PepGen Inc. has shared positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, the company’s lead investigational candidate for patients with Duchenne whose mutations are amenable to exon 51-skipping. PGN-EDO51 is an PMO-exon skipping therapy bound to a peptide which improves uptake into muscle cells, and targets those amenable to exon 51 skipping.

According to PepGen, in the ongoing CONNECT1-EDO51 Phase 2 open-label trial, PGN-EDO51 produced mean exon skipping in biceps tissue of 2.15% at week 13 compared to baseline. Additionally, change from baseline in total dystrophin production and muscle-adjusted dystrophin production (0.26% and 0.7% respectively) was comparable to, or higher than, previously reported studies of other oligonucleotide therapies of similar doses.

PepGen also emphasized that PGN-EDO51 is well tolerated and has demonstrated a favorable safety profile, which the company says may support the ongoing evaluation of the 10 mg/kg monthly dose cohort in CONNECT1. PepGen plans to present additional results from the 5 mg/kg cohort at a medical meeting later in the year, and to report initial results from the 10 mg/kg cohort in early 2025.

PepGen recently participated in the Research Row: Strategies to Restore Dystrophin session at PPMD’s 30th Annual Conference, sharing information about PGN-EDO51. Watch the session recording here. 

PPMD is pleased to learn this news and we look forward to future updates from PepGen on the development of PGN-EDO51.

Read PepGen’s press release here.