Clinical TrialNov 10, 2026, 08:01 AM
PepGen: Positive DM1 Trial Results; Splicing Correction at 46.5%
AI Summary
PepGen Inc. announced positive topline results from the 10 mg/kg cohort of its Phase 2 FREEDOM2-DM1 trial for myotonic dystrophy type 1 (DM1). The study demonstrated a mean splicing correction of 46.5%, the highest reported in a multiple-dose study, along with significant improvements in functional measures like hand opening time, grip strength, and mobility. The drug, PGN-EDODM1, was well-tolerated. The company also reported Q3 2026 financial results, with a net loss of $19.4 million, and stated it has sufficient cash to fund operations into 2028.
Key Highlights
- PepGen reports 46.5% mean splicing correction in Phase 2 FREEDOM2-DM1 10mg/kg cohort for DM1.
- Improvements observed in vHOT (52.7%), hand grip strength (16.2%), TUG (8.5%), and 10MWRT (6.8%).
- PGN-EDODM1 generally well tolerated; no serious adverse events or dose-limiting toxicities reported.
- 12.5 mg/kg FREEDOM2-DM1 cohort fully enrolled; results expected in 1H 2027.
- Interim FREEDOM-OLE data (5mg/kg clinical, 10mg/kg safety) expected January 2027.
- Company had $104.8M cash as of Sept 30, 2026, sufficient into 2028.
- Net loss for Q3 2026 was $19.4M, or $(0.28) per share.
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