- PepGen Inc. (PEPG, Financial) discontinues development of PGN-EDO51 after insufficient results in DMD trial.
- Focus shifts to promising DM1 program with data readouts expected in late 2025 and early 2026.
- PGN-EDODM1 shows robust target engagement with favorable safety profile in preliminary trials.
PepGen Inc. (PEPG) has announced the discontinuation of its PGN-EDO51 program following suboptimal results in the CONNECT1-EDO51 study. The trial, which involved a 10 mg/kg dose in Duchenne muscular dystrophy (DMD) patients, showed a mean increase of only 0.36% in dystrophin levels, far below the target.
Despite the setback, the study demonstrated a generally well-tolerated safety profile with only mild treatment-related adverse events reported. As a result, PepGen will cease all DMD-related research and development activities.
The company is now concentrating its efforts on its myotonic dystrophy type 1 (DM1) program. These initiatives involve PGN-EDODM1, PepGen's investigational drug designed to address DM1 pathology by targeting mis-splicing, which is a primary driver of the disorder. PGN-EDODM1 has demonstrated a mean mis-splicing correction of 29% in preliminary studies, with a favorable emerging safety profile.
PepGen plans to release data from its 15 mg/kg cohort in the FREEDOM-DM1 trial during the second half of 2025, and from its 5 mg/kg cohort in the FREEDOM2-DM1 trial in the first quarter of 2026. Both trials, focused on safety and efficacy endpoints, represent vital steps in PepGen’s clinical development pipeline.
DM1, a steadily progressive and life-shortening genetic disorder, affects approximately 40,000 people in the United States and more than 74,000 in Europe. The U.S. Food and Drug Administration has granted PGN-EDODM1 both Orphan Drug and Fast Track Designations, underscoring its potential impact in addressing this unmet medical need.
