Thursday, August 14, 2008


- 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

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

Tony Hesby
Executive VP Corporate Affairs
Phone: 780-413-7152

Amanda Stadel
Investor Relations Manager
Phone: 780-413-7152

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

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

-- 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

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

SOURCE Prime Therapeutics LLC

Copyright © 2008 PR Newswire. All rights reserved.

Tuesday, August 12, 2008

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

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:

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, the content of which is not part of this press release.


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

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 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:

IMPAX Laboratories, Inc.
Larry Hsu, Ph.D. President & CEO
510-476-2000, Ext. 1111
Arthur A. Koch, Jr., Sr. VP & CFO
Mark Donohue, Sr. Director IR
Investor Relations Contacts:
Lippert/Heilshorn & Associates, Inc.
Kim Sutton Golodetz
Bruce Voss

Tuesday, August 05, 2008

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.

Monday, August 04, 2008


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.

Saturday, August 02, 2008

Tysabri Product Use Disclaimer


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:

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.


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.

Biogen, Elan stand by MS drug after illnesses

The Associated Press
Published: August 1, 2008

NEW YORK: Biogen Idec Inc. and Elan Corp. defended their multiple sclerosis drug Tysabri on Friday after reporting two new cases of a potentially fatal side effect, saying the treatment is still worth the risk to patients.

There are no plans to pull the drug off the market, and the company said its current monitoring program for cases of a rare but sometimes fatal brain disorder is adequate.

The announcement of two new cases of the rare viral infection called progressive multifocal leukoencephalopathy, or PML, sent shares for both companies plummeting and reignited already simmering concerns about the drug's sales potential. It was pulled from the market in 2005 after being linked to the rare brain disease but was reintroduced under restricted sales conditions in mid-2006.

"These cases underscore the importance of continued clinical vigilance so PML can be discovered and managed appropriately," said Dr. Cecil B. Pickett, president of research and development at Biogen.

He and several other executives tried to allay concerns over additional cases during a conference call with financial investors and analysts Friday morning. The company said it would also hold calls with several physician and patient groups.

The risks associated with the drug are clearly labeled, the company said, and all patients are not only notified but have to sign a waiver acknowledging the risks. PML almost always occurs in people with a severe immune deficiency, as is the case with most patients taking Tysabri.

Multiple sclerosis is an autoimmune disorder that results in physical and neurological damage.

There are about 32,000 patients on Tysabri worldwide, with 17,800 in the U.S., the company said. It would not comment on its financial outlook, but did say future cases of Tysabri were anticipated and factored into the goal of eventually getting the drug to 100,000 patients worldwide.

The companies maintain the drug still has a favorable risk-reward profile, meaning it is more helpful than hurtful, and monitoring programs are set up in the U.S. and Europe to catch any potential cases of PML early. Those systems rely on physicians to test for the condition if they start to see symptoms. Biogen and Elan would not comment the number of suspected cases, only confirmed cases.

Both new cases came out of European, with the first patient having been on Tysabri for 17 months. Another, who is still hospitalized, had been on Tysabri for 14 months. Still, the company said it can count about 6,600 patients on the drug for about 18 months now, reinforcing its stance that the treatment is worth the risk.

Food and Drug Administration officials could not immediately be reached for comment as of Friday morning.

The broader market, meanwhile, remains concerned over the impact on sales, which nearly tripled to $200 million during the second-quarter. The treatments costs about $30,000 per year.

"We do not believe that Tysabri will be withdrawn, based on this data, but it is likely to spook investors as the true rate of PML in Tysabri monotherapy is unknown," Goldman Sachs analyst Stephen McGarry said in a note to Elan investors.

Shares of Cambridge, Massachusetts-based Biogen fell to $50.28, their lowest point in more than a year, while shares of Ireland-based Elan fell to $10.67, their lowest point in nearly three years.

Adult Stem Cells Reprogrammed To Become Myelin-Making Cells

Research published in Nature Neuroscience , electronic publication ahead of print) has shown that adult stem cells in mice that are developing into nerve cells can be redirected to turn into myelin-making cells by changing a single gene . This type of research may some day help repair the damage to myelin which occurs in multiple sclerosis (MS).

In people with MS the immune system can attack both myelin and myelin making cells (oligodendrocytes). Limiting the number of myelin making cells impairs the capacity to repair the damage to myelin. One potential treatment option currently being investigated involves encouraging immature stem cells that reside in the adult brain, called neural stem cells, to move to areas of damage and repair myelin.

When neural stem cells are grown in the laboratory scientists have been able to reprogramme them to develop into several different types of brain cells, including oligodendrocytes. This latest research which took place in The Salk Institute for Biological Studies in California sought to determine if it would be possible to repeat these experiments in the brain.

A gene called Asc1 which is associated with oligodendrocyte development was introduced into the stem cells in the brain and caused neural stem cells to develop into oligodendrocytes.

This study confirms that adult stem cells in the brain retain their ability to be converted to certain other types of brain cells. Further research is needed to determine the significance of these finding to myelin repair in people with MS.

Dr Laura Bell at the MS Society said: 'Finding a way to cause stem cells which are already present in the brain to repair damaged myelin is an attractive potential treatment option for people with MS. This is early research but it is an important step and we look forward to seeing how the work progresses.'

Biogen, Elan Report Brain Infection in Tysabri Users

By Tom Randall

July 31 (Bloomberg) -- Biogen Idec Inc. and Elan Corp. reported two confirmed cases of a deadly brain infection in patients taking the multiple sclerosis drug Tysabri, the first since the drug was reintroduced in the U.S. in 2006.

The report sent shares of both companies tumbling. The two patients were in the European Union, Biogen said today in a regulatory filing. The cases of the disease, progressive multifocal leukoencephalopathy, were confirmed this week, according to the company's statement.

Biogen and Elan voluntarily pulled the drug from the market in February 2005 after three patients unexpectedly contracted the disease, which caused two deaths. Tysabri was reintroduced in July 2006 in the U.S. and sold for the first time in the EU at the same time. One patient had been taking the drug for 17 months and the other took it for 14 months, Biogen said.

``This is going to be fairly disruptive to sales in the United States,'' said Mark J. Schoenebaum, an analyst with Deutsche Bank Securities in New York, by telephone. ``There is a real fear of the unknown here, and I think some patients and physicians are going to take a drug holiday'' until the company discloses more details.

More than 31,800 multiple sclerosis patients use Tysabri, Biogen spokeswoman Naomi Aoki said. About 14,000 patients have taken Tysabri for more than a year, and 6,500 patients have taken it for 18 months or longer.

Anticipated Cases

``We've said ever since the reintroduction that we anticipate seeing additional cases,'' Aoki said today in a telephone interview. ``Withdrawing the drug is not under consideration.''

The condition was included in the prescribing information on the drug as a possible side effect in 1 of every 1,000 patients taking the drug, Aoki said. One patient is clinically stable and is at home, Biogen said. The other patient is hospitalized.

The shares of Biogen, which is based in Cambridge, Massachusetts, fell by $14.06, or 20 percent, to $55.60 at 7:20 p.m. New York time, after the close of regular trading on the Nasdaq Stock Market. American depositary receipts of Elan, which is based in Dublin, fell by $7.98, or 39 percent, to $12.07. Each receipt equals one ordinary share.

``We expect the stock to open sharply down on Friday, but would not be buyers as we think investor sentiment could continue to weaken,'' Schoenebaum said in a note.

Goal of 100,000 Patients

On July 22, Biogen reported second-quarter net income rose 11 percent to $207 million on sales of Tysabri, its fastest- growing treatment. Biogen has estimated 100,000 patients will be taking Tysabri by 2010, a goal critical to the company's future growth, Thomas Weisel analyst Ian Somaiya said in a note to clients at the time.

Tysabri was approved in the U.S. in January to treat Crohn's disease, an inflammatory intestine and bowel disorder.

Shares of Elan, Ireland's biggest drugmaker, already were battered this week after Wyeth and Elan's experimental treatment for Alzheimer's disease, bapineuzumab, was linked in a study to a brain-swelling side effect.

To contact the reporters on this story: Tom Randall in New York at
Last Updated: July 31, 2008 19:29 EDT

Putting Drug Development In Patients' Hands

An Entrepreneur Stricken With Cancer Sets Up Firm To Develop 'Virtual' Biotechs
July 29, 2008

Jay M. Tenenbaum became a multimillionaire in the Internet boom of the late 1990s. But it wasn't until he was diagnosed with a lethal cancer that he found his calling as an Internet entrepreneur.

Partnering for a Cure: Patients with less common diseases often find that they need to get involved with the effort to find new therapies. WSJ's Amy Dockser Marcus talks to CollabRx founder Marty Tenenbaum and COO Jonathan Jacoby about the challenges patients face.

Dr. Tenenbaum learned in 1998 that he had melanoma, the most serious kind of skin cancer. He underwent surgery and took an experimental vaccine for a year. Then, nearly five years ago, the cancer returned, having spread to his liver. "That's when I started looking at my mortality seriously," says the 65-year-old from Portola Valley, Calif.

Frustrated with his treatment options, Dr. Tenenbaum began investigating other potential therapies. He found dozens of patient-advocacy organizations dedicated to melanoma that raised money and supported scientific research. They "all had good ideas," he says, "but no one had put the different pieces together in the right way that would let them make progress in finding a drug in the lifetime of a patient."

So he tapped his own Internet savvy -- and his connections -- to create a company aimed at helping patients develop new therapies faster and cheaper for less common diseases, like melanoma, that often don't attract major pharmaceutical company research funding. He set up his new company, called CollabRx, with $2 million he had available and is trying to raise $3 million more from family, friends and private investors.

[Jay Tenenbaum]
Thor Swift for The Wall Street Journal

Jay Tenenbaum started CollabRx to help patients hasten drug development.

Dr. Tenenbaum's idea taps into the recent phenomenon of patient-supported research -- a trend largely driven by people wealthy enough to help fund drug-discovery projects and who are affected by rare or overlooked diseases.

The Myelin Repair Foundation, founded by a patient with multiple sclerosis, set up a team of researchers to come up with promising drug leads. After spending $13 million over four years, the foundation will present its two best prospects to drug companies in coming months in the hopes of getting one of them to develop the drugs further. And the Cystic Fibrosis Foundation, started by a group of parents who had children with the disease, recently announced that a drug it is paying a biotech company to develop showed promising results in an ongoing trial. The foundation says its investment in the drug so far exceeds $75 million.

CollabRx aims to expand patient-funded research further by connecting individuals or small numbers of patients with the tools and services they need. Each CollabRx client is assigned a project manager, a specialist who works with patients to devise a research strategy, interpret the results and later steer any promising prospects toward development of possible treatments.

CollabRx calls such integrated projects virtual biotechs because they aim to replicate many of the steps typically taken as part of a pharmaceutical or biotech company's search for a new drug. As the number of private labs available to do sophisticated research grows, many parts of the drug-development process can now be contracted separately. Researchers in various locations can share information and material by means of a Web-based network created by CollabRx software engineers.

Management Fees

CollabRx's fees vary depending on the scope of a project. But Dr. Tenenbaum says he expects to charge most clients a management fee of 10% of a project's budget and to receive a 20% share of any intellectual property that emerges. Drug screens, clinical trials, and the costs of other contracted services are paid for by the clients.

Dr. Tenenbaum says patients can get started on a project with as little as $50,000 to $100,000. Sums like that, for example, could fund the creation of a molecular profile of a tumor to try to predict what combination of already approved drugs might be effective. If results proved promising, more money could be raised to set up a full-blown virtual biotech -- with a budget in the millions of dollars -- that might test cocktails of therapies in animal models and try grouping patients into subtypes to better tailor treatments for them, among other projects.

Bonnie J. Addario, a former oil-company executive in San Francisco, is a lung-cancer survivor. When she first started thinking about how to make a difference, she figured, "I'll run a gala and a golf tournament, raise money for research, and that will be it." Mrs. Addario, 60, raised $800,000 through a foundation she set up in 2006. She distributed the money to a number of researchers, and then realized, "there are a lot of wonderful people doing great work, but lung-cancer survival rates [of 15.5% after five years] haven't changed for 40 years. Why is that?"

To find answers, Mrs. Addario and her husband, along with David M. Jablons, her surgeon from the University of California, San Francisco, put together a two-day conference last fall of lung-cancer researchers from major institutions around the world. She says the group identified a number of problems that hinder progress toward a cure. Among them: Researchers didn't know what others were doing, tissue and blood specimens needed for experiments weren't centrally located or shared, and the findings of experiments weren't integrated to help assess what the key priorities should be.

Mrs. Addario started a new organization, the Addario Lung Cancer Medical Institute, and hired CollabRx to address some of these issues. The company is helping the institute build a virtual specimen bank where researchers participating in the project can share patient specimens and establish joint standards for collecting future specimens. Using the CollabRx Web-based network, the researchers can share research and ideas, and quickly reprioritize projects as new information comes up. Mrs. Addario says the institute expects to spend at least $5 million over the next year to set up the virtual biotech, fund researchers and establish the specimen bank.

It's far too early to tell whether CollabRx's approach will be more successful at finding new drugs cheaper or faster than the traditional methods of drug and biotech companies, a challenge acknowledged by scientists who target faster cures. "We're not discovering drugs slowly just because of a faulty business model," says John Wilbanks, executive director of Science Commons, a nonprofit in Cambridge, Mass., that seeks to make research more effective by encouraging more open access to scientific data. "We don't understand so many things about toxicity in the human body. It's hard because it's hard," says Mr. Wilbanks, who works with Dr. Tenenbaum on a separate project. On average, the pharmaceutical industry spends about $1 billion over 17 years to bring a new drug to market.

CollabRx's approach in some respects also goes against the cultural grain of scientific research. In order for academic researchers to get ahead at universities, they must be first to publish breakthrough findings. This competitive culture deters scientists from sharing information, and could make it difficult for CollabRx to get researchers to work together.

The Internet culture that drives CollabRx encourages collaboration, says Christopher P. Austin, director of the Chemical Genomics Center of the National Institutes of Health. "And unlike the Internet, where people were maniacal about sharing stuff, biomedical scientists are exactly the opposite," he says. "You have to drag their data from their cold dead fingers. They do not share." Dr. Austin is testing CollabRx's network with one of his lab's projects that seeks to develop therapies for a rare disease.

Internet Background

Dr. Tenenbaum, who has a Ph.D. from Stanford University in electrical engineering and computer science, was deeply involved in the Internet boom of the 1990s. He started a number of Web companies that helped pioneer the use of e-commerce. His most lucrative foray was as chief scientist and a board member of Commerce One Inc., which developed technology enabling the computer systems of big businesses to interact more readily. Dr. Tenenbaum, who goes by the nickname Marty, left Commerce One in 2002 as the company was struggling amid the tech bust early this decade.

Dr. Tenenbaum is engaged in another project with several partners that also borrows from the Internet culture -- creating a nonprofit marketplace for data, materials, resources and services needed for studying and treating disease, to be called Health Commons. The partners, which also include Science Commons, the Public Library of Science, and CommerceNet, plan to set up a site where people and companies in the life sciences can go to buy and sell goods and services.

Dr. Tenenbaum says that if Health Commons is successful, it could help CollabRx locate resources for its virtual biotechs. Even individual patients, should they want to pursue research and drug development on their own, could find information and services at the site, he says. (For a summary of the Health Commons project, go to

Chris and Hugh Hempel of Reno, Nev., last fall met with Dr. Tenenbaum a few weeks after learning that their 4-year-old twin daughters, Addi and Cassi, had been diagnosed with Niemann-Pick Type C, a genetic neurodegenerative disorder that usually is fatal by the age of 20. The Hempels joined with a group of parents to fund SOAR-NPC, a virtual biotech CollabRx set up.

The group decided that the fastest way to help the children would be to focus on a combination therapy of already approved drugs and other compounds that could prevent or significantly delay the onset and progression of NPC. Mrs. Hempel, who runs a public-relations firm, says she and her husband recently raised $500,000 at a gala event in Reno that will help fund the virtual biotech, whose total budget is $1.3 million for the first year. The project currently is testing a number of drug candidates at an NIH-run facility. Later this year, the first human observational study will start.

Melanoma Study

CollabRx also is helping to coordinate a melanoma study at the John Wayne Cancer Institute in Santa Monica, Calif., which received a grant from the Melanoma Research Alliance. The project, with a budget of $1 million over three years, is using genomic technologies to test melanoma tumors collected by the institute over the past two decades to find drugs that might work on clusters of individuals whose tumors exhibit certain similarities.

The melanoma alliance, set up with funding from Apollo Advisors founding partner Leon D. Black and his wife, Debra, and FasterCures, an organization created by financier Michael Milken, are also discussing a larger project with CollabRx to set up a melanoma virtual biotech. That project would test various melanoma cell lines against existing drugs and drug combinations with the aim of advancing promising prospects to clinical trials. Gregory C. Simon, president of FasterCures, says he sees the melanoma virtual biotech as a model that could be useful to other advocacy groups with which FasterCures works.

It is a project that Dr. Tenenbaum is following closely, not only in his role at CollabRx but also as someone who might benefit from any potential therapies that emerge. Mr. Tenebaum has had four surgeries to remove suspicious nodules since his cancer returned, and he gets scans every few months. Right now, there are no signs of the cancer. "But with melanoma," he says, "that can change overnight."

He plans to contribute funding for the melanoma virtual biotech out of his own pocket. Eventually, Dr. Tenenbaum says he plans to pay to have his own tumor cells tested with whatever drug combinations the virtual biotech finds are promising.

"The fact that there is no therapy right now that works in general for melanoma patients doesn't mean that there is no therapy that works on selected patients," Dr. Tenenbaum says. "That is where CollabRx comes in. I want to have that information in hand, just in case I ever need it."

Write to Amy Dockser Marcus at