PepGen reported topline results from the 5 mg/kg cohort of the Phase 2 FREEDOM2-DM1 trial on March 30, 2026, showing PGN-EDODM1 was well-tolerated with no serious adverse events25.
Mean splicing correction was 7.3% in treated patients (n=6) vs. 6.8% in placebo (n=2), rising to 22.9% when excluding one outlier patient with marked worsening2.
Promising trends in middle-finger vHOT (video hand opening time) favored treatment over placebo worsening, but no benefits in 10-meter walk/run or handgrip strength at this low dose2.
Phase 1 trial showed higher splicing correction (up to 53.7% at high dose) and supported Phase 2 initiation1.
FDA placed a partial clinical hold on U.S. sites due to preclinical mouse data concerns, but trial continues elsewhere3.
10 mg/kg cohort is over halfway enrolled, with data expected in H2 20262.
Sources:
1. https://musculardystrophynews.com/news/mda-2026-dm1-therapy-pgn-edodm1-shows-biomarker-activity/
2. https://www.stocktitan.net/sec-filings/PEPG/8-k-pep-gen-inc-reports-material-event-2a269b1453a5.html
3. https://www.biopharmadive.com/news/pepgen-fda-hold-myotonic-dystrophy-drug-trial/813883/