PepGen Inc (Nasdaq: PEPG), a clinical-stage biotechnology firm, on Monday announced a clinical hold by the U.S. Food and Drug Administration (FDA) on its IND application for the CONNECT2-EDO51 Phase 2 trial of PGN-EDO51 in Duchenne muscular dystrophy (DMD) patients. An official letter from the FDA is expected within 30 days.

CONNECT2-EDO51, a 25-week, multinational, double-blind, placebo-controlled trial, was designed to evaluate multiple ascending doses of PGN-EDO51 in DMD patients. The study remains active in the United Kingdom.

PGN-EDO51, developed using PepGen's Enhanced Delivery Oligonucleotide (EDO) technology, is aimed at exon 51 skipping to restore the dystrophin protein in approximately 13% of DMD patients. The candidate has received Orphan Drug and Rare Pediatric Disease Designations from the FDA.

Separately, PepGen continues to advance its CONNECT1-EDO51 trial, with full enrollment achieved in the 10 mg/kg cohort. The company remains committed to leveraging its EDO platform to develop next-generation oligonucleotide therapies for severe neuromuscular and neurological conditions.