Muscular Dystrophy Association Invests $1.4 Million to Help Advance the Families of SMA Quinazoline Drug Program for Spinal Muscular Atrophy at Repligen Corporation.

Dec 15, 2010, 13:13 ET from Families of Spinal Muscular Atrophy

CHICAGO, Dec. 15, 2010 /PRNewswire-USNewswire/ -- The MDA has committed to invest funds in the FSMA Quinazoline Program at Repligen Corporation to assist in advancing the program to an IND filing with the FDA.

FSMA began the Quinazoline, or DcpS inhibitor RG3039, 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 done.  FSMA fully funded the program until 2009, when our investments over of $13 Million provided the positive results to leverage larger funding amounts for clinical development from Repligen Corporation.  

Repligen Corporation is now responsible for coordinating and funding the clinical development phase of the program.   This current MDA grant is the second outside funding award obtained for this particular program.  In 2009, Christine DiDonato of Northwestern University received a NIH grant using data obtained with FSMA funding to support mouse model studies on this compound.

The follow-on investments made by the government, the company Repligen, and now the MDA in this particular program help demonstrate the value and success of the FSMA model and its scientific expertise.  

One of the goals at FSMA is to fund and de-risk early stage drug discovery programs for SMA to the point that other groups are willing to invest.  At the earliest stages of drug development programs have less than a 1% chance of FDA approval.  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 early stage 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.

In keeping with our commitment to build a robust drug pipeline for SMA, in order to mitigate the inherent risk associated with even the most promising approaches, FSMA recently released a Request for Proposals for new preclinical drug discovery programs.  

Partnerships 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 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.

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.

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SOURCE Families of Spinal Muscular Atrophy