Friday, December 12, 2008

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

* 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.

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


[1.] Novartis. Data on file.
[2.] World Health Organization. Neurology atlas, 2004. (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.

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Central media line: +41 61 324 2200

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Thursday, December 11, 2008

Elan seeks deals to raise cash

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:

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

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MS services are poor

Greg Baxter

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

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:

Monday, November 10, 2008

Multiple Sclerosis Progression Can Be Predicted With MRI

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:

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

Avigen Announces Restructuring and Strategic Direction

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."


-- 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

-- 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

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

The Avigen, Inc. logo is available at

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,

SOURCE: Avigen, Inc.

Avigen, Inc.
Michael Coffee, Chief Business Officer
1301 Harbor Bay Parkway
Alameda, CA 94502

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

[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

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.



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

"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: 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.

Friday, October 31, 2008

Interferon Could Be A Key To Preventing Or Treating Multiple Sclerosis

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

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

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

Wednesday, October 29, 2008

Novel Oral Agent for MS May Reduce Brain Lesions

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

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

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.


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

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 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 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.

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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.

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

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

The Avigen, Inc. logo is available at

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,

SOURCE: Avigen, Inc.

Avigen, Inc.
Michael Coffee, Chief Business Officer

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

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.