Sarepta Therapeutics Announces Positive Data for Duchenne Muscular Dystrophy Amenable to Skipping Exon 51

Sarepta Therapeutics reported positive data for SRP-5051, their next-generation exon skipping agent designed for individuals amenable to skipping Exon 51.

In part B of the Phase 2 MOMENTURM study, ambulatory and non-ambulatory individuals who received the high dose of 30 mg/kg once every 4 weeks had an average dystrophin expression of 5.17% at 28 weeks.

This is significantly higher levels of dystrophin than seen with Sarepta’s FDA-approved agent, Exondys 51 (eteplirsen). Compared to Exondys 51, SRP-5051 contains a peptide designed to increase the ability of the exon-skipping agent to get into cells, and is dosed once every four weeks rather than every week.

In terms of safety concerns, hypomagnesemia has been previously identified with SRP-5051 treatment, and in this study was monitored and managed by having participants take magnesium supplements prophylactically.

The press release, which contains more information about both the low and high-dose cohorts, is HERE

@sareptatherapeutics said that they are requesting a pre-NDA meeting with the FDA for later this year (Q3 2024).