Sarepta has announced positive results relating to its dose-ascending Phase 2 clinical trial of SRP-5051, a treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping. SRP-5051 is Sarepta’s first “next-generation” PPMO (peptide-conjugated PMO) therapy.
In biopsies taken at 3 months, following monthly dosing, 30 mg/kg of SRP-5051 resulted in 18 times the exon skipping and eight times the dystrophin production achieved by eteplirsen dosed weekly for 6 months. (Eteplirsen is Sarepta’s already-approved PMO therapy for DMD patients amenable to Exon 51 skipping). One major adverse event reported from the SRP-5051 trial was reduced levels of magnesium, which can lead to heart damage and seizures. It was reported that this was successfully resolved with oral magnesium supplementation.
The CEO of Sarepta said that the early-stage POC data confirm the potential of Sarepta’s PPMO platform to be a step order improvement over its current PMO platform. Company modelling suggests that dystrophin expression levels of 10% of normal or greater will be seen over time with SRP-5051. The company now plans to meet with the FDA to seek an accelerated approval pathway for SRP-5051 in the US.
Sarepta has currently five more PPMO therapies in preclinical development, all of which target DMD.
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