PepGen Inc. (PEPG) Halts DMD Program After Trial Miss; Shifts Focus to DM1

Boston-based biotech PepGen Inc. (NASDAQ:PEPG) announced today it will discontinue all Duchenne muscular dystrophy (DMD) research after its lead candidate, PGN-EDO51, failed to achieve target dystrophin levels in the CONNECT1-EDO51 trial. Despite being well tolerated at 10 mg/kg, PGN-EDO51 only increased dystrophin to 0.59% of normal levels, falling short of expectations. All treatment-related adverse events were mild, and no serious events were reported.

PepGen Inc. (PEPG) Halts DMD Program After Trial Miss; Shifts Focus to DM1

A healthcare professional in a meeting with a patient discussing care options using digital technology.

“We are disappointed by the results and will wind down our DMD program,” said CEO James McArthur, PhD, thanking patients and staff for their support.

PepGen Inc. (NASDAQ:PEPG) will now focus on its promising myotonic dystrophy type 1 (DM1) program, PGN-EDODM1, which has shown robust target engagement and a favorable safety profile.

PepGen Inc. (NASDAQ:PEPG) expects data from its Phase 1 FREEDOM-DM1 trial’s 15 mg/kg cohort in the second half of 2025, and from the Phase 2 FREEDOM2-DM1 5 mg/kg cohort in early 2026. PGN-EDODM1, which has Orphan Drug and Fast Track status, targets the genetic root of DM1, a rare, life-shortening disorder with no approved treatments.

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