Sarepta hits high on Phase I/IIa DMD readout

Sarepta Therapeutics Inc. (NASDAQ:SRPT) hit an all-time high Tuesday morning, adding $71.26 (68%) to $176.50 and pushing the company's market cap over $11 billion, after reporting preliminary data from a Phase I/IIa trial evaluating AAVrh74.MHCK7.micro-Dystrophin to treat Duchenne muscular dystrophy. The stock settled during afternoon trading and ended the day up $38.69 (37%) to $143.93.

In three DMD patients in the open-label, U.S. trial, the microdystrophin gene therapy led to mean sarcolemma-localized transduced microdystrophin expression, as measured by the percentage of microdystrophin-positive fibers, of 76.2% and a mean fiber intensity of 74.5% compared with normal controls. In posttreatment biopsies, Sarepta reported mean microdystrophin levels as measured by Western blot of 38.2% of normal, or 53.7% of normal adjusted for fat and fibrotic tissue. The gene therapy also reduced mean serum creatinine kinase levels by over 87% at day 60...