2013 Update from Families of Spinal Muscular Atrophy on the SMA Drug Pipeline

Elk Grove Village, IL, May 02, 2013 --(PR.com)-- 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 basic research to identify therapeutic approaches and then applied drug development research to make drugs. FSMA has funded almost two thirds of all the ongoing drug programs for SMA. Clear evidence of progress is being seen:

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. Two more are expected to file Investigational New Drug (IND) applications to the FDA to begin Phase I trials over the next year.

2) There is broad diversity of approaches in development. Diversity is important in case a particular approach turns out not to have benefit in patients. Currently there are six drug strategies being advanced for SMA: a) Small molecule SMN enhancers, b) Neuroprotectants, c) ASOs to correct SMN2 splicing, d) Stem cell therapy, e) Gene therapy, and f) Regulators of muscle function.

3) The total number of programs is growing. There are now 15 novel SMA therapeutic programs in various stages of preclinical and clinical development, up from just 1 a decade ago. Three programs newly funded by FSMA have been added to the pipeline in just the past few months. This volume is needed to balance the low probability of programs successfully advancing to final FDA approval.

4) Funding and resources from government and industry are increasing. There are now 11 companies actively investing in the SMA drug pipeline. Industry expertise and resources are essential to advance programs through the expensive and complicated clinical stages of development.

Clinical Programs
The SMA community has seen success over recent years in advancing basic research discoveries from the bench to the clinic. There are now 3 novel programs testing drugs in human clinical trials for SMA:

-Trophos with Olesxime currently in Phase II/III clinical trials.
-Isis Pharmaceuticals and Biogen Idec with Antisense in Phase II clinical trials.
-Pfizer with Quinazoline currently in Phase I clinical trials.

Increased Funding to Build the SMA Drug Pipeline
Families of SMA has funded nearly two thirds of all the ongoing drug programs for SMA. In the past 18 months, FSMA has funded five additional new drug programs, including the following in just the past few months: assessing second-generation ASO sequences for SMA; using a patient derived motor neuron screen to identify novel SMA Therapeutics; and testing in SMA mice a drug that enhances muscle function now in Phase II trials for ALS. Families of SMA is attacking the disease from every angle possible, and there are now more new drug programs in development than at any other time.

Failure is a Normal Part of the Drug Development Process
While there are currently 15 active new drug programs in development, over the previous decade six additional programs have failed, giving us a total cumulative SMA drug pipeline of 21. It is this overall volume and breadth that will lead to the ultimate desired goal of approved treatments and a cure 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. The FSMA strategy for drug discovery is to provide early seed funding for new projects with the goal of leveraging bigger funding from government and industry. FSMA is proud to have led the way in collaborations for many of the current programs in the SMA drug pipeline.