Splicing correction in myotonic dystrophy
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PepGen Reports Highest Splicing Correction In Dystrophy Patients, Stock Doubles
Benzingaยท 2025-09-25 13:46
Core Viewpoint - PepGen Inc. has seen a significant increase in stock price following the release of positive clinical data from its FREEDOM-DM1 Phase 1 study for myotonic dystrophy type 1, indicating strong investor interest and confidence in the company's pipeline [1][8]. Clinical Data Summary - The FREEDOM-DM1 study reported a mean splicing correction of 53.7% in patients receiving a 15 mg/kg dose of PGN-EDODM1, with all six patients (100%) showing improved splicing correction [3][4]. - Previous doses of 5 mg/kg and 10 mg/kg showed mean splicing corrections of 12.3% and 29.1%, respectively, indicating a greater than dose-proportional increase in splicing correction with higher doses [4]. - Muscle tissue concentrations of PGN-EDODM1 also increased proportionally across the different doses at Day 28 [4]. Safety and Tolerability - PGN-EDODM1 was generally well-tolerated at the 15 mg/kg dose, with no serious treatment-related adverse events reported [4]. - All treatment-related adverse events were mild or moderate and transient, with one case of dose-limiting toxicity classified as a mild adverse event that resolved without intervention [5][6]. Future Expectations - PepGen plans to report results from the FREEDOM2-DM1 MAD study 5 mg/kg cohort in Q1 2026 and will begin dosing the 10 mg/kg cohort in the same timeframe [7]. - The company has priced an offering of 31.25 million shares at $3.20 per share, expecting gross proceeds of $100 million [7]. Stock Performance - As of the latest publication, PepGen shares rose by 122.18% to $5.91, trading within a 52-week range of $0.88 to $9.93 [8].