Core Insights - PepGen Inc. announced positive clinical data from the FREEDOM-DM1 Phase 1 study, showing a mean splicing correction of 53.7% in patients with myotonic dystrophy type 1 (DM1) after a single 15 mg/kg dose of PGN-EDODM1, which is significantly higher than previous results [1][2][5] Clinical Study Results - The FREEDOM-DM1 study achieved all key objectives, demonstrating unprecedented splicing correction with 100% of patients in the 15 mg/kg cohort showing improvement [2][5] - Previous doses of 5 mg/kg and 10 mg/kg resulted in mean splicing corrections of 12.3% and 29.1% respectively, indicating a greater than dose-proportional increase in splicing correction [5] - Muscle tissue concentrations of PGN-EDODM1 also showed greater than dose-proportional increases across the different dosing cohorts [5] Safety and Tolerability - PGN-EDODM1 was generally well-tolerated at the 15 mg/kg dose, with all treatment-related adverse events being mild or moderate and transient [5][6] - No serious treatment-related adverse events were reported, and there were no electrolyte-related adverse events [5][6] Future Developments - PepGen anticipates reporting results from the FREEDOM2-DM1 multiple ascending dose study in the first quarter of 2026, with the 5 mg/kg cohort results expected first [6][9] - The company is also preparing to begin dosing the 10 mg/kg cohort in the same timeframe [6] About PGN-EDODM1 - PGN-EDODM1 is designed to restore normal splicing function of MBNL1, addressing the underlying cause of DM1 by binding to pathogenic CUG repeat expansions [8] - The therapy has received Orphan Drug and Fast Track Designations from the U.S. FDA for the treatment of DM1 [8] About Myotonic Dystrophy Type 1 - DM1 is a progressive disorder affecting skeletal, cardiac, and smooth muscle, with a global prevalence estimated at 1 in 8,000 people [11][12] - The disease can lead to various complications, including myotonia, muscle weakness, and cognitive impairments, significantly impacting life expectancy [12]