PepGen reports positive 10 mg/kg data from FREEDOM2-DM1 trial in DM1

PepGen reported on November 10, 2026 that it released positive topline results from the 10 mg/kg multiple ascending dose cohort of the ongoing Phase 2 FREEDOM2-DM1 trial (NCT06667453) evaluating PGN-EDODM1 in patients with myotonic dystrophy type 1.¹

Topline data from the 10 mg/kg dose cohort demonstrated a mean splicing correction of 46.5%, the highest reported to date in a multiple dose study.¹ Five of six participants demonstrated greater than 20% splicing improvement, and four participants had greater than 50% splicing improvement at Week 13.¹ PGN-EDODM1 continued to be generally well tolerated at 10 mg/kg in the FREEDOM2 study with no treatment discontinuations, no serious or severe adverse events, and no dose-limiting toxicities reported.¹

An independent Data and Safety Monitoring Board recommended advancing the trial into the third and highest dose cohort, 12.5 mg/kg, which is fully enrolled with all participants having received up to three doses, and results are expected in the first half of 2027.¹ The DSMB also approved dose escalation in the open-label extension study from 5 mg/kg to 10 mg/kg, and to date more than 35 doses at 10 mg/kg and 100 doses at 5 mg/kg have been delivered, with 19 participants now enrolled in the OLE.¹ Interim 10 mg/kg OLE safety and 5 mg/kg OLE clinical data are expected in January 2027.¹

On regulatory matters, data from the FREEDOM2 and FREEDOM-OLE studies are expected to support an end-of-Phase 2 meeting in the second half of 2027, pending removal of the partial clinical hold.¹ That hold was issued by the FDA in March 2026 citing nonclinical findings in mice.¹ Separately, in October 2026, under the currently open FDA IND, the agency selected PGN-EDODM1 for its CMC Development and Readiness Pilot program.¹

On finances, cash, cash equivalents and marketable securities were $104.8 million as of September 30, 2026, which PepGen said is sufficient to fund operations into 2028.¹