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Sarepta Therapeutics Announces Positive Clinical Results From MOMENTUM, a Phase 2 Clinical Trial of SRP-5051 in Patients With Duchenne Muscular Dystrophy Amenable to Skipping Exon 51


Sarepta Therapeutics, Inc., the leader in precision genetic medicine for rare diseases, announced results from the ongoing MOMENTUM study (Study 5051-201), a global Phase 2 clinical trial, of SRP-5051, its next-generation treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. This is the first clinical data from SRP-5051, an investigational treatment that uses Sarepta’s peptide phosphorodiamidate morpholino oligomer (PPMO) technology. PPMO technology includes a proprietary cell-penetrating peptide that is conjugated to Sarepta’s PMO backbone with the goal of increasing cellular uptake of drug in the muscle tissue.

Results from Part A of the multi-ascending dose MOMENTUM study found consistently higher tissue exposure, exon-skipping and dystrophin production in patients taking a monthly dose of SRP-5051 compared to baseline. SRP-5051 was generally well-tolerated across all doses studied, with no clinical or laboratory findings reported. The results support continued dose escalation of SRP-5051 and further clinical development.

 

Sarepta Therapeutics Announces Positive Clinical Results From MOMENTUM, A Phase 2 Clinical Trial Of SRP-5051 In Patients With Duchenne Muscular Dystrophy Amenable To Skipping Exon 51