PepGen Abandons Duchenne Program, Shifts Spotlight To Another Rare Disease Candidate

Core Viewpoint - PepGen Inc. has decided to focus on its myotonic dystrophy type 1 (DM1) program while discontinuing its Duchenne muscular dystrophy (DMD) research due to unsatisfactory results in dystrophin levels from its clinical study [1][4]. Group 1: DMD Program Discontinuation - The company has voluntarily discontinued the development of PGN-EDO51 and plans to wind down all DMD-related research activities [1]. - In the CONNECT1 study's 10 mg/kg cohort, PGN-EDO51 showed a mean increase of 3.5% in exon 51 skipped transcripts, but total dystrophin only increased to 0.59% of normal levels, indicating limited efficacy [2]. - The safety profile of PGN-EDO51 was generally favorable, with all treatment-related adverse events being mild and no serious adverse events reported [3]. Group 2: Focus on DM1 Program - PepGen's investigational drug PGN-EDODM1 for DM1 has shown robust target engagement, achieving a mean mis-splicing correction of 29% after a single 10 mg/kg dose, with a favorable safety profile [5]. - The company anticipates reporting data from its FREEDOM-DM1 15 mg/kg cohort in the second half of 2025, which is a Phase 1 trial focusing on safety and splicing correction [5]. - Data from the 5 mg/kg cohort of the FREEDOM2-DM1 study is expected in the first quarter of 2026, which is a Phase 2 trial assessing multiple doses and their effects [6]. Group 3: Market Reaction - Following the announcement, PepGen's stock fell by 6.69%, trading at $1.47 during the premarket session [6].