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– PGN-EDO51 at 5 mg/kg was well tolerated, and all patients continued to long-term extension portion of trial. Dosing of second cohort at 10 mg/kg is ongoing –
– Four doses of PGN-EDO51 at 5 mg/kg achieved mean exon skipping levels of 2.15% after three months of dosing –
– PGN-EDO51 at 5 mg/kg showed mean muscle-adjusted dystrophin level of 1.49%, a 0.70% increase from baseline, after three months of dosing –
– PGN-EDO51 at 5 mg/kg showed mean absolute dystrophin level of 0.61%, a 0.26% increase from baseline, after three months of dosing –
– Conference call scheduled for 4:30 p.m. ET –
PepGen Inc. (NASDAQ:PEPG), 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 positive clinical data from the first dose cohort (5 mg/kg) of PGN-EDO51, its lead investigational candidate for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach. In the ongoing CONNECT1-EDO51 Phase 2 open-label trial, PGN-EDO51 demonstrated higher levels of exon skipping than previously reported studies with other oligonucleotide therapies at similar PMO dose levels in DMD patients. The Company also reported that change from baseline in total dystrophin production and muscle-adjusted dystrophin production was comparable to, or higher than, previously reported studies with other oligonucleotide therapies at similar PMO dose levels in DMD patients. Today at 4:30 p.m. ET, the Company will host a conference call with the CONNECT1 lead investigator Dr. Hugh McMillan to discuss the data being presented.
Posted In: PEPG
