PepGen says FREEDOM clinical study achieves all key objectives

PepGen (PEPG) announced positive clinical data from the 15 mg/kg dose cohort of its ongoing FREEDOM-DM1 Phase 1 single ascending dose study in patients with myotonic dystrophy type 1. These latest results demonstrated a mean splicing correction of 53.7% following a single 15 mg/kg dose of PGN-EDODM1, substantially higher than any previously reported splicing correction in DM1 patients. “We are delighted to report that the FREEDOM clinical study achieved all of its key objectives, including unprecedented splicing correction following a single dose of PGN-EDODM1 at 15 mg/kg,” said Paul Streck, MD, MBA, Executive Vice President of Research and Development at PepGen. “Since mis-splicing is the underlying cause of DM1, we believe high levels of splicing correction have the potential to reverse the underlying molecular defects, and produce functional improvements in multiple outcome measures, including myotonia and muscle weakness, in repeat dose studies.” James McArthur, PhD, PepGen’s President and Chief Executive Officer, added, “These data build upon and reinforce our previously reported splicing correction levels seen in the FREEDOM single dose cohorts of 5 and 10 mg/kg of PGN-EDODM1. Additionally, we are excited to report that 100% of patients in the 15 mg/kg cohort of our FREEDOM trial showed improved splicing correction following treatment. We look forward to reporting data from the first cohort of FREEDOM2, our multiple ascending dose (MAD) study currently underway, in the first quarter of 2026.”