PepGen reported positive results from the 10 mg/kg cohort of its Phase 2 FREEDOM2-DM1 trial evaluating PGN-EDODM1 for myotonic dystrophy type 1, while ending the third quarter of 2026 with $104.8 million in cash and investments.
The cohort produced mean splicing correction of 46.5%, which PepGen described as the highest reported in a multiple-dose study. Five of six treated participants showed more than 20% improvement, while four exceeded 50% at week 13.
The study also produced improvements across measures of hand opening, grip strength, walking, and mobility. PGN-EDODM1 was generally well tolerated at 10 mg/kg, with no treatment discontinuations, serious or severe adverse events, or dose-limiting toxicities reported in the cohort.
An independent monitoring board recommended advancing the trial to its third and highest dose cohort at 12.5 mg/kg. That cohort is fully enrolled, and participants have received as many as three doses. Results are expected during the first half of 2027.
PepGen is also continuing an open-label extension study. Interim safety data from the 10 mg/kg group and clinical data from participants receiving 5 mg/kg are expected in January 2027.
The FDA placed the U.S. portion of FREEDOM2 under a partial clinical hold in March because of findings from mouse studies. PepGen is conducting additional nonclinical work and remains in discussions with the agency. Trials outside the U.S. continue.
Research and development expense increased to $14.5 million from $13.4 million. General and administrative expense rose to $5.9 million from $5.2 million.
The quarterly net loss widened to $19.4 million, or $0.28 per share, from $18 million, or $0.52 per share. PepGen expects its cash to support planned operations into 2028.
KEY QUOTE:
“The positive 10 mg/kg cohort results we announced today were made possible by our team’s focused execution. We are very confident in PGN-EDODM1’s potential to deliver meaningful benefit to the DM1 community.”
James McArthur, PhD, President and Chief Executive Officer of PepGen
