Sarepta Therapeutics, Inc. today announced positive data from Part B of SRP-5051-201, the MOMENTUM study. MOMENTUM is a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for individuals with Duchenne who are amenable to exon 51 skipping.
Results 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. Sarepta also shared that consistent dystrophin expression was seen in ambulatory and non-ambulatory participants at 28 weeks.
PPMD is pleased to learn these results, which suggest a favorable benefit-risk profile for SRP-5051. We look forward to future updates from Sarepta on SRP-5051.
Read Sarepta’s press release here.