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Families of Spinal Muscular Atrophy Announces Repligen Corporation Receives FDA Approval to Begin Phase I Clinical Trial in SMA

CHICAGO, May 19, 2011 /PRNewswire-USNewswire/ -- Families of Spinal Muscular Atrophy announced today that Repligen Corporation has received approval from the U.S. Food and Drug Administration to commence a Phase I safety study in healthy volunteers for RG3039, formerly called Quinazoline495, which is being developed for Spinal Muscular Atrophy.

FSMA began the Quinazoline program in 2000 at the very initial stages of drug development, when risk is the highest.  It was the very first industrial drug program for SMA ever conducted.  FSMA fully funded the program with investments of $13 Million.  The direction from FSMA provided the positive results necessary to license the program to Repligen Corporation and leverage larger funding for clinical development.

This clinical trial will be a double-blind, single ascending dose, Phase I study in healthy, adult volunteers to evaluate the pharmacokinetic and safety profile of RG3039 in up to 40 subjects.  The study will be the first step in the clinical development of RG3039 as a potential treatment for SMA.

"It is very exciting after 10 years of hard work by FSMA leading this program, including a significant financial investment, for the drug candidate to be licensed to Repligen in 2009 and to now be able to announce the approval to advance into human clinical trials.  This is a tremendous milestone for our community to have reached as we make progress toward a treatment for SMA," stated Jill Jarecki, Ph.D.  Research Director at FSMA.  "We are very pleased to have received approval to initiate human clinical trials with RG3039," stated Walter C. Herlihy, President and Chief Executive Officer of Repligen Corporation.  

SMA, the leading genetic killer of children under the age of two, is typically marked by the degeneration of muscle movement including the muscles that control crawling, walking, swallowing and breathing.  There are no approved therapies for the treatment of SMA to date, which affects one in every 6,000 babies. One in every 40 people carry the gene that causes SMA, indicating approximately 7.5 million carriers in the United States.

One of the goals at FSMA is to fund early stage drug discovery programs for SMA to the point where other groups will invest.  At the earliest stages of drug development, programs have less than a 1% chance of success.  This inherent risk along with low potential for profit due to a small patient population has traditionally hindered industry from working on orphan diseases.  

FSMA has actively reduced the barriers to SMA drug discovery programs by providing:  1) early seed funding, 2) access to tools and reagents, 3) expert SMA advisors, and 4) established clinical trial protocols and networks.

FSMA recently released a Request for Proposals for new preclinical drug discovery programs to build a robust drug pipeline for SMA.  Partnerships such as this one with Repligen, between non-profits, the government and companies are a very effective way to share the risks of developing rare disease treatments.  This FSMA approach also enables the correct expertise for a particular stage of development to be brought into a program.

About Families of Spinal Muscular Atrophy: Families of SMA is a non-profit 501(c)3 tax exempt organization with 30 Chapters throughout the United States and over 70,000 members and supporters. 

Families of SMA funds and directs the leading SMA research programs.  The successful results and progress from basic research to drug discovery programs to clinical trials provide real hope for families and patients.

Families of Spinal Muscular Atrophy is 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. Web site:

SOURCE Families of Spinal Muscular Atrophy
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