PepGen To Voluntarily Pause Mid-Stage Duchenne Muscular Dystrophy Study, Stock Falls
PepGenPepGen(US:PEPG) Benzinga·2025-03-04 18:41

Core Insights - PepGen Inc. has decided to voluntarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) to review results from the 10 mg/kg cohort of the CONNECT1 study [1][2] - The first two cohorts of the CONNECT1 study are fully enrolled, with data from the 10 mg/kg cohort expected in Q3 2025, and no new safety issues have been reported since the last update in January 2025 [1][4] Group 1 - The pause in the CONNECT2 study allows the company to gather additional safety data and assess the impact of the 10 mg/kg dose on dystrophin levels, potentially improving the design of CONNECT2 [2][3] - The company is focusing resources on completing the CONNECT1 study and advancing the FREEDOM studies in myotonic dystrophy type 1 with PGN-EDODM1, which has shown encouraging initial clinical data [3] Group 2 - Initial data from the FREEDOM-DM1 trial indicated significant dose-dependent splicing correction, with mean corrections of 12.3% at 5 mg/kg and 29.1% at 10 mg/kg at 28 days post-dosing [4] - Following the announcement, PEPG stock experienced a decline of 21%, trading at $2.22 [4]