Sarepta shares PhII data for next-gen exon skipping drug, but regulatory pathway is unclear
Sarepta Therapeutics announced Monday that its experimental Duchenne muscular dystrophy drug led to some expression of dystrophin — the muscle protein that is mutated in people with Duchenne — in a Phase II study.
In 20 patients who received a high dose of SRP-5051 for 28 weeks, Sarepta reported a mean 5.17% in dystrophin expression, and results were roughly similar between ambulatory and non-ambulatory patients. At the low dose, mean dystrophin expression was 2.81% in 20 patients. Those expression levels equated to a change of 4.53% and 2.00%, respectively, in expression levels from baseline.
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