FDA Partially Holds PepGen’s FREEDOM2 Phase 2 Trial; 5 mg/kg Data Due Q1 2026
FDA placed a partial hold on PepGen’s FREEDOM2-DM1 Phase 2 trial of PGN-EDODM1 for DM1, citing questions on preclinical pharmacology and toxicology. PepGen is submitting analyses, dosing 10 mg/kg cohorts in Canada and UK, has cleared sites in New Zealand and South Korea and expects 5 mg/kg data in Q1 2026 and 10 mg/kg in H2 2026.
1. Partial Clinical Hold Details
The U.S. Food and Drug Administration has placed a partial clinical hold on the FREEDOM2-DM1 Phase 2 multiple ascending dose trial of PGN-EDODM1 in myotonic dystrophy type 1 patients, raising concerns over preclinical pharmacology and toxicology study submissions. The hold does not relate to blinded Phase 1 data, which PepGen is providing in supplemental analyses as it works to address the FDA’s questions.
2. Ongoing Trial Activities and Timeline
PepGen continues to dose patients at 10 mg/kg in Canada and the UK following a Data Safety Monitoring Board recommendation and has regulatory clearance for FREEDOM2 sites in New Zealand and South Korea. Patients from prior cohorts are moving into an Open Label Extension study, and PepGen forecasts reporting 5 mg/kg cohort results in the first quarter of 2026 and 10 mg/kg cohort data in the second half of 2026.