- Sarepta Therapeutics Inc SRPT announced positive results from Part A in a tiny group of 4 patients of the MOMENTUM Phase 2 study evaluating SRP-5051 in Duchenne muscular dystrophy (DMD) patients amenable to exon 51 skipping.
- SRP-5051 is the company's next-generation peptide phosphorodiamidate morpholino oligomer (PPMO).
- At a median of week 12, 30 mg/kg of SRP-5051 resulted in mean dystrophin production of 6.55% of normal. Dystrophin expression was measured by western blot.
- It is twice the dystrophin expression compared to the 20 mg/kg cohort at week 12 (mean expression of 3.06%) and eight times that of Sarepta's already approved DMD treatment Exondys 51 (eteplirsen) comparison group (mean expression of 0.82%).
- At week 12, 30 mg/kg of SRP-5051 dosed monthly resulted in mean exon skipping of 10.79%, more than four times increase in exon skipping compared to the 20 mg/kg cohort of SRP-5051 (mean exon skipping of 2.57%, n=2) and an 18x increase in exon skipping compared to a weekly 30 mg/kg dose of eteplirsen at 24 weeks (mean exon skipping of 0.59%, n=16).
- Sarepta's predictive model indicates that SRP-5051 at 30 mg/kg will achieve greater than 10% dystrophin with monthly chronic dosing.
- Two cases of hypomagnesemia were identified in patients taking SRP-5051. Cases have resolved with magnesium supplementation, and analysis of all available data indicates that the hypomagnesemia is monitorable and manageable.
- Dystrophin is a protein found in muscle cells, and lack of it causes muscle damage and progressive weakness.
- Price Action: SRPT shares are up 8.24% at $76.68 in the market trading session on the last check Monday.
© 2024 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
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