Sarepta Therapeutics Inc.'s shares jumped about 15 percent on Wednesday after the company's drug to treat patients with a form of a fatal muscle-wasting disease met the main goal of an early-stage trial.
Duchenne Muscular Dystrophy is a rare and progressive genetic disorder that hampers muscle movement and Sarepta said its drug was successful in improving the production of dystrophin, a protein that helps keep muscles intact.
DMD, which affects about one in every 3,500 to 5,000 males, happens when an exon, or exons – one of the vital parts that make a gene – are deleted or absent, interfering with the rest of the gene being pieced together.
Sarepta's drug, golodirsen, is for patients who are amenable to what is known as exon 53 skipping, which allows the other exons to form a complete gene.
The 'exon 53 skipping' was successful in all the 25 patients tested in the trial, Sarepta said.
"Golodirsen does appear to be a more proficient exon skipper than Exondys 51," Leerink analyst Joseph Schwartz wrote in a client note.
Exondys 51 is Sarepta's other DMD drug that was approved in September last year after the U.S. Food and Drug Administration bowed to patient pressure and went against the recommendation of its top scientists and a panel of outside advisers.
"This (positive data on golodirsen) reduces any uncertainty around the efficacy of Exondys 51 and decreases any risk around the pending confirmatory study," Needham analyst Chad Messer said.
Sarepta's shares were up about 15 percent at $47.30 in premarket trading on Wednesday. The company will ring the closing bell on Nasdaq on Wednesday in recognition of World Duchenne Awareness Day on Sept. 7.
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