit from Biogen Idec's significant products that address diseases such as lymphoma, multiple sclerosis, and rheumatoid arthritis. For product labeling, press releases and additional information about the company, please visit www.biogenidec.com.

For more information contact:

Media Contacts:

Shannon Altimari
617-914-6524

Investor Contacts:

Eric Hoffman
617-679-2812

Acorda Therapeutics Presents Additional Data from Second Positive Phase 3 Study of Fampridine-SR at World Congress on Treatment and Research in Multip

2008-09-22T09:05:00.002-06:00

- Similar Response Rates Seen Across Different MS Subtypes and Background Therapies -

HAWTHORNE, N.Y.--(BUSINESS WIRE)--Sept. 20, 2008--Acorda Therapeutics, Inc. (NASDAQ: ACOR) today announced additional data from its second Phase 3 clinical trial of Fampridine-SR (MS-F204) on walking ability in people with multiple sclerosis (MS) at the late breaking news session of the World Congress on Treatment and Research in Multiple Sclerosis, being held in Montreal, Canada. Previously, the Company announced the trial met its primary endpoint with a significantly greater proportion of people taking Fampridine-SR having a consistent improvement in walking speed compared to people taking placebo (42.9% vs. 9.3%), as measured by the Timed 25-Foot Walk (p less than 0.001). The study also met its secondary outcome measure, leg strength, showing a statistically significant increase in the Fampridine-SR Timed Walk responders compared to placebo (p = 0.028).

New findings and information presented at the meeting from the study included:

-- The response rate for Fampridine-SR treated patients was
higher than placebo across all MS subtypes. Response rates for
the four major MS subtypes in the study were:
relapsing-remitting: 37.2%; secondary-progressive: 45.9%;
primary-progressive: 50.0%; and progressive-remitting: 40.0%.

-- Response rates were similar between study participants who
were being treated with immunomodulators and those who were
not.

-- The Fampridine-SR treated group showed improvement in the
Ashworth Score (a physician-reported measure of spasticity),
which was significant in an unplanned analysis.

-- Baseline demographic and disease characteristics of study
participants were also presented, including the percentage of
Fampridine-SR treated patients with each subtype of MS
(relapsing-remitting: 35.8%; primary-progressive: 8.3%;
secondary-progressive: 51.7%; and progressive-relapsing: 4.2%)
and the mean duration of disease in Fampridine-SR treatment
patients (14.43 years).

"People with MS often cite walking disability as one of the most challenging aspects of their disease, and there are no therapies currently indicated for improving walking in MS," said Andrew Goodman, M.D., Director of the Multiple Sclerosis Center at the University of Rochester, who presented the data. "The results of this study, which were consistent with the first Phase 3 Fampridine-SR trial, show that Fampridine-SR may provide benefit to people with walking difficulties."

Study Design

The double-blind, placebo-controlled trial was designed to evaluate the safety and efficacy of Fampridine-SR in improving walking ability in people with MS. The primary endpoint of the study was response on the Timed 25-Foot Walk. A Fampridine-SR Timed Walk responder was defined as a study participant whose walking speed was faster during at least three of the four on-drug visits than any speed measured during the five off-drug visits. This Timed Walk Response was validated for clinical meaningfulness in previous trials by showing that the response was associated with significant improvements in day-to-day disability, as measured by the 12-item MS Walking Scale (MSWS-12), and was also associated with significant improvements in both Subject and Clinician Global Impression scores. The trial, which enrolled 240 individuals at 39 MS centers in the United States and Canada, recruited patients between 18 and 70 years old with a definite diagnosis of MS and some degree of walking disability. Subjects were randomized to treatment with Fampridine-SR (n=120), at a dose of 10mg twice a day, or placebo (n=119), and the study was open to people with all four major types of MS: primary-progressive, secondary-progressive, relapsing-remitting and progressive-relapsing. Participants were permitted to remain on a stable regimen of their current medications, including immunomodulators.

Safety Statement

In this study, adverse events were generally mild to moderate and largely consistent with the safety profile observed in previous studies of Fampridine-SR in people with MS. The most common adverse events reported in the Fampridine-SR treatment group compared to the placebo group included: urinary tract infection (17.5% vs. 8.4%), falls (11.7% vs. 16.8%), insomnia (10.0% vs. 1.7%), headache (9.2% vs. 0.8%), asthenia (8.3% vs. 4.2%), dizziness (8.3% vs. 0.8%), nausea (8.3% vs. 0.8%), back pain (5.8% vs. 2.5%), balance disorder (5.8% vs. 1.7%), upper respiratory tract infection (5.8% vs. 6.7%), arthralgia (5.0% vs. 4.2%), nasopharyngitis (5.0% vs. 4.2%) and paraesthesia (5.0% vs. 1.7%).

There were three serious adverse events (SAEs) that led to discontinuation: two in the placebo group and one in the Fampridine-SR group. In the placebo group, one participant experienced a possible complex partial seizure and another experienced a combination of chest tightness and gastric reflux. Both of these events were judged by investigators, who were blinded at the time, to be possibly related to treatment. In the Fampridine-SR group, one participant had a patellar fracture, which was judged not to be treatment related. In addition, one participant treated with Fampridine-SR experienced an episode of syncope (fainting) one day after completing the treatment phase of the study. This was judged to be possibly related to treatment, but the participant was not discontinued from the trial. Follow-up assessment by the clinical investigators determined that these SAEs resolved completely with no residual effects. No deaths occurred during the study.

As of August 5, 2008, the total exposure in our MS studies to Fampridine-SR at 10mg twice a day, including both double-blind and open-label studies, is approximately 1,200 patient years. The incidence of seizures in these studies at the 10mg dose has been within the rates reported for placebo-treated groups in long-term controlled studies of immunomodulator drugs in MS patients. These rates have ranged up to two percent of patients in a two-year study, or one seizure per 100 patient years. The overall incidence of seizure appears to be dose-related.

About MS

Multiple sclerosis is a chronic, usually progressive disease in which the immune system attacks and degrades the function of nerve fibers in the brain and spinal cord. Over 400,000 Americans have MS, and someone is newly diagnosed with MS every hour in the United States. Most are between the ages of 20 and 50, and women are affected two to three times as much as men. Worldwide, MS may affect 2.5 million individuals.

According to the National Multiple Sclerosis Society (NMSS), the direct costs of medical care for MS patients in the United States exceed $6 billion annually. Additionally, a NMSS analysis estimated the total cost of MS, including medical and non-medical care, production losses, and informal care, at more than $47,000 per U.S. patient per year. Complications from MS may make it harder for people to work and may interfere with their ability to perform common daily activities.

For most people with MS, the disease slowly progresses with a series of unpredictable flare-ups, also called relapses. But for some, the progression of the disease is rapid. Each relapse tends to lead to increasing disabilities such as walking impairment, muscle weakness or speech or vision impairments. Research indicates 64% -85% of people with MS have difficulty walking, and 70% of people with MS who have difficulty walking report it to be the most challenging aspect of their MS. Within 15 years of an MS diagnosis, 50 percent of patients often require assistance walking and, in later stages, up to a third of patients are unable to walk.

About Fampridine-SR

Fampridine-SR is a sustained-release tablet formulation of the investigational drug fampridine (4-aminopyridine or 4-AP). In laboratory studies, fampridine has been found to improve impulse conduction in nerve fibers in which the insulating layer, called myelin, has been damaged. Fampridine-SR completed a second Phase 3 clinical trial to evaluate its safety and efficacy in improving walking ability in people with MS in June 2008.

Fampridine-SR and MS

A nerve cell has one extension, called an axon, which it uses to communicate via electrical signals to other nerve cells. All but the smallest axons have a special covering of a fatty substance called myelin that acts as insulation to preserve and speed these nerve signals, much like the insulating cover of an electrical cord helps preserve the transmission of electricity.

In MS, the myelin becomes damaged and the axon cannot effectively transmit electrical impulses. Specifically, the damaged myelin exposes channels in the membrane of the axon, which allow potassium ions to leak from the axon, dissipating the electrical current. In published studies, fampridine has been shown to block these exposed channels and help the electrical signals to pass through areas of damage.

About Acorda Therapeutics

Acorda Therapeutics is a biotechnology company developing therapies for spinal cord injury, multiple sclerosis and related nervous system disorders. The Company's marketed products include Zanaflex Capsules(R) (tizanidine hydrochloride), a short-acting drug for the management of spasticity. Acorda's lead clinical product, Fampridine-SR, recently completed a second Phase 3 clinical trial to evaluate its safety and efficacy in improving walking ability in people with MS. The Company's pipeline includes a number of products in development for the treatment, regeneration and repair of the spinal cord and brain.

Forward-Looking Statement

This press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, regarding management's expectations, beliefs, goals, plans or prospects should be considered forward-looking. These statements are subject to risks and uncertainties that could cause actual results to differ materially, including delays in obtaining or failure to obtain FDA approval of Fampridine-SR, the risk of unfavorable results from future studies of Fampridine-SR, Acorda Therapeutics' ability to successfully market and sell Fampridine-SR, if approved, and Zanaflex Capsules, competition, failure to protect its intellectual property or to defend against the intellectual property claims of others, the ability to obtain additional financing to support Acorda Therapeutics' operations, and unfavorable results from its preclinical programs. These and other risks are described in greater detail in Acorda Therapeutics' filings with the Securities and Exchange Commission. Acorda Therapeutics may not actually achieve the goals or plans described in its forward-looking statements, and investors should not place undue reliance on these statements. Acorda Therapeutics disclaims any intent or obligation to update any forward-looking statements as a result of developments occurring after the date of this press release.

CONTACT: Acorda Therapeutics
Jeff Macdonald, 914-347-4300 ext. 232
Cell: 917-371-0940
jmacdonald@acorda.com
or
Tierney Saccavino, 914-347-4300 ext. 104
Cell: 917-783-0251
tsaccavino@acorda.com

SOURCE: Acorda Therapeutics, Inc.

Opexa Announces Top-Line Results from Phase IIb Clinical Trial of Tovaxin® for the Treatment of Multiple Sclerosis

2008-09-22T09:00:00.002-06:00

September 19, 2008 02:55 PM Eastern Daylight Time

Tovaxin Shows Favorable Annualized Relapse Rate and Excellent Safety Profile

MONTREAL--(BUSINESS WIRE)--Opexa Therapeutics, Inc. (NASDAQ:OPXA), a company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases such as multiple sclerosis (MS) and diabetes, today announced top-line data from the company’s Phase IIb TERMS (Tovaxin® for Early Relapsing Multiple Sclerosis) study. Top-line results from the study demonstrated a positive trend in the reduction in annualized relapse rate (ARR) for patients treated with Tovaxin as compared to placebo. However, this finding did not achieve statistical significance. In addition, the study did not achieve statistical significance with its primary endpoint, the cumulative number of gadolinium-enhanced brain lesions.

Top-line results from the study showed that Tovaxin-treated patients experienced an ARR of 0.214 as compared to 0.339 for placebo-treated patients. Despite the low relapse rate in the placebo arm, this still represented a 37 percent decrease in ARR for Tovaxin as compared to placebo in the general population. Additionally, in the group of patients who had an ARR > 1 at study entry, Tovaxin demonstrated a 55 percent reduction in ARR as compared to placebo.

The study also demonstrated that Tovaxin was safe and well tolerated with no serious adverse events related to treatment. The most common adverse event related to Tovaxin was mild injection site reaction. Opexa believes that this favorable safety profile may be an important advantage as patient compliance represents a significant challenge due to serious side effects associated with many currently available MS treatments.

It is important to note that initial review of data revealed that patients in the study’s Tovaxin arm, on average had a substantially greater number of MRI brain lesions and corresponding lesion volumes at baseline compared to the average number of MRI brain lesions and lesion volumes per patient in the placebo group. The company believes that this unexpected imbalance may have contributed to the study not achieving its primary and secondary endpoints as patients in the Tovaxin arm began the study with greater disease burden and increased severity of disease.

“The annualized relapse rate of 0.214 seen in the Tovaxin treatment arm is on par with the lowest relapse rates observed with currently available MS treatments which range from 0.2 to 0.9. This rate is also consistent with ARRs that we have seen in the Tovaxin treatment arms in each of the three previously conducted Tovaxin clinical studies,” stated Neil K. Warma, president and chief executive officer of Opexa. “Findings further showed Tovaxin to possess an impressive safety profile with no serious adverse events related to treatment. This level of safety and tolerability addresses a critical unmet need for MS patients. We believe that these positive ARR results combined with an excellent safety profile and convenient dosing place Tovaxin in a very favorable position for continued development as an innovative MS therapy.”

Top-line data from the TERMS study were presented today by Edward J. Fox, M.D., Ph.D., director of the Multiple Sclerosis Clinic of Central Texas and the study’s principal investigator, at the World Congress on Treatment and Research in Multiple Sclerosis in Montreal, Canada.

“Multiple sclerosis is a disease that affects individual patients in distinctly different ways, highlighting the desire for a safe, effective and patient-specific therapy such as Tovaxin. With this in mind, we are pleased with the positive efficacy trend and excellent safety results witnessed in the TERMS study,” stated Dr. Fox. “The Tovaxin-induced reduction in ARR is particularly exciting as it suggests a reduction of clinical activity associated with MS. These study results are encouraging and supportive of further analysis of Tovaxin.”

About the TERMS Study

The TERMS study was a Phase IIb multi-center, randomized, double blind, placebo-controlled trial in 150 patients with Relapsing-Remitting Multiple Sclerosis or high risk Clinically Isolated Syndrome (CIS). The study involved 2:1 randomization with 100 patients receiving Tovaxin and 50 receiving placebo. According to the study protocol, patients received a total of five subcutaneous injections at weeks 0, 4, 8, 12 and 24. The primary efficacy endpoint of the TERMS trial was the cumulative number of gadolinium-enhanced brain lesions (CELs) using MRI scans summed over weeks 28, 36, 44 and 52. The trial’s secondary efficacy endpoints included annualized relapse rate (ARR), new CELs at weeks 28 through 52 and T2-weighted lesion volume compared to baseline.

Top-line data from the TERMS trial is as follows:

* ARR for Tovaxin-treated patients was 0.214 as compared to 0.339 for placebo-treated patients. Consistent with ARRs seen in previously conducted clinical trials for Tovaxin, this result is at the lower end of the spectrum of documented relapse rates demonstrated in controlled two-year clinical studies of currently marketed products (range from 0.2 to 0.9).

* For patients who had an ARR > 1 in the year prior to the study, Tovaxin demonstrated a 55 percent reduction in ARR as compared to placebo.

* Tovaxin was safe and well tolerated with no serious adverse events related to Tovaxin treatment. The most common adverse event was injection site irritation.

* Only 18 patients (12 percent) withdrew from the study prior to completion. The dropout percentage was identical for the Tovaxin and placebo arms of the study, providing further evidence of Tovaxin’s excellent safety and tolerability.

“We are especially pleased with the TERMS study’s ARR results, as this represents the most common efficacy endpoint evaluated by the FDA when approving MS therapeutics. Opexa expects that ARR will serve as the primary endpoint in any pivotal Phase III Tovaxin study,” commented Mr. Warma.

Opexa intends to complete a comprehensive analysis of all data from the TERMS study over the next several months. Based on the TERMS study results, Opexa expects to conduct a Phase II close-out meeting with the United States Food and Drug Administration during the first half of 2009. This meeting, along with the comprehensive results of the TERMS study, will provide important guidance as Opexa plans to advance Tovaxin into Phase III development.

Additionally, Opexa is conducting a one-year, open-label extension trial of the TERMS study called OLETERMS. Approximately 90 percent of patients in the TERMS study have elected to enroll in the OLETERMS trial.

Investigator Q&A Session

Opexa will host an investigator Q&A session following the conclusion of today’s programs at the World Congress on Treatment and Research in Multiple Sclerosis. A live webcast of the Q&A session will be available on Opexa’s web site at www.opexatherapeutics.com beginning at 6:00pm Eastern. The webcast will be archived until October 19, 2008.

Conference Call and Webcast

Opexa will host a conference call and webcast with company management to discuss the Phase IIb TERMS data and provide a corporate update on Monday, September 22, 2008, at 8:30 a.m. Eastern. The conference call can be accessed by dialing 800-230-1074 from the U.S. and 612-332-0228 internationally. Additionally a live webcast of the call will be available on Opexa’s web site at www.opexatherapeutics.com. The webcast will be archived until October 22, 2008.

A replay of the call can be accessed until September 29, 2008 at 11:59 p.m., by dialing 800-475-6701 from the U.S. and 320-365-3844 internationally, and entering the following access code: 960761.

About Tovaxin

Tovaxin is developed using Opexa’s proprietary method for the production of patient-specific T-cell vaccines. To produce the Tovaxin vaccine, Opexa isolates disease-causing T-cells from blood taken from an MS patient and expands them in the laboratory to create an appropriate therapeutic dose. The attenuated T-cells, which comprise the Tovaxin vaccine, are reintroduced into the patient via subcutaneous injection to trigger a therapeutic immune system response. Tovaxin is manufactured in Opexa’s in-house cGMP facility.

Opexa believes that Tovaxin may possess the following competitive advantages:

* Efficacy – Clinical trials conducted to date demonstrate that Tovaxin may result in a reduction in ARR (a key measure of MS treatment effectiveness) for patients with Clinically Isolated Syndrome (CIS), Relapsing-Remitting MS (RRMS) and Secondary-Progressive MS (SPMS) patients comparable to currently available MS therapeutics.
* Safety and Tolerability – Tovaxin treatment selectively targets and depletes the pathogenic T-cell population. It is not a general immune suppressant and accordingly, is not associated with the serious side effects seen with those MS treatments that function by systemically suppressing the immune system. In clinical trials conducted to date, including the 150-patient Phase IIb study, there have been no serious adverse events associated with Tovaxin treatment.

* Improved Compliance – In clinical trials, Tovaxin is administered only five times per year. This patient-friendly treatment regimen may provide significant compliance benefits compared to currently available MS treatments (at least once per month and, in some cases, as frequently as every day.

* Customized Therapy – Using the company’s proprietary Epitope Analysis Assay (EAA) to profile an individual’s disease profile, Opexa can continually customize treatments to specifically target an individual’s disease progression and/or modification.

About Opexa Therapeutics

Opexa Therapeutics is a biotechnology company dedicated to the development of patient-specific cellular therapies for the treatment of autoimmune diseases. The company’s leading therapies currently in development have the potential to address significant unmet medical needs in several large patient populations including multiple sclerosis (MS) and diabetes. The company's lead product is Tovaxin, a T-cell therapy for MS which recently completed a Phase IIb trial. The company also holds an exclusive worldwide license for adult multi-potent stem cells derived from mononuclear cells of peripheral blood. The technology provides means to differentiate these stem cells into other tissue types such as pancreatic islets. By using an individual’s own cells, this approach may minimize threat of treatment rejection. This technology serves as the basis for Opexa’s preclinical diabetes program, which is focused on the generation of insulin-secreting pancreatic-like cells. For more information visit the Opexa Therapeutics website at www.opexatherapeutics.com.

Cautionary Statement Relating to Forward - Looking Information for the Purpose of "Safe Harbor" Provisions of the Private Securities Litigation Reform Act of 1995

This press release contains forward-looking statements which are made pursuant to the safe harbor provisions of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. The forward-looking statements in this release do not constitute guarantees of future performance. Investors are cautioned that statements in this press release which are not strictly historical statements, including, without limitation, statements regarding current or future financial performance and position, management's strategy, plans and objectives for future operations, plans and objectives for product development, plans and objectives for present and future clinical trials and results of such trials, plans and objectives for regulatory approval, litigation, intellectual property, product development, manufacturing plans and performance, constitute forward-looking statements. Such forward-looking statements are subject to a number of risks and uncertainties that could cause actual results to differ materially from those anticipated, including, without limitation, risks associated with: the success of collaborative relationships, our ability to compete with larger, better financed pharmaceutical and biotechnology companies, new approaches to the treatment of our targeted diseases, our expectation of incurring continued losses, our uncertainty of developing a marketable product, our ability to raise additional capital to continue our treatment development programs, the success of our clinical trials, our ability to develop and commercialize products, our ability to obtain required regulatory approvals, our compliance with all Food and Drug Administration regulations, our ability to obtain, maintain and protect intellectual property rights for our products, the risk of litigation regarding our intellectual property rights, our limited manufacturing capabilities, our dependence on third-party manufacturers and value added resellers, our ability to hire and retain skilled personnel, our volatile stock price, and other risks detailed in our filings with the Securities and Exchange Commission. We assume no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.
Permalink: http://www.businesswire.com/news/opexa/20080919005674/en

One-In-Three Multiple Sclerosis Patients Admit They Don’t Like Injecting Their Medication Due to Discomfort, Survey Reveals

2008-09-22T08:46:00.003-06:00

~ Removing Injection Discomfort and Anxiety Key to Medication Adherence in Early Stages of Disease ~

TORONTO – Sept. 11, 2008 – According to a recent North American survey, almost all (97 per cent) people living with Multiple Sclerosis (MS) are committed to controlling their lifelong condition by any means necessary.1 Nonetheless, they are faced with significant barriers that may prevent them from adhering to a treatment regimen. For example, the majority of patients (56 per cent) stated at least one barrier about injections that makes them uncomfortable; most often cited was the length of a needle (33 per cent), followed by the thickness (31 per cent).1 In addition, anxiety was the most common negative emotion around injections (61 per cent).

“The data identifies a need for an improved, patient friendly and effective device that can help patients adhere to therapy, which translates to better management of their symptoms and improved quality of life, with less chance of relapse occurring,” said Nathalie Girouard, RN, Therapeutics Nurse, Ottawa Hospital/MS Clinic. “Adherence to effective and consistent treatment in the early stages is critical in delaying the progression of the disease and providing protection against the development of definite MS.”

According to the survey, 90 per cent of people consider the strongest motivator for starting MS treatment is realizing that it will help slow disease progression.1 Patient motivation can be increased by offering solutions to the barriers, including using the thinnest needle available (70 per cent), co-pay assistance or other financial support (69 per cent), having a good injection technique (69 per cent), and using an autoinjector (55 per cent).1

New Autoinjector Responds to Patients’ Needs

Bayer has launched the new Betaject Lite autoinjector and a 30 gauge needle. It has thinnest needle of all disease modifying therapies, resulting in less pain, fewer injection site reactions, less needle anxiety, and better adherence and long-term outcomes.

The new Betaject Lite autoinjector uses the effective BETASERON® (interferon beta-1b) therapy. The Betaject Lite autoinjector enhances compliance, so patients can benefit from BETASERON in delaying progression of their MS and helping to maintain their quality of life. Since BETASERON was first approved for use in Europe and the United States, this year marks 15 years of achievements for BETASERON in the treatment of patients with MS.

Eighty-two per cent of people surveyed see benefits to using a thinner needle, including less pain during injection (55 per cent), greater comfort during injection (54 per cent), less bruising (42 per cent), less pain after injection (40 per cent), less anxiety immediately before injection (34 per cent) and less impact on mood when anticipating injections (30 per cent).1

“MS is a disabling disease and it is always important to have new treatment options available to manage the complexity of this disease in the easiest most successful way possible,” said Nathalie Girouard, RN, Therapeutics Nurse, Ottawa Hospital/MS Clinic. “I am confident that the Betaject Lite autoinjector and 30 g needle will help to reduce the negative emotions and anxiety that are associated with injections, empower MS patients to better adhere to their medication regimen and take control of their lives.“

The survey also determines that nurses play a pivotal role in supporting and motivating MS patients and helping them cope with the daily challenges of their disease. Patients also value the advice and tips nurses recommend, including ways to avoid side effects and injection site reactions (68 per cent), information on financial assistance (58 per cent), offering a variety of injection techniques (54 per cent) and helping patients choose proper rotation of injection sites (49 per cent).1

About Multiple Sclerosis Canadians have one of the highest rates of MS in the world. 2 This is a common occurrence in countries that are situated further from the equator.2 An estimated 55,000-75,000 Canadians live with multiple sclerosis. 2 It can occur at any age and is usually diagnosed between the ages of 15 to 40. 2 There is no cure for MS.2 It is an unpredictable, often disabling disease of the central nervous system — the brain and spinal cord.2 It can cause loss of balance, impaired speech, extreme fatigue, double vision and paralysis.2 In its most common form, MS has well defined attacks followed by complete or partial recovery. 2

Symptoms vary from person to person and may include double or blurred vision, extreme fatigue, loss of balance, problems with coordination, stiffness of muscles, speech problems, bladder and bowel problems, short-term memory problems, and even partial or complete paralysis.2 Most people who have MS can expect to live an average or close to average life span, due to improvements in the treatment of symptoms and advancements in therapies.2

About Betaject Lite Autoinjector The Betaject Lite autoinjector is approved by Health Canada. It is simple to use, convenient and portable. Features include: a thinner needle (30 gauge instead of the previous 27 gauge); a modern ergonomic design; a built in safety lock at the firing button to reduce the risk of accidental injections; a simple loading procedure and small number of handling steps; and a visual marker that alerts the user when the injection process is over. The Betaject Lite autoinjector is to be used with a new diluent syringe that has an easy to read label, a twist off rubber cap, and larger finger grips and thumb plate designed to simplify manipulation.

BETASERON (interferon beta1-b) is indicated for the treatment of patients with a single demyelinating event accompanied by at least two clinically silent lesions typical of MS on magnetic resonance imaging, to delay progression to definite MS. It is also indicated for the reduction of the frequency of clinical exacerbations in ambulatory patients with relapsing-remitting (RR) MS, characterized by recurrent attacks of neurologic dysfunction followed by complete or incomplete recovery. In addition, BETASERON is indicated for the slowing of the progression in disability and the reduction of the frequency of clinical exacerbations in patients with secondary-progressive (SP) MS.

BETASERON is demonstrated to be highly effective in the treatment of clinically isolated syndrome (CIS) in patients with a first clinical demyelinating event suggestive of MS3,4,+ BETASERON delayed the progression to clinically definite MS (CDMS) by one year.3,4,+

About Bayer Inc.

Bayer Inc. (Bayer) is a Canadian subsidiary of Bayer AG, an international research-based group with core businesses in health care, crop science, and innovative materials.

Headquartered in Toronto, Ontario, Bayer Inc. operates the Bayer Group's HealthCare and MaterialScience businesses in Canada. Bayer Crop Science Inc., headquartered in Calgary, Alberta operates as a separate legal entity in Canada. Together, the companies play a vital role in improving the quality of life for Canadians - producing products that fight diseases, protecting crops and animals, and developing high-performance materials for applications in numerous areas of daily life. Canadian Bayer facilities include the Toronto headquarters and offices in Ottawa and Calgary.

Bayer Inc. has approximately 1,000 employees across Canada and had sales of over $986 million CDN in 2007. Globally, the Bayer Group had sales of over 32 billion Euro in 2007. Bayer Inc. invested approximately $45 million CDN in research and development in 2007. Worldwide, the Bayer Group spent the equivalent of over 2.5 billion Euro in 2007 in R&D.

– 30 –

For more information or to set up an interview, please contact:

Laura Colpitts Mary-Anne Cedrone Bayer Inc. Manning Selvage & Lee (MS&L) Tel: 416-240-5466 Tel: 416-847-1342

This news release contains forward-looking statements based on current assumptions and forecasts made by Bayer Group management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in our public reports filed with the Frankfurt Stock Exchange and with the U.S. Securities and Exchange Commission (including our Form 20-F). The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.)

References

1 Russell Research Survey, 2008 2 MS Society of Canada. Website. Available at: http://www.mssociety.ca/en/information/faq.htm#3: 3 Kappos L et al. Treatment with interferon beta-1b delays conversion to clinically definite and McDonald MS in patients with clinically isolated syndromes. Neurology 2006; 67(7):1242-9. 4 BETASERON® Product Monograph. Bayer Inc., December 21, 2007.

+ Double-blind, placebo-controlled, randomized, parallel-group clinical trail in patients aged 18 to 45 years with a single clinical demyelinating event suggestive of MS and an expanded disability status scale (EDSS) score of <5.0. Patients were randomized to receive either 250ug BETASERON (n=292) or placebo (n=176), administered subcutaneously every other day for a treatment duration of up to 2 years. BETASERON prolonged the time to CDMS by 363 days, from 255 days in the placebo group to 618 days in the BETASERON group (based on the 25th percentiles).

Positive Sativex® Study Confirms Long Term Efficacy in MS Neuropathic Pain

2008-09-08T09:00:00.002-06:00

08/09/2008

Results Support Designof Ongoing Phase III MS Spasticity Study

Porton Down, UK, 8 September 2008: GW Pharmaceuticals plc (GWP:AIM) announces positive results from a placebo-controlled “randomized withdrawal” study of Sativex® in patients with neuropathic pain due to Multiple Sclerosis (MS). This study design is described by regulators as being sufficient to satisfy the need for long-term efficacy data.

This randomized withdrawal study evaluated 42 MS patients with central neuropathic pain who had previously been in a Sativex Phase III MS neuropathic pain study and who continued to take Sativex on an open label basis for 12 weeks. They were then randomized to Sativex or placebo for a further 4 weeks in a double-blinded manner. During the randomized period, patients were not permitted to adjust their dose. The purpose of this blinded 4-week “randomized withdrawal” study was to assess the maintenance of pain control in patients who remain on Sativex versus those who switch to placebo.

In the patients who were randomized to Sativex pain scores remained stable. In the patients randomized to placebo, pain and sleep scores deteriorated. The prospectively defined primary efficacy endpoint of the study - the time to treatment failure - was statistically significantly in favour of Sativex (p=0.036). The difference between Sativex and placebo was also significant for mean pain score (p=0.028) and sleep quality (p=0.015). The results of all other symptom-related endpoints showed that Sativex patients maintained or improved their response whilst the symptoms of those who switched from Sativex to placebo worsened in the 4 weeks following cessation of active treatment. During the randomized withdrawal period, there were 2 patients with adverse events on Sativex, and 5 on placebo. One patient on placebo withdrew from the study. There was no evidence of any withdrawal syndrome.

Until now, all the evidence for long-term maintenance of efficacy of Sativex has come from long-term open-label exposurei. The results reported today confirm in the context of a placebo-controlled double-blind study that efficacy is indeed maintained in long-term use.

The results of this study are of further significance to GW since the design bears important similarities to the ongoing Phase III MS spasticity study requested by the UK regulator prior to granting approval for Sativex. This ongoing Phase III study involves all patients receiving Sativex for 4 weeks, following which Sativex responders are randomized to continue on Sativex or switch to placebo for a further 12 weeks. This study is due to report results in Q1 2009 with a regulatory submission targeted for H1 09.

Dr Stephen Wright, GW’s R&D Director, said: “This is the first placebo-controlled study showing that Sativex provides long term efficacy for MS patients with neuropathic pain and supplements previously published open-label studies. In addition, these results support the design of the ongoing Phase III trial in MS spasticity. It is encouraging to note that if the difference between Sativex and placebo achieved in the results today are replicated in the ongoing Phase III MS spasticity study, this Phase III study will meet its objectives.”

Enquiries:

GW Pharmaceuticals plc (Today) + 44 20 7831 3113
Dr Geoffrey Guy, Executive Chairman (Thereafter) + 44 1980 557000
Justin Gover, Managing Director

Financial Dynamics + 44 20 7831 3113
David Yates / John Dineen

Notes to Editors

About GW
GW was founded in 1998 and listed on the AiM, a market of the London Stock Exchange, in June 2001. Operating under license from the UK Home Office, the company researches and develops cannabinoid pharmaceutical products for patients who suffer from a range of serious ailments, in particular pain and other neurological symptoms. GW has assembled a team of over 100 scientists with extensive experience in developing both plant-based prescription pharmaceutical products and medicines containing controlled substances. GW occupies a world leading position in cannabinoids and has developed an extensive international network of the most prominent scientists in the field.

iRog DJ et al. Clinical Therapeutics. 2007; 29: 2068-2079