FDA Places Partial Clinical Hold on PepGen’s Phase 2 Myotonic Dystrophy Trial
FDA placed a partial hold on PepGen’s Phase 2 FREEDOM2-DM1 trial over pharmacology and toxicology questions, requiring submission of Phase 1 FREEDOM data. PepGen has clearance for FREEDOM2 in South Korea, Australia and New Zealand and plans 5 mg/kg data in Q1 2026 and 10 mg/kg data in H2 2026.
1. FDA Partial Clinical Hold
The FDA has placed a partial hold on PepGen’s Phase 2 FREEDOM2-DM1 multiple ascending dose trial in myotonic dystrophy type 1, citing questions on preclinical pharmacology and toxicology studies without querying blinded clinical data from earlier Phase 1.
2. Required Data Submission
PepGen is submitting additional analyses to address the FDA’s concerns, including data from the 15 mg/kg cohort of its Phase 1 FREEDOM single ascending dose study in DM1 patients.
3. International Trial Approvals
Despite the U.S. hold, PepGen has clearance to initiate FREEDOM2 dosing in South Korea, Australia and New Zealand, with the 10 mg/kg cohort ongoing in the U.K. and Canada.
4. Future Data Milestones
PepGen expects to report FREEDOM2 5 mg/kg cohort results in the first quarter of 2026 and 10 mg/kg cohort data in the second half of 2026, supporting its development pipeline beyond the current hold.