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Sarepta: Eteplirsen Shows Positive Results in Phase IIb Study of DMD Patients

Jan. 17, 2014

Cambridge, Mass.-based Sarepta Therapeutics says its drug eteplirsen has shown continued stability in six-minute walk distances in patients with Duchenne Muscular Dystrophy (DMD).

The Wednesday announcement cited results from a randomized, double-blind, placebo-controlled Phase IIb trial of 12 boys, ages seven to 13 years, with amenable genotypes. Those results showed that patients dosed with intravenous eteplirsen once a week showed 64.9 and 58.0 meters less of decline in walking ability, respectively, at 120 weeks than patients given placebo.

Additionally, eteplirsen showed good safety and tolerability levels, without a single adverse event, hospitalization or discontinuation, the drugmaker said.

The results provided Sarepta with some relief as a recent Phase III failure of GlaxoSmithKline and Prosensa’s similar drug drisapersen has raised doubts among some FDA reviewers about the clinical benefit of Sarepta’s dystrophin biomarker. The agency in November requested a discussion of different clinical endpoints, along with a new confirmatory trial. — Lena Freund

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