PepGen Inc. announced the voluntary discontinuation of its PGN-EDO51 program for Duchenne muscular dystrophy (DMD) and all related research and development activities. This decision was made following disappointing results from the 10 mg/kg cohort of the CONNECT1-EDO51 Phase 2 study.
In the CONNECT1 study, PGN-EDO51 increased exon 51 skipped transcripts to 4.26%, representing a mean increase of 3.5%. However, total dystrophin levels only rose to 0.59% of normal levels, a mean increase of 0.36%, which did not meet the company's target dystrophin levels. The safety profile of PGN-EDO51 was generally favorable, with all treatment-related adverse events being mild.
With the discontinuation of the DMD programs, PepGen will now focus its resources entirely on advancing its PGN-EDODM1 program for Myotonic Dystrophy Type 1 (DM1), which is currently in Phase 2 clinical development. The company expects to report data from the FREEDOM-DM1 15 mg/kg cohort in the second half of 2025 and from the FREEDOM2-DM1 5 mg/kg cohort in Q1 2026.
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