Here is STAT’s biotech scorecard, our regular ledger of stock-moving biotech events, for the first quarter:
To secure approval for its first-ever “micro-dystrophin” gene therapy for Duchenne muscular dystrophy, Sarepta Therapeutics needs to deliver a positive outcome from a randomized, placebo-controlled clinical trial — something the biotech has never accomplished before, despite already winning approval for two drugs to treat Duchenne. Sarepta is hoping to show that its one-time, potentially curative treatment called SRP-9001 can improve muscle function in patients with Duchenne. The study’s readout is expected early in the quarter, perhaps in January.
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