Sarepta Therapeutics (NASDAQ:SRPT) revealed a notable update on Monday, sharing additional results from its Phase 2 MOMENTUM study for SRP-5051 (vesleteplirsen). The investigational therapy is aimed at addressing the neuromuscular disorder, Duchenne Muscular Dystrophy (DMD).

Duchenne Muscular Dystrophy (DMD) stems from mutations in the DMD gene, responsible for orchestrating the production of dystrophin, a pivotal protein for muscular functionality.

The fresh data from Part B of the trial showcased a mean dystrophin expression of 5.17% at Week 28 with monthly dosing of SRP-5051 at the higher target dose of approximately 30 mg/kg. According to Sarepta, the mean exon 51 skipping – a mechanism tailored to facilitate the generation of a specific form of dystrophin protein – reached an approximate value of 11.11% over 28 weeks. Notably, about 5% of ambulatory DMD patients and 6% of non-ambulatory patients exhibited consistent dystrophin expressions.

While seven serious, treatment-emergent adverse events, with four linked to hypomagnesemia and three associated with hypokalemia, were reported in terms of safety, the trial did not witness any treatment-related discontinuations.

Part B of the trial enrolled 40 DMD patients aged between 8 and 21 across the U.S., Canada, and Europe. A prior readout from MOMENTUM, pertaining to Part A of the trial, indicated that after 30 mg/kg dosing, the mean dystrophin expression had reached 6.55% by week 12.