PepGen Temporarily Pauses Exon 51 Skipping Study

PepGen is “temporarily paused” a Phase 2 study of its exon 51 skipping (PGN-EDO51) drug for Duchenne muscular dystrophy.

PepGen, a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced its voluntary decision to temporarily pause the Phase 2 CONNECT2-EDO51 study of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) until the Company can review results from the 10 mg/kg cohort in the ongoing Phase 2 CONNECT1-EDO51 study.

The first two cohorts of the CONNECT1 study are fully enrolled and data from the 10 mg/kg cohort are expected during the third quarter of 2025. No new safety issues related to PGN-EDO51 have been observed since the Company’s last safety update as of January 23, 2025.

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