Sarepta's Phase II study with Duchenne candidate SRP-5051 looks encouraging

US precision genetic medicines firm Sarepta Therapeutics (Nasdaq: SRPT) has announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase II, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged eight to 21 years.

SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

Data from Part B of MOMENTUM found that at the higher, target dose, approximately 30 mg/kg dosed every four weeks, SRP-5051 resulted in mean dystrophin expression of 5.17%, and mean exon skipping of 11.11% at 28 weeks (n=20). Consistent dystrophin expression was seen in ambulatory (4.76%, n=11) and non-ambulatory (5.67%, n=9) participants at 28 weeks. Hypomagnesemia has previously been identified in patients taking SRP-5051 and was managed and monitored through prophylactic magnesium supplementation as part of the study protocol.