FDA Places Partial Clinical Hold on PepGen's FREEDOM2-DM1 Trial
The FDA has placed a partial clinical hold on PepGen's Phase 2 FREEDOM2-DM1 trial for myotonic dystrophy type 1, citing questions about preclinical pharmacology and toxicology studies. The company is submitting additional analyses to address the FDA's concerns.
PepGen Inc. (NASDAQ: PEPG) announced that the U.S. Food and Drug Administration has placed a partial clinical hold on the company's FREEDOM2-DM1 Phase 2 clinical trial. The hold relates to questions about previously submitted preclinical pharmacology and toxicology studies for PGN-EDODM1, the company's investigational treatment for myotonic dystrophy type 1.
The FDA did not cite concerns regarding blinded clinical data from the Phase 1 FREEDOM study that was previously submitted to support the FREEDOM2 trial initiation in the U.S. PepGen stated it is submitting additional analyses, including recently unblinded FREEDOM data, to address the FDA's questions.
No U.S. patients have been enrolled in FREEDOM2. The company received regulatory clearance to initiate FREEDOM2 in South Korea, Australia, and New Zealand. The trial continues dosing patients at 10 mg/kg in Canada and the UK following a Data Safety Monitoring Board recommendation to escalate doses.
PepGen maintained its timeline for reporting data from the 5 mg/kg cohort in the first quarter of 2026 and from the 10 mg/kg cohort in the second half of 2026. Patients from the FREEDOM and FREEDOM2 studies in Canada are continuing into an Open Label Extension study, with regulatory clearance received for the UK.
PGN-EDODM1 uses the company's Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide designed to restore normal splicing function of MBNL1, a key RNA splicing protein. The FDA has granted the drug both Orphan Drug and Fast Track designations for DM1 treatment.