Sarepta leaps on mainly positive SRP-5051 results in patients with DMD

4 May 2021

Shares of Sarepta Therapeutics (Nasdaq: SRPT) closed up nearly 9% at $77.11 yesterday, after the US rare diseases specialist announced positive results from Part A of the MOMENTUM study (Study 5051-201), a global, Phase II, multi-ascending dose clinical trial of SRP-5051, its next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping.

In biopsies taken at a median of 12 weeks and after only three doses, results from Part A of MOMENTUM study found that the 30mg/kg of SRP-5051 dosed monthly resulted in 18 times the exon skipping and eight times the dystrophin production as eteplirsen, dosed weekly for 24 weeks. Exon-skipping and dystrophin production in the 30mg/kg cohort were also consistently higher than the 20mg/kg cohort of MOMENTUM.

Adverse event reported, but seen as manageable

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