Update on the Spinal Muscular Atrophy Drug Development Pipeline From Families of SMA

| Source: Families of SMA

ELK GROVE VILLAGE, Ill., Jan. 19, 2012 (GLOBE NEWSWIRE) -- Developing new therapies to treat and ultimately cure SMA is the driving force behind Families of SMA. To achieve this goal, Families of SMA aggressively invests in drug development research, funding the most successful and innovative scientists and organizations.

Several key details demonstrate the great progress being made:
1) Programs are advancing into the clinical trial stage of testing.
There are now 3 novel programs actively being testing in clinical trials for SMA.
2) There is a broad diversity of approaches in development.
This breadth is important in case one particular approach to treatment turns out not to have benefit in patients.
3) The total number of programs is growing.
There are now 13 novel SMA therapeutic programs in various stages of preclinical and clinical research, up from just 2 ten years ago. This volume is necessary to balance the low odds of only 1 in 10 programs successfully advancing through clinical trials to final FDA approval.
4) Funding and resources from industry are increasing.
There are now 9 companies actively investing in the SMA drug pipeline which is essential to advance programs through the expensive and complicated later clinical stages.

Clinical Programs.
Our community has been successful over recent years in advancing basic research discoveries from the bench to the clinic, with three novel programs now testing drugs in human clinical trials. These include:
-Trophos with Olesxime currently in Phase II clinical trials.
-Repligen Corporation with Quinazoline currently in Phase I clinical trials.
-Isis Pharmaceuticals and Biogen Idec with Antisense currently in Phase I clinical trials.
The average time for a program to complete all clinical trial phases is 7 years.

New Increased Funding.
Families of SMA invests in broad research initiatives. A major goal at FSMA has been to help build the SMA drug pipeline. Even with the community's current progress, FSMA believes it is critical to continue to grow the SMA drug pipeline. Statistics show that only 10% of all drugs initiating human clinical trials ultimately receive FDA approval. FSMA has been investing in and advancing novel pre-clinical drug research since 2000. The goal for our drug discovery funding is to build a diverse therapeutic pipeline to maximize the chances for success in finding a treatment for SMA.

FSMA will announce new funding for drug discovery programs in the coming months. This new funding is significant as FSMA will now begin to fund several programs concurrently which have different approaches to developing a therapy for SMA. This will both enhance our overall chances of success and also accelerate the timeline to approved SMA drugs. With this new funding, FSMA will have been involved in funding half of all the ongoing novel drug programs for SMA.

Partnering with Companies and Government.
Drug development in SMA has seen advances because of collaborative efforts between academic, government, pharmaceutical, and non-profit organizations. These collaborations are essential for effective drug development in orphan diseases.

FSMA is proud to have led the way in collaborations for many of the current programs in the SMA drug pipeline, including:
-Licensing the Quinazoline program to Repligen Corporation. Which was the first new drug designed for SMA to receive approval to start clinical trials.
-Licensing the Antisense program to ISIS Pharmaceuticals. This program received FDA approval to being clinical trials for SMA in December 2011.
-Obtaining NINDS grant award support for Paratek Pharmaceuticals with potential funding of $5 Million.

Families of SMA is attacking the disease from every angle possible, and there are now more drug programs in development than at any other time. FSMA is the leading active funder of SMA drug development research in the world.

Additional Drug Pipeline Notes:
-The companies now involved in SMA drug programs include: Trophos, Repligen Corporation, Isis Pharmaceuticals, Biogen Idec, PTC Therapeutics, Roche, Novartis, California Stem Cell, and Paratek Pharmaceuticals.
-Four SMA drug development programs are no longer included in the pipeline due to the programs being cancelled for either scientific or financial reasons.

Please contact Kenneth Hobby at Kenneth@fsma.org or call 1-800-886-1762 if you have any questions.

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The following pictures are available for download:

[Image] 2012 FSMA Drug Pipeline