Sarepta Therapeutics Announces Significant Clinical Benefit With Eteplirsen After 36 Weeks in Phase IIb Study for the Treatment of Duchenne Muscular Dystrophy

Sarepta Therapeutics (formerly AVI  BioPharma) announced that its exon-skipping compound, eteplirsen, achieved a significant clinical benefit in a Phase IIb trial in Duchenne patients. CureDuchenne, along with Children 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.


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