CureDuchenne - Together we Can CureDuchenne
August 2012

CureDuchenne Supported Sarepta Therapeutics Research Study for the Treatment of Duchenne Muscular Dystrophy

Max McNary, 10, participates in the Sarepta trial.
Max Leclaire, 10, participates in the
Sarepta trial.

CureDuchenne is pleased by Sarepta Therapeutics (formerly AVI BioPharma) announcement on July 24 that its exon-skipping compound, eteplirsen, achieved a significant clinical benefit in a Phase IIb trial in Duchenne patients. CureDuchenne, along with Children’s National Medical Center in Washington, DC and the Foundation to Eradicate Duchenne, provided funding in 2010 for this research to progress into human clinical trials.

This is a milestone for the company and patients alike. It demonstrated for the first time that eteplirsen achieved a highly significant clinical benefit on the 6-minute walk test, over a placebo/delayed treatment cohort in a Phase IIb trial in DMD patients. Eteplirsen was administered once weekly at 50mg/kg over 36 weeks, and patients on treatment demonstrated a 69.4 meter benefit (227 feet benefit) compared to those who received placebo/delayed treatment. The drug was well tolerated; there were no treatment-related adverse events, no serious adverse events and no discontinuations.

The next milestone is at 48 weeks, and the data is expected in October. It will include analysis of dystrophin levels from muscle biopsies in addition to the clinical outcome measures. If it goes as planned, the result will pave the way for an end of phase II meeting between the company and the FDA to discuss and agree to the plan for a phase III registration trial.

“This is a good day for Duchenne, and I’d like to thank CureDuchenne for their support and funding as well as many other organizations and institutions that provided support over the last few years,” said Chris Garabedian, CEO of Sarepta Therapeutics.

“Our family is so grateful to CureDuchenne for helping to fund this research which has given us hope for our two boys future, for the first time ever,” said Jenn McNary, whose 10-year-old son Max is one of the boys enrolled in the Sarepta trial. “It is with tears in my eyes that I wish to thank CureDuchenne, Sarepta, Nationwide Children's Hospital research team and everyone else who is so dedicated to finding a cure.”

According to Debra Miller, founder and CEO of CureDuchenne, “This is the news the Duchenne community has been waiting for; a safe and effective disease-modifying treatment is within our reach and it allows us to focus on the parallel development of next round of drugs that target the other exons. We must remain cautious since there is still a long way to prove this drug will provide significant benefits over time, many drugs fail to achieve FDA approval at this stage.”

Click here to read the Sarepta Therapeutics press release.

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