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

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Fifteen new drugs now in development for SMA with 3 in active clinical trials and 11 companies investing in SMA drug programs.

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 applies 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 tested 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
A major goal at FSMA has been to help build the SMA drug pipeline. FSMA has been investing in and advancing novel pre-clinical drug research since 2000. The goal is to build a diverse therapeutic pipeline to maximize the chances for success in finding a treatment for SMA. 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.

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, including:

  • Licensing the Quinazoline program to Repligen Corporation, which is now being advanced by Pfizer. This was the first new drug designed for SMA to receive approval to start clinical trials.
  • Licensing the Antisense program to ISIS Pharmaceuticals. This program will initiate Phase IIB trials in 2013.
  • Obtaining a NINDS grant award for Paratek Pharmaceuticals. With potential funding of $5 Million.
  • Collaborating with NINDS and Nationwide Children’s for an award to support a CNS-directed gene therapy. With potential funding of $3.8 Million.

About Families of Spinal Muscular Atrophy
Families of Spinal Muscular Atrophy is the world’s leader focused on funding SMA research to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $55 million in research and has been involved in funding over half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 90,000 members and supporters throughout the United States. The organization’s work has produced major discoveries, including identification of the underlying cause and a back-up gene for the disease, which provides a clearly defined target for disease altering therapies. The organization is also dedicated to supporting SMA families through networking, information and services and to improving care for all SMA patients.

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