Families of Spinal Muscular Atrophy Evaluating Six New Drug Discovery Programs for New Funding in 2011

Online, December 29, 2010 (Newswire.com) - An update on the new Translational Drug Discovery Program at FSMA.

In response to our October Request for Proposals (RFP) for new drug programs to develop therapies for Spinal Muscular Atrophy we received nine Letters of Intent for potential programs. Our Translational Advisory Committee (TAC) reviewed and then selected six of these for further consideration for funding. Each of these six applicants will now submit a full detailed proposal, which will be due in February 2011.

FSMA is aiming to fund two new preclinical drug development programs in 2011 from these six proposals, with more to follow. These multi-million dollar collaborations will focus on innovative methods of developing novel therapies for SMA, including both biologic and small molecule approaches. These programs will be multi-year in scope with a typical duration of three years.

The requested proposals will be reviewed by the FSMA Translational Advisory Committee (TAC) next spring. Our TAC is made up of experts from multiple facets of drug development. The use of TAC to select drug programs for funding fits perfectly with the overall FSMA research-funding model, which is based on the need for expert and independent prioritization and oversight of research programs. This approach ensures that FSMA funds only the most promising research, and that funded programs are run in a professional and efficient manner under the guidance of world-class experts.

This program follows a long legacy for Families of SMA in enabling drug development programs for SMA. FSMA has been investing in and advancing pre-clinical drug research since 2000, with a total investment of $17 million in this area.

As demonstrated by our past funding of preclinical drug programs, one of the long term goals at FSMA is to fund and de-risk early stage drug discovery programs for SMA. At the very earliest stages of drug development programs have less than a 1% chance of FDA approval. This inherent risk along with low potential for profit because of the small patient population has traditionally hindered industry from working on orphan diseases.

The follow-on investments made by the government, industry, and other groups in FSMA programs help demonstrate the value and success of the FSMA model and its scientific expertise. 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.

Our overall goal in this area to build a deep and diverse therapeutic pipeline to maximize our chances for success in finding a treatment for SMA. 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.