Cure SMA Announces Expanded Phase 3 of SMA Industry Collaboration

Cure SMA is pleased to announce the launch of an expanded Phase 3 of our SMA Industry Collaboration. The SMA Industry Collaboration is a multi-faceted partnership that brings together pharmaceutical companies, Cure SMA, and other nonprofit organizations, to share information, ideas, and data. The SMA Industry Collaboration works together to address scientific, clinical and regulatory topics that are critical to advancing drug development in SMA and will benefit the broader SMA community.

In this third phase, three new members—Novartis, Scholar Rock, and SMA Europe—join existing partners Astellas, AveXis, Biogen, Cytokinetics, and Genentech/Roche. With this expanded structure, Cure SMA and the SMA Industry Collaboration will be able to move forward into several new areas, including clinical trial expansion in Europe.

The pharmaceutical partners in the SMA Industry Collaboration also reflect the diversity of approaches in the SMA drug pipeline. We recently released a new version of this pipeline, showing that both the total number of programs and the number of programs in clinical trials have reached record highs. We know that it will likely take a variety of these drugs, working in combination, to provide the best possible outcome for those at all ages, types, and stages of SMA.

Through the collaboration, developers of muscle drugs will work alongside developer of drugs that target the underlying genetic cause of SMA. Each partner will bring their distinct perspective to shared goals that will benefit the entire SMA community.

Goals of Collaboration

The goals for this next phase of the SMA Industry Collaboration include:

  • Publishing the Voice of the Patient Report and results of research on the Benefit-Risk Preferences of parents/caregivers and individuals affected with SMA – Distributed to FDA in January 2018/ Manuscript submitted for publication, October 2018
  • Developing patient-reported outcome measures to ensure that SMA clinical trials are measuring the results most important to those affected by SMA – Grant Awarded to Dr. Heatwole, March 2018
  • Leveraging partnerships with organizations including the American Academy of Pediatrics, American Academy of Neurology, and the National Organization for Rare Disorders, to further reduce diagnostic delays – Ongoing
  • Developing a clinical trial readiness toolkit to increase access to trials by increasing the number of sites that are able to participate. – Completed and shared with trial sites May-Present
  • Working directly with sites that are not currently SMA clinical trials sites, to bring them online – 10 Pilot Sites were completed by year end 2018 /Ongoing 2019
  • Executing a European clinical trial capacity pilot – Completed, October 2018
  • Launching SMArt Moves, a national awareness campaign targeted to both parents and health care professionals, with the goal of reducing the length of SMA “diagnostic odyssey” – Launched November 2018
  • Publishing results of research on the on the economic burden of SMA, to increase understanding of the full impact of the disease – early 2019

Past milestones include:

  • Executing a Patient Focused Drug Development Meeting with the FDA
  • Writing and disseminating the SMA Voice of the Patient Report, based on the findings of the PFDD meeting.
  • Conducting and publishing the results of a benefit-risk survey.
  • Conducting an SMA diagnosis needs assessment for families and health care providers
  • Launching a webpage specifically created for parents who suspect their child may have SMA
  • Surveying existing and prospective SMA clinical trial sites to determine barriers to increased participation
  • Developing a clinical trial readiness checklist

Collaborative industry groups like this one are a rare opportunity to advance goals important to the SMA community. We thank our industry partners for their example of “the strong sense of collaboration that is helping to bring … therapies for SMA to patients and families,” as Dr. Jonathan Goldsmith of the FDA recently at the SMA Patient-Focused Drug Development Meeting.

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