Sarepta Therapeutics has announced positive data from Part B of the MOMENTUM study (Study SRP-5051-201), a global, Phase 2, multi-ascending dose clinical trial of SRP-5051 (vesleteplirsen) that enrolled patients aged 8 to 21 years. SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy 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.
“SRP-5051 dosed every four weeks is showing substantially higher increases in dystrophin and exon skipping compared to eteplirsen dosed weekly. The data suggest a favorable benefit-risk profile for SRP-5051 and we look forward to discussing the results and next steps with FDA. As the leader in Duchenne, Sarepta is committed to advancing meaningful treatments for those with Duchenne and other rare diseases where there is unmet need.”Louise Rodino-Klapac, Ph.D., executive vice president, chief scientific officer and head of research and development at Sarepta Therapeutics
The MOMENTUM study is currently underway in Canada at London Health Sciences Centre in London, Ontario. Defeat Duchenne Canada welcomes the positive efficacy and safety results for SRP-5051. These findings suggest a promising benefit-risk profile for Duchenne patients, and we look forward to hearing more updates from Sarepta regarding SRP-5051.