Sarepta rallies after presenting 9-month DMD gene therapy data

Sarepta Therapeutics presented this morning nine-month functional and creatine kinase data from baseline from the four patients in the Phase 1 open-label study of the company’s micro-dystrophin gene therapy candidate for Duchenne muscular dystrophy. The biomarkers show “large” magnitude of effect within three months for both CK and dystrophin, Sarepta said. Further, initial functional data shows “consistent and persistent” improvement from baseline to day 270, the company adds. Sarepta also sees a “favorable” safety profile to date with nine months of follow-up. The company is currently enrolling Study-102, which contains 24 patients and is placebo controlled. Shares of Sarepta are up 3%, or $3.94, to $126.20 in premarket trading.

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