Repligen Licenses Potential Treatment for Spinal Muscular Atrophy

Friday, October 23, 2009 General News
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WALTHAM, Mass., Oct. 22 Repligen Corporation (Nasdaq: RGEN) announced today that it has entered into an exclusive license agreement with Families of Spinal Muscular Atrophy (FSMA) for intellectual property covering compounds which may have utility in treating Spinal Muscular Atrophy (SMA). SMA is an inherited neurodegenerative disease in which a defect in the SMN1 ("survival motor neuron") gene results in low levels of the protein SMN and leads to progressive damage to motor neurons, loss of muscle function and, in many patients, early death. The licensed compounds increase the production of SMN in cells derived from patients. Further testing of these compounds in two transgenic mouse models of SMA demonstrated significantly increased survival suggesting potential clinical utility. There is currently no treatment or cure for SMA.

"Families of SMA has made remarkable progress in defining a series of highly potent compounds which may be clinical candidates for SMA," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation. "We look forward to working with FSMA and their collaborators in the development of what we hope will be an important new treatment for SMA."

"At this point in the program, joining forces with a corporate partner to advance into clinical studies is the best way to meet our objective of accelerating drug development for SMA," stated Kenneth Hobby, President of Families of SMA. "We view Repligen as an ideal partner for this program with the necessary resources and expertise to invest in and focus on successfully developing an effective treatment for our patients."

Patients lacking a functional SMN1 gene survive only because humans carry a second gene, known as SMN2, which produces an identical protein but at much lower levels. Genetic analysis of SMA patients has revealed a striking correlation between disease severity and the number of copies of SMN2 carried by the patient. Patients with 2 copies of the SMN2 gene are usually unable to ever sit without assistance while patients with 4 copies develop few symptoms before adulthood. Thus, a doubling of the SMN2 gene copy number dramatically alters disease course and suggests that a therapy which increases the level of SMN protein in motor neurons may provide a significant clinical benefit. Symptoms of SMA typically emerge before the age of 2 and often progress to severe physical disability or loss of life. SMA is diagnosed in approximately one in every 6,000 births in the United States and Europe where the estimated prevalence is approximately 20,000 patients.

Conference Call Friday, October 23, 2009

Repligen will host a conference call and webcast on Friday, October 23, 2009 at 8:30 a.m. EDT to provide an update on this corporate development. Walter Herlihy, President and CEO of Repligen will be joined on the call by Kenneth Hobby, President of FSMA, Dr. Jill Jarecki, Research Director of FSMA and Dr. Kathryn Swoboda, University of Utah School of Medicine who cares for SMA patients. This call may be accessed via Repligen's website at or by calling (800) 901-5259 for domestic calls and (617) 786-4514 for international calls. Participants must provide the following passcode: 80325454.

About Families of SMA

Families of Spinal Muscular Atrophy is a non-profit organization dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating health professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. FSMA's vision is a world where Spinal Muscular Atrophy is treatable and curable.

About Repligen Corporation

Repligen Corporation is a biopharmaceutical company focused on the development of novel therapeutics for diseases that affect the central nervous system. In addition, we are the world's leading supplier of recombinant Protein A, the sales of which partially fund the advancement of our development pipeline while supporting our financial stability. Repligen's corporate headquarters are located at 41 Seyon Street, Building #1, Suite 100, Waltham, MA 02453. Additional information may be requested from

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 such as the anticipated growth in the monoclonal antibody market and our other target markets and projected growth in product sales, 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 current and future collaborative relationships, the market acceptance of our products, 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 product revenues and profits, our ability to generate future revenues, our ability to raise additional capital to continue our drug 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 sales and 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 Repligen's filings with the Securities and Exchange Commission. Repligen assumes no obligation to update any forward-looking information contained in this press release or with respect to the announcements described herein.

SOURCE Repligen Corporation

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