PepGen declares positive data from Phase II trial of Duchenne muscular dystrophy

PepGen has announced positive results from the first dose cohort (5mg/kg) of its CONNECT1-EDO51 Phase II trial for PGN-EDO51, an investigational therapy for Duchenne muscular dystrophy (DMD) targeting exon 51 mutations.

This open-label study evaluates PGN-EDO51 at multiple dose levels, starting with 5mg/kg administered intravenously every four weeks over 12 weeks. Key endpoints include safety, dystrophin production, and exon skipping, with additional pharmacokinetic parameters assessed.

Results showed PGN-EDO51 achieved higher exon skipping levels compared to other therapies and a mean muscle-adjusted dystrophin level of 1.49% of normal. The 5mg/kg dose was well tolerated, with no discontinuations, interruptions, or significant adverse events reported. All three patients in this cohort continue treatment in the long-term extension phase.

Dosing has begun at 10mg/kg, with results expected in early 2025. PGN-EDO51 demonstrated meaningful exon skipping and dystrophin increases, highlighting the efficacy of PepGen’s Enhanced Delivery Oligonucleotide technology.