Sarepta stumbles with Phase II setback in Duchenne muscular dystrophy

8 January 2021

Shares in rare disease specialist Sarepta Therapeutics (Nasdaq: SRPT) fell around 45% after hours on Thursday, following mixed results from a Phase II study in Duchenne muscular dystrophy (DMD).

The firm announced top-line results from Part 1 of Study 102, a trial testing the safety, efficacy and tolerability of a single dose of gene transfer therapy candidate SRP-9001.

The therapy is intended to deliver its micro-dystrophin-encoding gene to muscle tissue for the targeted production of the micro-dystrophin protein.

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