Opexa Announces Top-Line Results from Phase IIb Clinical Trial of Tovaxin® for the Treatment of Multiple Sclerosis

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.
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One-In-Three Multiple Sclerosis Patients Admit They Don’t Like Injecting Their Medication Due to Discomfort, Survey Reveals

~ 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

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

New Drugs in late-stage trials offer promise for sufferers of the chronic and crushing disease multiple sclerosis

Two Steps Forward

New drugs in late-stage trials offer promise for sufferers of the chronic and crushing disease multiple sclerosis

Mary Ellen Egan 08.07.08, 6:00 PM ET
Forbes Magazine dated September 01, 2008




Robin and Clifford Giese, with Kevin Giese at right.


Robin Giese, 59, kicks off each day by getting out of her wheelchair for a half-hour ride on a stationary bike followed by 30 minutes of stretching exercises. Most afternoons she visits friends or one of of her five grandchildren, and in the evenings she and her husband, Clifford, entertain guests or go out to dinner.

Giese hasn't always been so active. She has multiple sclerosis, the degenerative disease of the central nervous system that afflicts 400,000 Americans. In MS the immune system attacks myelin, a fatty substance that protects nerve fibers much the way insulation protects electrical wires. When the unprotected nerve fibers, or axons, are damaged, signals are blocked or delayed traveling to and from the brain. This causes a variety of symptoms that can include blurred vision, incontinence, difficulty walking and paralysis.

Over three decades multiple sclerosis has slowly robbed Giese of her mobility and weakened her muscles, and without treatment she would be all but immobilized in her wheelchair. But an experimental drug has changed the course of her disease--and her husband's career path.

The compound, called dirucotide, is a chain of 17 amino acids that mimics a portion of the protein in myelin. It works by acting as a decoy to divert the attacking immune cells. It has had such a profound impact on Robin's condition that her husband has started a company, BioMS Medical, to bring it to market. Today dirucotide is one of two novel MS drugs in late-stage clinical trials. The other, from a small firm called Acorda, improves muscle strength.

There are four kinds of MS. Most sufferers are first given a diagnosis of a mild relapsing form of the disease marked by occasional flare-ups followed by months or years without symptoms. Ninety percent of patients with this condition eventually develop a progressive MS characterized by continuous deterioration. The two remaining types of MS, less common, entail a rapid decline.

Most existing MS therapies work by suppressing the immune system, and they're generally effective only when the disease is at an early stage. They include Biogen Idec (nasdaq: BIIB - news - people )'s monoclonal antibody Tysabri, beta interferons and anticancer drugs. They can be helpful (there is no cure), but their side effects can range from flu-like symptoms to fatal viral infections of the brain.

Dirucotide began with research conducted at the University of Alberta by doctors Kenneth Warren and Ingrid Catz. In 1989 Warren developed a synthetic peptide that mimics myelin protein. He began testing his compound on MS patients in 1994, and Robin Giese, who became a patient of his that same year, received her first infusion in 1996. Three weeks later, she says, her energy level had increased dramatically, and her head, which had felt "fuzzy" for years, was suddenly "clear." "It was the best I'd felt in years," she says.

Even though the drug didn't allow her to throw away her wheelchair, she credits it with restoring her zest in life. "Before I started taking it, I couldn't predict how I'd feel or what I could do each day. Now I wake up feeling great and have energy for the entire day." She gets dirucotide infusions twice a year.

The university lacked money for clinical trials, so Clifford, who had made a fortune with a chain of Canadian oil-change stores, stepped in to help. He and his brother, Kevin, licensed the drug from the university and founded BioMS in September 2000. The following year they started selling shares to the public, and they've raised $180 million so far on top of the $1 million that they estimate they invested themselves at the outset. Kevin took the role of chief executive, and Clifford became chairman.

BioMS moved to late-stage clinical trials of dirucotide in January 2005 and has done them with 611 patients with the progressive form of MS. Testing should wrap up in mid-2010.

The results so far have been very promising. Patients with either of two genes associated with autoimmune disorders, HLA-DR2 and HLA-DR4, have gone five years without any progression of the disease. Those genes are found in 65% to 75% of all MS patients, and because of that analysts estimate that the potential market for drugs like dirucotide, effective for patients at later stages of the disease, could reach $10 billion a year. The current market for all existing MS drugs is $6 billion.

Acorda's drug, Fampridine-SR, works differently from dirucotide and specifically addresses one of the most devastating symptoms of the disease, loss of muscle strength. Its key compound, 4-aminopyridine, has been around for 100 years. Academic researchers originally used the synthesized chemical to study nerve cell conduction. Not until the 1980s did they figure out how it works and how it might help with MS.

In MS the damage to myelin exposes channels on the surface of the axon, allowing potassium ions to leak out and thus dissipate the electrical current that carries nerve signals. The 4-aminopyridine molecule patches the exposed channels so the current can pass through.

But, early on, using the compound in humans proved to be tricky. Dosages were hard to control, and patients given too much had seizures. The drug languished until the mid-1980s, when Elan Corp. (nyse: ELN - news - people ), an Irish firm, began looking for ways to reformulate it.

In 1994, after a decade of tinkering, Elan began testing a sustained-release version in patients with MS. A year later Acorda, then privately held, approached Elan for permission to test Fampridine-SR, as the new version was named, in spinal cord injury patients. Acorda began clinical trials in 1998 and, five years later, when Elan was struggling, secured the rights for Fampridine-SR for all applications.

Acorda started its tests of Fampridine-SR in MS patients in late 1999, and in June this year the now public company completed late-stage trials in 540 patients with all four types of MS. The results are impressive: 43% of the patients showed consistent improvement in walking speed, as against 9% of patients on a placebo.

"One of the first questions patients ask is if they'll be in an wheelchair. Anything that helps to keep them out of a wheelchair longer is very important," says Dr. Hillel Panitch, one of the drug's clinical investigators and director of the University of Vermont's Multiple Sclerosis Center. Acorda plans to submit its data to the FDA early next year and ask the agency for fast-track approval.

"One of the first questions patients ask is if they'll be in an wheelchair. Anything that helps to keep them out of a wheelchair longer is very important," says Dr. Hillel Panitch, one of the drug's clinical investigators and director of the University of Vermont's Multiple Sclerosis Center. Acorda plans to submit its data to the FDA early next year and ask the agency for fast-track approval.

BIOMS MEDICAL’S LEAD DRUG, DIRUCOTIDE (MBP8298) FOR THE TREATMENT OF MULTIPLE SCLEROSIS, RECEIVES FAST TRACK DESIGNATION FROM FDA

Edmonton, Alberta, September 4, 2008 – BioMS Medical Corp. (TSX: MS), a leading developer in the treatment of multiple sclerosis (MS), today announced that the Food and Drug Administration (FDA) of the United States has granted fast track designation for the Company’s lead drug, dirucotide (MBP8298), for the treatment of secondary progressive MS (SPMS). Dirucotide (MBP8298) is currently being evaluated in a U.S. pivotal phase III trial, named MAESTRO-03, at 68 sites with approximately 510 patients.

Fast track designation is an FDA status reserved for products that are intended to treat a serious or life-threatening condition and that demonstrate the potential to address unmet medical needs for that condition. Fast track designation can potentially facilitate development and expedite the review process.

“Our receipt of fast track designation for dirucotide in the U.S. is a significant milestone for both BioMS Medical and the MS community," said Kevin Giese, President and CEO of BioMS Medical. “Based on previous clinical results, we believe dirucotide is well-positioned to become a first-in-class treatment for secondary progressive MS patients, a large patient population with very limited treatment options.”

About MAESTRO-03
The MAESTRO-03 U.S. pivotal phase III clinical trial is a randomized, double-blind study that has completed recruitment of approximately 510 patients at 68 clinical sites who will be administered either dirucotide (MBP8298) or placebo intravenously every six months for a period of two years. The primary clinical endpoint for the trial is defined as a statistically and clinically significant increase in the time to progression of the disease as measured by the Expanded Disability Status Scale (EDSS), in patients with HLA-DR2 and/or HLA-DR4 immune response genes (up to 75% of all MS patients are HLA-DR2 and/or HLA-DR4 positive).



About Dirucotide (MBP8298)
Dirucotide (MBP8298) is a synthetic peptide that consists of 17 amino acids having a sequence identical to that of a portion of human myelin basic protein (MBP). Dirucotide is being developed for the potential treatment of multiple sclerosis (MS), an autoimmune disease caused by immune attack against normal components of the central nervous system. The sequence of dirucotide is associated with the autoimmune process in MS patients with certain immune response genes (HLA types DR2 and/or DR4); MS patients having these genes represent 65 to 75 percent of all MS patients.

The drug’s apparent mechanism of action is the induction or restoration of immunological tolerance with respect to ongoing immune attack as a result of high doses of peptide periodically delivered intravenously. The potential benefit of the drug for any individual patient is therefore expected to be related to the role this peptide plays in that patient’s immune system. The degree of immunomodulation achieved will depend on the relationship among the peptide, HLA molecules and T cells.

The results of phase II and long-term follow-up treatment of MS patients with MBP8298 (dirucotide), published in 2006 in the European Journal of Neurology (EJN), showed that MBP8298 (dirucotide) safely delayed median time to disease progression for five years (versus placebo) in progressive MS patients with HLA types DR2 and/or DR4. Thus, dirucotide (MBP8298), if approved, has the potential to be used as a tailored therapy for patients genetically determined to express the appropriate HLA molecules.

Dirucotide (MBP8298) is being studied in four late-stage clinical trials:

• MAESTRO-01: A pivotal phase II/III trial for secondary progressive MS (SPMS) patients in Canada and Europe.
• MAESTRO-02: An open-label safety extension study to MAESTRO-01.
• MAESTRO-03: A pivotal phase III trial for SPMS patients in the United States.
• MINDSET-01: A phase II trial for relapsing-remitting MS (RRMS) patients in Europe.



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

BIOMS MEDICAL ANNOUNCES POSITIVE INTERIM ANALYSIS ON PHASE III TRIAL OF DIRUCOTIDE (MBP8298) FOR MULTIPLE SCLEROSIS

- Milestone triggers $10 million payment from Eli Lilly and Company -


Edmonton, Alberta, August 13, 2008 – BioMS Medical Corp. (TSX: MS), a leading developer in the treatment of multiple sclerosis (MS), today announced that the independent Drug Safety Monitoring Board (DSMB) for the MAESTRO-01 trial has conducted the scheduled interim analysis of efficacy and safety and has recommended that the trial continue to completion. MAESTRO-01 is the pivotal phase II/III Canadian and European study of dirucotide (MBP8298) in patients with secondary progressive MS.

The interim analysis included patients from the first 200 to complete MAESTRO-01 and assessed the likelihood of the study reaching its primary endpoint at the end of the trial in MS patients with the target HLA-DR2 and/or HLA-DR4 immune response genes. The DSMB analysis also included a scheduled review of safety information.

Based on the DSMB decision, Eli Lilly and Company has agreed to provide the $10 million milestone payment to BioMS as part of the terms of the licensing and collaboration agreement.

“We are very encouraged by the safety board’s recommendation,” said Kevin Giese, President and CEO of BioMS Medical. “This positive review is an important milestone for BioMS and our partner, Eli Lilly and Company, and moves us one step closer to our goal of bringing this important therapy to multiple sclerosis patients.”

"We are pleased by the results of the interim analysis and look forward to final efficacy and safety data from this trial next year," said Dr. Mark Freedman, Professor of Neurology at the University of Ottawa and Director of the MS Research Clinic at the Ottawa Hospital. "If successful, this novel therapy administered only twice per year, could help a large underserved population with late stage MS."



About MAESTRO-01
Dirucotide (MBP8298) is being studied in four late-stage clinical trials:

• MAESTRO-01: A pivotal phase II/III trial for secondary progressive MS (SPMS) patients in Canada and Europe.
• MAESTRO-02: An open-label safety extension study to MAESTRO-01.
• MAESTRO-03: A pivotal phase III trial for SPMS patients in the United States.
• MINDSET-01: A phase II trial for relapsing-remitting MS (RRMS) patients in Europe.

MAESTRO-01 is a multi-center, double-blind, placebo-controlled trial designed to evaluate the safety and efficacy of dirucotide (MBP8298) in patients with secondary progressive MS. The study is being conducted at 47 sites across Canada and nine countries in Europe and includes 611 patients being administered either dirucotide (MBP8298) or placebo intravenously every six months for a period of two years. The primary clinical endpoint for the trial is defined as a statistically and clinically significant increase in the time to progression of the disease, as measured by the Expanded Disability Status Scale (EDSS), in patients with HLA-DR2 and/or HLA-DR4 immune response genes. Time to disease progression in patients with other HLA-DR types will be assessed separately as an exploratory arm of the same study.

About Dirucotide (MBP8298)
Dirucotide (MBP8298) is a synthetic peptide that consists of 17 amino acids having a sequence identical to that of a portion of human myelin basic protein (MBP). Dirucotide is being developed for the potential treatment of multiple sclerosis (MS), an autoimmune disease caused by immune attack against normal components of the central nervous system. The sequence of dirucotide is associated with the autoimmune process in MS patients with certain immune response genes (HLA types DR2 and/or DR4); MS patients having these genes represent 65 to 75 percent of all MS patients.

The drug’s apparent mechanism of action is the induction or restoration of immunological tolerance with respect to ongoing immune attack as a result of high doses of peptide periodically delivered intravenously. The potential benefit of the drug for any individual patient is therefore expected to be related to the role this peptide plays in that patient’s immune system. The degree of immunomodulation achieved will depend on the relationship among the peptide, HLA molecules and T cells.

The results of phase II and long-term follow-up treatment of MS patients with MBP8298 (dirucotide), published in 2006 in the European Journal of Neurology (EJN), showed that MBP8298 (dirucotide) safely delayed median time to disease progression for five years (versus placebo) in progressive MS patients with HLA types DR2 and/or DR4. Thus, dirucotide (MBP8298), if approved, has the potential to be used as a tailored therapy for patients genetically determined to express the appropriate HLA molecules.

About Multiple Sclerosis
Multiple sclerosis (MS) is thought to affect as many as 2.5 million people worldwide, including approximately 75,000 in Canada, 400,000 in the United States and more than 500,000 in Europe. It is a disease that affects more women than men, with onset typically occurring between 20 and 50 years of age. MS is caused by damage to myelin, the protective sheath surrounding nerve fibers in the central nervous system, which interferes with messages from the brain to the body. Symptoms of MS may include vision problems, loss of balance, numbness, difficulty walking and paralysis. Approximately 40 percent of all MS patients have the secondary progressive form of the disease.


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

Europe assessing Tysabri brain disease reports

14 August 2008
Europe’s medicines agency yesterday said it is currently in the process of assessing the two additional reports of the rare brain disease progressive multifocal leukoencephalopathy in multiple sclerosis patients receiving Elan Pharma’s Tysabri (natalizumab).

The two cases reported at the end of July occurred in patients who had been receiving Tysabri for more than 12 months, but renewed old fears for investors and the firm’s share price – along with that of its US partner Biogen Idec – sank on the news. The Ireland-based firm crashed almost 75% - reflecting the sensitivity and importance of the medicine to its future prospects, though it was also impacted by concerns over its Alzheimer’s disease drug bapineuzumab being developed with Wyeth.

The agency has also asked Elan to provide any additional information it may have, at which point its scientific advisory panel will decide whether the medicine’s product labeling needs updating. Tysabri has been under intense scrutiny since it was reintroduced in the USA and approved in Europe two years ago – and these are the first new cases to surface since that time. It was originally withdrawn from the market in 2005 after three patients developed PML, but following its reintroduction has gone on to be a big earner – pulling in some $200 million in the second quarter of this year alone. Almost 32,000 patients were receiving Tysabri as of the end of June.

Most analysts do seem to remain convinced of the value of Tysabri – certainly the firms do not intend to withdraw the drug again, stressing the stringent safety monitoring programme in place.

Icahn takes stake in Biogen on Tysabri woes
And rather than backing off from the stock, maverick investor Carl Icahn has seen the dip in Biogen’s value as an opportunity to snap up some cut-price shares – increasing his stake in the company to 6% from 4.3%. All eyes are on his movements: earlier on this year he criticised the board for not looking hard enough to find a buyer as well as its decision to take down the ‘for sale’ sign in December last year month after no serious offers were made. He could be gearing up to push for another sale.

Prime Therapeutics Releases Annual Drug Trend Insights Report

Annual spending on prescription drugs posts lowest increase ever recorded; rising acceptance of generic medications and flat usage rates offset effects of inflation


ST. PAUL, Minn., Aug. 13 /PRNewswire/ -- Prime Therapeutics, a thought
leader in pharmacy benefit management, today released its 2008 Drug Trend
Insights report which revealed that its 2007 prescription drug trend increased
by just 2.9 percent -- the lowest annual increase it ever recorded, and less
than half of what the company recorded in 2006.

Tim Dickman, Prime Therapeutics' president and CEO, said the biggest
factor in the low drug trend was the rising acceptance of generic medications,
which shifted the drug mix toward lower cost drugs and offset cost increases
due to inflation. Prime's generic drug utilization rose five percent during
2007, to 56.7 percent of total prescriptions filled.

"We're very encouraged with how we've been able to drive generic
utilization for our members and consumers because we know that the use of
generic medications is the most effective way to keep prescription drug
spending manageable," said Dickman. "A lack of blockbuster drugs in the
pharmaceutical development pipeline along with increasing acceptance of
generic drugs by consumers suggests that the low increase we saw during 2007
will be sustained for some time to come."

According to the U.S. Centers for Medicare and Medicaid Services, spending
on prescription drugs accounts for approximately ten percent of total health
care expenditures each year. As the most commonly accessed health benefit, the
sector provides unique opportunities to slow or reduce the rising costs of
health care, helping to keep coverage as affordable and accessible as
possible.


In addition to overall drug spending, the 2008 Drug Trend Insights found
that during 2007:


-- Prescription fill rates were relatively flat -- up just two percent
from 2006.

-- Cholesterol (lipid lowering) medications represented the largest drug
cost category (8.6 percent of total drug spending), followed by medications
for blood pressure management and depression.

-- Lifestyle drugs (contraceptives, smoking cessation medications, acne
treatments and other prescriptions pursued for reasons other than illness) saw
the greatest annual spending increase among all drug categories, at 24
percent.

-- The fastest growing per member per month (PMPM) drug spending was seen
for attention deficit hyperactivity disorder (ADHD), anticonvulsant (for
management of seizures) and respiratory disorder medications, respectively.

-- Specialty drugs (those drugs generally prescribed for people with
complex, ongoing medical conditions such as multiple sclerosis, hemophilia,
hepatitis and rheumatoid arthritis) accounted for 14.1 percent of drugs
prescribed, and an overall increase in spending over 2006 of 8.9 percent.


Most notable in the area of quality improvement, the report cited two
Prime studies that found a correlation between out-of-pocket costs and the
length of time members continue taking prescribed medications. Specific to
specialty drugs, where out-of-pocket costs can be extremely high under
traditional plan structures, Prime found that members facing a copay greater
than $250 were 4.6 times more likely to decline to fill the prescription. This
can result in decreased quality of life and lead to even more expensive
in-hospital costs. For this reason, Prime recommends that plans consider
including an out-of-pocket maximum on co-pays for specialty drugs.


Drug Trend Insights is Prime's annual report on the factors that influence
prescription drug spending, along with a review of its efforts to control cost
increases while improving health care quality. Prime publishes this report to
help clients better understand the role of pharmacy benefits within the larger
health care environment. Offering detailed data as well as insights on
industry trends, the report can be used as a tool to guide future pharmacy
benefit decisions.


For a copy of Prime Therapeutics 2008 Drug Trend Insights, visit
http://www.primetherapeutics.com.


Prime Therapeutics LLC is a pharmacy benefit management company dedicated
to providing innovative, clinically-based, cost-effective pharmacy solutions
for clients and members. Providing pharmacy benefit services nationwide to
approximately 14.6 million covered lives, its client base includes Blue Cross
and Blue Shield Plans, employer and union groups, and third party
administrators. Headquartered in St. Paul, Minnesota, Prime Therapeutics is
collectively owned by 10 Blue Cross and Blue Shield Plans, subsidiaries or
affiliates of those Plans. Learn more at http://www.primetherapeutics.com.



SOURCE Prime Therapeutics LLC



Copyright © 2008 PR Newswire. All rights reserved.

World-Class MS Information At The Touch Of A Button

People affected by the debilitating neurological condition multiple sclerosis (MS) now have world-class information at their fingertips thanks to a UK first by the MS Society.

The charity, which is the largest of its kind supporting people affected by MS, has created an online library of the thousands of books, journals, papers and magazines that it has in its collection, searchable from anywhere with an internet connection.

The database opens up the library to the world and now anyone interested in any aspect of MS can see what's available, download documents and texts and request loans.

MS Society librarian, David Bates, said: "For years the MS Society has had a library available to support the information needs of people affected by MS, but there has been no way for people to search it themselves and access the documents.

"This new facility opens up the world-class information we have from leading authors to people across the UK, and around the world."

The library includes information aimed at lay audiences, children and professionals and features the full text of MS Society publications including all of the Essentials series and a wide selection of articles from MS Society membership magazine MS Matters from the last four years.

Journals are available too. If the Library subscribes to the relevant journal, or the article is open access, there will be a link straight to the full article. If not, the abstract will be available and the full article available on request.

"If you can't access an article electronically, or want to request something we don't already have, get in touch," David added. "This is a fantastic resource and the first of its kind in the UK."

To access the library, go to http://www.mssociety.org.uk/library

Pluristem's PLX-MS Shows Potential Benefit in the Prevention of Multiple Sclerosis

Monday August 11, 7:00 am ET

NEW YORK--(BUSINESS WIRE)--Pluristem Therapeutics Inc. (NasdaqCM:PSTI) (DAX:PJT) a bio-therapeutics company dedicated to the commercialization of non-personalized (allogeneic) cell therapy products for a variety of degenerative, ischemic and autoimmune indications, today announced that the Company’s PLacental eXpanded (PLX-MS) cells have demonstrated in vivo efficacy in the prevention of Multiple Sclerosis (MS). PLX cells are Pluristem’s placental-derived mesenchymal stromal cells (MSCs) that have been expanded in the Company’s proprietary PluriX™ 3-D bioreactor.

In a further analysis aiming to demonstrate the in vivo efficacy of PLX-MS cells for the prevention of MS, Experimental Autoimmune Encephalitis (EAE) was induced in mice via immunization with the MOG35-55 protein on day 0. EAE is an autoimmune inflammatory disease of the CNS that represents the paradigmatic model for MS. The animals then received, on day 8, intravenously either PLX-MS or PlasmaLyte, which served as a control. PLX-MS administration prevented the appearance of clinical symptoms and signs associated with MS throughout the 35-day study period compared to those animals receiving the control. Additionally, the beneficial effects were similar to when Zappia et. al. used MSCs that were non-placental in origin in this EAE animal model†.

Mr. Zami Aberman, Pluristem’s President and CEO, commented: “This trial’s remarkable results demonstrated our PLX-MS cells’ ability to prevent the appearance of multiple sclerosis symptoms and showed the potential for our PLX cells to treat global autoimmune diseases. As a cellular therapy, our PLX cells, which are derived from human placenta, a non-controversial, non-embryonic, adult stem cell source, and stored ready-to-use, could prove to be a readily available preventive therapeutic alternative for these disorders."

Pluristem is initiating repeated sets of EAE experiments at the Berlin-Brandenburg Center for Regenerative Therapy (BCRT) at Charité - University Medicine Berlin, one of the largest independent clinical research centers in Europe.

†Zappia et. al. Mesenchymal stem cells ameliorate experimental autoimmune encephalitis inducing T cell anergy. Blood. 2005;106: 1755-1761

About Multiple Sclerosis (MS)

Multiple sclerosis (MS), also known as disseminated sclerosis or encephalomyelitis disseminate, is an autoimmune condition in which the immune system attacks the central nervous system (CNS), leading to demyelination. Myelin is the insulating sheath that surrounds nerve cells (neurons). MS may cause numerous physical and mental symptoms, and often progresses to physical and cognitive disability. The World Health Organization (WHO) estimates that over 2.5 million people globally suffer from MS, which represents a current market of approximately $5.4 billion for disease-modifying agents to treat the disorder.

About Pluristem

Pluristem Therapeutics Inc. is a bio-therapeutics company dedicated to the commercialization of non-personalized (allogeneic) cell therapy products for the treatment of several severe degenerative, ischemic and autoimmune disorders. The Company is developing a pipeline of products, stored ready-to-use, that are derived from human placenta, a non-controversial, non-embryonic, adult stem cell source.

These placental mesenchymal stromal cells (MSCs) are expanded in the Company's proprietary PluriXTM 3D bioreactor, which imitates the natural microstructure of bone marrow and does not require supplemental growth factors or other exogenous materials. Pluristem believes that the resultant PLX (PLacental eXpanded) cells are multi-potent and able to differentiate into a variety of cell types. Recent evidence also suggests their efficacy may be related to the secretion of cytokines or other potent immune modulators. Furthermore, PLX cells are immune privileged and have immunomodulatory properties, thus protecting the recipient from immunological reactions that often accompany transplantations.

Pluristem's first product in development, PLX-PAD, is intended to improve the quality of life of millions of people suffering from peripheral artery disease (PAD). The Company's products in development also include PLX-BMT, targeting the global shortfall of matched tissue for bone marrow transplantation (BMT) by improving the engraftment of hematopoietic stem cells (HSCs) contained in umbilical cord blood; PLX-STROKE, targeting ischemic stroke; PLX-MS, targeting Multiple Sclerosis; and PLX-IBD, targeting Inflammatory Bowel Disease (IBD), which includes Crohn’s disease and Ulcerative Colitis.

Pluristem has offices in the USA with research and manufacturing facilities in Israel.

See our product animation on YouTube: http://www.youtube.com/watch?v=OFhWXyJT6Us

Safe Harbor Statement

This press release contains forward-looking statements within the meaning of the "safe harbor" provisions of the Private Securities Litigation Reform Act of 1995 and federal securities laws. For example, when we say that the trial’s remarkable results demonstrated our PLX-MS cells’ ability to prevent the appearance of multiple sclerosis symptoms and showed the potential for our PLX cells to treat global autoimmune disease, or that as a cellular therapy, our PLX cells could prove to be a readily available preventive therapeutic alternative for these disorders, we are using forward-looking statements. These forward-looking statements are based on the current expectations of the management of Pluristem only, and are subject to a number of factors and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. The following factors, among others, could cause actual results to differ materially from those described in the forward-looking statements: changes in technology and market requirements; our technology may not be validated as we progress further and our methods may not be accepted by the scientific community; we may be unable to retain or attract key employees whose knowledge is essential to the development of our products; unforeseen scientific difficulties may develop with our process; results in the laboratory may not translate to equally good results in real surgical settings; our patents may not be sufficient; our products may harm recipients; changes in legislation; inability to timely develop and introduce new technologies, products and applications; loss of market share and pressure on pricing resulting from competition, which could cause the actual results or performance of Pluristem to differ materially from those contemplated in such forward-looking statements. Except as otherwise required by law, Pluristem undertakes no obligation to publicly release any revisions to these forward-looking statements to reflect events or circumstances after the date hereof or to reflect the occurrence of unanticipated events. For a more detailed description of the risk and uncertainties affecting Pluristem, reference is made to Pluristem's reports filed from time to time with the Securities and Exchange Commission.

For more information visit our website at www.pluristem.com, the content of which is not part of this press release.




Contact:

Pluristem Therapeutics Inc.
William Prather RPh, MD, 303-883-4954
Sr. VP Corporate Development
bill@pluristem.com

Source: Pluristem Therapeutics Inc.

High risk of conversion to multiple sclerosis predicted by gene activity

Source: UCSF News Services
Date: 11 August 2008

Scientists have identified a pattern of gene activity that predicts which patients who experience the first clinical symptoms of multiple sclerosis – known as clinically isolated syndrome – are at high risk of converting to the full blown disease.

The finding, reported in the current issue of Proceedings of the National Academy of Sciences (Aug. 5, 2008), identifies potential candidates who may benefit from early therapy for preventing conversion to multiple sclerosis, the researchers say. It also reveals a genetic landscape for studying the earliest molecular events of the disease.

"This is a very exciting development," says the senior author of the study, Sergio E. Baranzini, PhD, assistant professor of neurology and a member of the Multiple Sclerosis Research Group at University of California, San Francisco.

Currently, there is no definitive way of predicting whether patients who present with clinically isolated syndrome will convert to multiple sclerosis. Magnetic resonance imaging of the brain is key in the diagnosis and clinical surveillance of MS, but it is only moderately effective in forecasting conversion in CIS patients. There also are no known biological markers in the spinal fluid or blood that accurately predict conversion to MS.

"This creates a dilemma for neurologists," says Baranzini. "They don’t know when – or if – to begin treatment. If neurologists knew which patients were at high risk for rapid progression to MS, they could treat them with disease-modifying therapy that appears to be beneficial in early MS."

Patients with clinically isolated syndrome experience a single neurological insult, such as vision or gait problems, that lasts for several days. The symptoms result from an attack by cells of the immune system on myelin, the sheath that insulates nerve cells in the central nervous system. The attack, which disrupts communication between nerve cells, is the hallmark of multiple sclerosis. Approximately one third of CIS patients progress to relaxing-remitting multiple sclerosis, the most common form of the disease, within a year, and about half do so after two years. An estimated 10 percent remain free of further attacks forever.

In the study, led by Jean-Cristophe Corvol, PhD, at the time a postdoctoral fellow in the Baranzini lab, the UCSF team set out to examine the genes expressed in the immune system's CD4+ T cells, which are known to be one of the key culprits in the attack against myelin. They obtained CD4+ T cells from blood samples of 37 patients who had just been diagnosed with CIS and 29 healthy people, or "controls." Then, using a technique known as microarray analysis, a computational technique that reveals gene activity on a glass slide, they documented the pattern of genes expressed at the time of diagnosis and after one year. They also followed the patients clinically and with MRI for at least 30 months, so by end of the study they knew which converted to MS and which did not.

The researchers first focused on the circa 1,700 genes in patients' CD4+ T cells whose expression varied most across all samples. They then honed in on 975 genes that showed a distinct molecular signature between CIS patients and healthy controls.

On the basis of how actively genes were transcribed, the genes segregated CIS patients into four distinct subgroups. Significantly, patients in "subgroup 1," representing 108 genes, converted to multiple sclerosis much more quickly than those in the other groups and had a much higher risk of conversion.

"Remarkably, 100 per cent of the patients in subgroup 1 converted to MS within nine months," says Baranzini. "In contrast, only 20 per cent of patients from the other groups converted in the same period of time. And even after 30 months, only 50 percent of them converted."

Of particular note, a gene known as TOB1 was consistently down regulated, in sub group 1. TOB1 normally functions as a key regulator of CD4+ T cell proliferation. When it is normally expressed, it prevents cells from proceeding through their cell cycle, a process that culminates with the cells dividing and proliferating.

"The fact that the gene is less active in these cells suggests that CIS patients at high risk of conversion have impaired regulation of CD4+ T cell quiescence, possibly resulting in earlier activation of pathogenic CD4+ T cells," says Jorge R Oksenberg PhD, another key member of the study.

"We're not sure if what we're seeing is a cause or earlier molecular effect already going on in these patients. Regardless, we believe TOB1 is part of a signature that together with other cell cycle genes suggests a pathway for therapeutically targeting patients with CIS."

Other co-authors of the study were Daniel Pelletier, MD; Stacy J. Caillier, BSc; Joanne Wang, MPH; Derek Pappas, PhD; Simona Casazza, PhD; Darin T. Okuda, MD, and Stephen L. Hauser, MD, all of the UCSF Department of Neurology, and Roland G. Henry, PhD, UCSF Department of Radiology.

The study was funded by the National Multiple Sclerosis Society.

UCSF is a leading university dedicated to promoting health worldwide through advanced biomedical research, graduate-level education in the life sciences and health professions, and excellence in patient care.

IMPAX Reports Positive Results in Phase III Trial with IPX056

-Provides Update on Brand Pharmaceutical Program-

-Company Presenting Today at the Bank of America Specialty Pharmaceutical Conference-

HAYWARD, Calif.--(BUSINESS WIRE)--IMPAX Laboratories, Inc. today announced that IPX056, an investigational extended-release formulation of baclofen, has met its clinical endpoints in a Phase III study of spasticity in multiple sclerosis patients. The Company also is providing an update on its brand pharmaceutical product program, which resides in its IMPAX Pharmaceuticals Division.

In a 173-patient, placebo and active comparator-controlled double blind Phase III study with a seven-week open label follow on, IPX056 was shown to be effective versus placebo in reducing spasticity in multiple sclerosis patients. IPX056 is an extended-release formulation of baclofen, the drug of choice in the treatment of spasticity, which has the potential to offer improved control of symptoms and dosing convenience.

“We are very excited with the results of this study, and are planning to meet with the U.S. Food and Drug Administration (FDA) in the fourth quarter of this year to discuss the results of this study and determine the next steps in the submission of a new drug application (NDA) for IPX056,” said Larry Hsu, Ph.D., president and chief executive officer of IMPAX Laboratories. “Such spasticity treatments represent a $1.6 billion market in the U.S. and IPX056 may fill an unmet medical need in these patients.”

The IMPAX Pharmaceuticals division of IMPAX Laboratories currently has two products in its development pipeline, directed to neurology as a therapeutic focus, with four other central nervous system (CNS) specific products undergoing feasibility assessment. The Company filed an Investigational New Drug application for IPX066, a controlled-release formulation of Carbidopa/Levodopa in July 2008, and expects to initiate studies in Parkinson’s disease patients by the end of this year and is targeting an NDA submission in mid-2011.

Michael Nestor, divisional president of IMPAX Pharmaceuticals said, “We are very pleased with the progress we have made in advancing our proprietary, brand products and expect to continue this progress with an expansion in our R&D staff this year. The CNS space is ideally suited to our core competency in drug delivery, as so many products are candidates for improved dosing and administration and the market is large and growing at almost 10% annually, faster than the total U.S. pharmaceutical market. In addition, the neurology market is readily addressable by a small and focused sales force, given the concentration of physicians writing prescriptions. We currently have 66 specialty sales representatives to market our products, and to serve as a contract force for others,” Mr. Nestor added.

As previously announced on July 31, 2008, Arthur A. Koch, Jr., senior vice president and chief financial officer of IMPAX Laboratories, will present at the Bank of America 2008 Specialty Pharmaceuticals Conference today at 9:30 a.m. Eastern time. The conference will be held at the Southampton Inn, Long Island, New York.

Individuals may listen to the live or an archived presentation made at the conference, which will be posted in the investor relations section of the Company’s web site at www.impaxlabs.com. To listen to the live presentation, please go to the web site 15 minutes prior to its start to register, download, and install the necessary audio software. This presentation will be archived on the Company’s web site for 90 days. The Company’s regular Corporate presentation will be updated and posted on the Company’s web site today as well.

About IMPAX Laboratories, Inc.

IMPAX Laboratories, Inc. is a technology based specialty pharmaceutical company applying its formulation expertise and drug delivery technology to the development of controlled-release and specialty generics in addition to the development of brand products. IMPAX markets its generic products through its Global Pharmaceuticals division and will market its brand products through the IMPAX Pharmaceuticals division. Additionally, where strategically appropriate, IMPAX has developed marketing partnerships to fully leverage its technology platform. IMPAX Laboratories is headquartered in Hayward, California, and has a full range of capabilities in its Hayward and Philadelphia facilities. For more information, please visit the Company's Web site at: www.impaxlabs.com.

IMPAX Laboratories, Inc.
Larry Hsu, Ph.D. President & CEO
510-476-2000, Ext. 1111
or
Arthur A. Koch, Jr., Sr. VP & CFO
215-933-0351
or
Mark Donohue, Sr. Director IR
215-933-3526
www.impaxlabs.com
or
Investor Relations Contacts:
Lippert/Heilshorn & Associates, Inc.
Kim Sutton Golodetz
kgolodetz@lhai.com
212-838-3777
or
Bruce Voss
bvoss@lhai.com
310-691-7100
www.lhai.com

Expert Opinion Paper - National MS Society Makes Recommendations Regarding Therapeutic Use Of Cannabis

Washington, DC: Cannabis has the potential to treat symptoms of multiple sclerosis as well as limit the progression of the disease, according to an expert opinion paper published by the US National Multiple Sclerosis Society. However, the Society stopped short of recommending that MS patients use the drug medicinally.

“Although it is clear that cannabinoids have potential both for the management of MS symptoms such as pain and spasticity, as well as for neuroprotection, the Society cannot at this time recommend that medical marijuana be made widely available to people with MS for symptom management,” the Society concludes. “This situation might change, should better data become available that clearly demonstrate benefit.”

The Society recommends that future clinical trials focus on methods of cannabinoid administration that deliver the drug to the bloodstream rapidly, such as vaporization.

The Society also recommends clinical trials be performed to investigate and quantify cannabis’ potential to slow disease progression, citing “anecdotal reports from patients … that cannabis reduces the frequency of their MS attacks.”

Investigators at Plymouth’s Peninsula Medical School in Britain recently announced that they had recruited nearly 500 MS patients for a three-year clinical trial assessing whether the use of oral THC can significantly slow the onset of multiple sclerosis.

Clinical data reported in 2006 from an extended open-label study of 167 multiple sclerosis patients found that the use of whole plant cannabinoid extracts relieved symptoms of pain, spasticity, and bladder incontinence for an extended period of treatment (mean duration of study participants was 434 days) without requiring subjects to increase their dose.

Results from a separate two-year open label extension trial in 2007 also reported that the administration of cannabis extracts was associated with long-term reductions in neuropathic pain in select MS patients. On average, patients in the study required fewer daily doses of the drug and reported lower median pain scores the longer they took it.

Commenting on the MS Society report, NORML Deputy Director Paul Armentano said: “The MS Society’s recommendations are a positive step, but they don’t go far enough. Surveys indicate that as many as one out of two MS patients use cannabis therapeutically, yet this report does nothing to challenge these patients legal status as criminals.”



For more information, please contact Paul Armentano, NORML Deputy Director, at: paul@norml.orgThis e-mail address is being protected from spam bots, you need JavaScript enabled to view it

Full text of the MS Society paper, “Recommendations Regarding the Use of Cannabis in Multiple Sclerosis”. Additional information on cannabinoids and multiple sclerosis is available from NORML. The paper can also be downloaded from the NMSS web site in PDF format by clicking on the title of this posting.

http://norml.org/

CARIS REITERATES SELL ON ELAN

On July 31, Biogen (BIIB) and Elan (ELN) reported two new cases of brain disease in multiple sclerosis [MS] patients treated with Tysabri. Caris analyst David Moskowitz says despite a number of PML reports in FDA's Adverse Events database, Elan continued to posture that these incidences were of no concern. In fact, he says the company's recent strategy has been to push drug aggressively.

Moskowitz says today's cases should not be a surprise. He believes that if the drug is not withdrawn from market, sales growth is likely to be severely hampered, given this news and the likelihood that more cases will emerge.

He cuts in-market sales estimates for the product to $781 million from $876 milion in 2009 and to $934 million from $1.39 billion in 2010. With his peak sales estimate for Tysabri declining from $3.6 billion to $1.34 billion, he lowers price target on shares from $15 to $9.

Tysabri Product Use Disclaimer

EDITORIAL COMMENTARY

August 1, 2008

As many of you know, and believe me, I am not patting myself on the back, I was an early opponent of Tysabri and was early in predicting the removal of Tysabri from the market in 2005.

As many of you also know, I was vehemently opposed to the FDA's decision to approve Tysabri, for re-introduction in the U.S., even with the so-called "black box label." The fact that European drug agencies had approved the drug for the treatment of multiple sclerosis (MS) did not provide me any comfort.

Please, please, take note of the following press release from Biogen-Idec and Elan, manufactures/marketers of Tysabri:

http://www.bloomberg.com/apps/news?pid=conewsstory&refer=conews&tkr=BIIB:US&sid=a40u7vb1OLaw

In short, progressive multifocal leukoencephalopathy has now been found among a small number - very small at this point - of patients taking the drug for MS. Many prominent neurologists - many whom I deeply respect - have been supporters of this "treatment." Again, despite this, I have remained adamantly opposed to the use of this drug.

Why? I am sorry. But the risk of developing a deadly brain virus, even if the manufacturers quantify the risk at very low percentages, just seems a bit too HIGH for me relative to a) the fact that MS, itself, is not deadly, and b) there are other options. AND, while those options are not yet perfect, there are several new drugs in late stage clinical trials, including oral medications, that so far are proving far more effective in treating MS.

Biogen-Idec and Elan state that they have no intention of voluntarily pulling Tysabri from the market - once again.

As the news report from Bloomberg, a major business news sources, reports, the companies still plan to have 100,000 people on Tysabri by 2010. However, as of today, the stock market has made the assumption, by dropping the prices of the stocks, that the market for Tysabri will soon evaporate. As, in my humble opinion, it should.

For far too long, people with MS and others diseases, have been used as a guinea pig population, with the FDA permitting companies to rush new compounds to market. There is a trade-off here. I understand that. People want treatments. They want effective treatments. Many people that suffer from diseases such as MS want new treatments because they feel increasingly feel frustrated - and rightly so (being one of them) - with the persistent march of their illnesses and the negative impacts on the quality of their lives.

I understand all of that. I also understand that two pharmaceutical companies, Merck and Pfizer, that have performed clinical trials on very inexpensive drugs - statins - for the potential treatment of MS, seem to have no interest in pursuing the MS market with those soon-to-be off patent protection Pfizer's Lipitor), or already off patent protection (in the case of Merck's Zocor/simvastatin) drugs. After all, the MS market is far more lucrative if you can offer a new drug for $2,000 - $4,000 monthly instead of $23 - $100 a month. Keep in mind, in the U.S., there are only about 400,000 known cases of MS. So it's not a huge market.

I hope that you can take time to pass this Tysabri news on to people that may be interested.

Let me see. The possibility of death vs. living with MS. For me, however small the risk of progressive multifocal leukoencephalopathy, I will happily live with MS.

Sincerely,

Cary J. Polevoy

Multiple Sclerosis: New MRI Contrast Medium Enables Early Diagnosis In Animal Model

ScienceDaily (Aug. 1, 2008) — In an animal model of multiple sclerosis (MS), neuroradiologists and neurologists of the University hospitals of Heidelberg and Würzburg have been able to visualize inflammatory tissue damage, most of which had remained unrecognized up to now, with the aid of a new contrast medium, Gadofluorine M, in magnetic resonance imaging.

In particular at the early stage of the disease, drug treatment is effective. Up to now, how-ever, an early diagnosis is frequently not established with certainty, especially if no (or very few) inflammatory lesions are present on MRI. "With this new contrast medium, we were able to visualize five to ten times more foci of inflammation in comparison to conventional MRI images and contrast media", reports Professor Dr. Martin Bendszus, Medical Director of the Department of Neuroradiology at the University hospital of Heidelberg.

Previously unrecognized patches of demyelination visible in MRI

MS is a chronic inflammatory disease of the central nervous system of unknown cause. It usually begins in young adults, and women are affected more frequently. In Germany, ap-proximately 120,000 patients are afflicted. MS is characterized by multiple inflammatory le-sions in which nerve fibers lose their myelin sheath. These patches of demyelination cause neurological malfunctions that may regress upon remyelination. At later stages, MS may re-sult in a loss of nerve fibers, leading to irreversible damage and persistent neurological symptoms. MRI plays a crucial role in the early diagnosis of MS and monitoring of the dis-ease.

The scientists from Heidelberg and Würzburg examined brains and spinal cords of animals at different stages of the disease with the new contrast medium and found significantly more inflammatory lesions than with conventional contrast media. Examinations of tissue sections from these lesions showed that these were actually foci of inflammation. The application of this new contrast medium was clearly superior to conventional contrast media, especially for the spinal cord or optical nerve, nerve regions that are particularly difficult to examine on MRI.

New contrast medium accumulates better in MS lesions

The results of the study could help dramatically improve the diagnostic work-up in MS with a potential impact on early treatment. "MS is the most frequent cause of occupational disability and handicap in young adults", explains Professor Bendszus. "New therapies have a positive influence on the course of the disease, but are often not initiated at early stages since the diagnosis of MS is not yet established. "

The new contrast medium gadofluorine M supposedly visualizes MS lesions better because it binds especially well to certain components of the extracellular matrix of inflammatory foci. Because of this, it accumulates in these lesions in higher concentrations.

Now, the next objective of the interdisciplinary working group is to further develop the new MRI contrast medium for application in clinical practice. As of now, the contrast medium is not yet approved. Additional preclinical tests are necessary for the planned clinical application.

Journal reference:

1. Bendszus et al. Gadofluorine M enhancement allows more sensitive detection of inflammatory CNS lesions than T2-w imaging: a quantitative MRI study. Brain, 2008; DOI: 10.1093/brain/awn156

Adapted from materials provided by University Hospital Heidelberg, via EurekAlert!, a service of AAAS.