Sarepta Therapeutics Halts Development of Vesleteplirsen, a Next-Generation Exon-Skipping Therapy for Duchenne Muscular Dystrophy

Discontinuation of Vesleteplirsen Development:
Sarepta Therapeutics has announced the discontinuation of its vesleteplirsen (SRP-5051) development program, a next-generation exon-skipping therapy for Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.

Reasons for Discontinuation:
The decision was based on an updated risk-benefit analysis, feedback from the FDA, and the evolving therapeutic landscape for DMD, including the approval of Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for DMD.

Safety Concerns:
The Phase 2 MOMENTUM study identified cases of hypomagnesemia (low blood magnesium levels) and a decline in kidney function in some patients, which persisted even after discontinuation of vesleteplirsen.

Impact on Sarepta’s Portfolio:
Sarepta will continue to develop other molecular candidates, including two late-stage gene therapies for limb-girdle muscular dystrophy, one of which is expected to be submitted for approval next year.

Financial Performance:
Sarepta reported strong third-quarter 2024 financial results, with net product revenues totaling $429.8 million, a 39% increase over the same quarter of the prior year, largely driven by the success of Elevidys.