Sarepta Therapeutics Plunges on Disappointing DMD Trial Results
Sarepta Therapeutics shares cratered 37% in pre-market trading after revealing lackluster data from a pivotal Duchenne muscular dystrophy study. The nine-year trial of casimersen and golodirsen failed to show statistically significant improvement in motor function, casting doubt on the company's flagship PMO-based therapy platform.
The biotech firm reported merely a 0.05-step improvement in stair-climbing ability after 96 weeks of treatment—a marginal gain that fell far short of investor expectations. This setback arrives as Sarepta seeks to expand its genetic medicine portfolio for rare neuromuscular disorders.