PepGen reported third-quarter 2026 results alongside positive early data from a mid-dose group in its Phase 2 trial of PGN-EDODM1, an investigational treatment for myotonic dystrophy type 1 (DM1). The company also outlined plans to test a higher dose, extend treatment follow-up and report additional data in 2027.
The update centers on a small group of patients: six participants received 10 mg/kg in the FREEDOM2-DM1 multiple-dose study. PepGen said the results showed substantial correction of abnormal RNA splicing and improvements across several measures of physical function. The treatment remains in clinical development.
Trial results and safety
- At 10 mg/kg, mean splicing correction was 46.5% at Week 13, which PepGen described as the highest reported in a multiple-dose study. Five of six participants had more than 20% improvement, and four had more than 50%.
- The company also reported improvements in video Hand Opening Time, grip strength, the 10-Meter Walk/Run Test and Timed Up and Go. It did not provide numerical changes for those measures in the results summarized here.
- Treatment was generally well tolerated in the 10 mg/kg group. There were no serious or severe adverse events, dose-limiting toxicities or treatment discontinuations.
Next trial steps
- The independent safety board recommended advancing FREEDOM2-DM1 to a 12.5 mg/kg cohort after reviewing available safety data from the 10 mg/kg group and the separate long-term extension study. The highest-dose cohort is fully enrolled, and all participants have received up to three doses. PepGen expects results in the first half of 2027.
- PepGen plans to start an additional expansion cohort alongside the 12.5 mg/kg group, aiming to gather longer-term data over as much as nine months.
- The safety board also approved raising the dose in the FREEDOM-OLE extension study from 5 mg/kg to 10 mg/kg. Nineteen participants are enrolled. More than 35 doses at 10 mg/kg and 100 doses at 5 mg/kg had been administered; the company reported no serious adverse events or dose-limiting toxicities to date. One participant discontinued after a hypersensitivity reaction.
- PepGen expects interim safety data from the 10 mg/kg extension group and clinical data from the 5 mg/kg group in January 2027.
Regulatory and financial position
- PepGen said results from FREEDOM2-DM1 and the extension study are expected to support an end-of-Phase 2 meeting with regulators in the second half of 2027, contingent on removal of a partial clinical hold on FREEDOM2.
- The company ended September with $104.8 million in cash, which it said is sufficient to fund operations into 2028.
The 10 mg/kg findings provide an early efficacy and safety readout, while the fully enrolled 12.5 mg/kg cohort is the next planned dose test. The regulatory timeline remains conditional on resolving the partial clinical hold.