Sarepta shares surge 14% on positive early/mid-stage clinical trial results in DMD

Sarepta Therapeutics Inc. shares surged 14.4% in premarket trade Wednesday on positive early/mid-stage clinical trial results for its golodirsen therapy in Duchenne muscular dystrophy. In a phase 1/2 trial in Europe that enrolled 25 boys with DMD, there was a 100% response rate, "demonstrating proof of mechanism," and average dystrophin protein had a nearly elevenfold increase from baseline, the company said. DMD is a rare, progressive muscle disease; boys who have it lack the protein dystrophin that's critical to muscle function. Golodirsen was tested in boys with the DMD gene amenable to skipping exon 53. Sarepta's other, controversial DMD drug, eteplirsen, was the first approved for DMD and was approved for patients with the DMD gene amenable to skipping exon 51. Sarepta shares have surged 30.4% over the last three months, compared with a 1.2% rise in the S&P 500 .

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