Today, Sarepta shared an update for its investigational PPMO SRP-5051 clinical trial study. MOMENTUM, the name of their phase 2 clinical trial peptide phosphorodiamidate morpholino oligomer study is for patients with Duchenne Muscular Dystrophy amenable to skipping exon 51.
- Results from the multiple-ascending dose trial demonstrate proof-of-concept for SRP-5051 and support continued dose escalation
- At a total dose exposure approximately 10x lower than eteplirsen, SRP-5051 at 20 mgs/kg showed enhanced tissue exposure, greater exon skipping, and greater dystrophin production with no negative renal or other laboratory findings
- These are the first clinical results from the Company’s peptide phosphorodiamidate morpholino oligomer (PPMO) technology, its next-generation chemistry platform