Positive IIb Results for Sarepta

Sarepta Therapeutics has posted data through Week 96 from Study 202, a Phase IIb open-label extension study of eteplirsen in patients with Duchenne muscular dystrophy (DMD). Results through nearly two years showed a continued stabilization of walking ability in eteplirsen-treated patients evaluable on the six-minute walk test (6MWT). Study 202 earlier met its primary endpoint of increased novel dystrophin, as assessed by muscle biopsy at Week 48, and is now in the long-term extension phase in which patients continue to be followed for safety and clinical outcomes.

Through 96 weeks, eteplirsen was well tolerated and there were no reported clinically significant treatment-related adverse events, no treatment-related serious adverse events, hospitalizations or discontinuations. Sarepta plans to file an NDA for eteplirsen in 1H14.