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PepGen Announces Positive Phase 2 FREEDOM2-DM1 10 mg/kg Results Demonstrating Highest Reported Splicing Correction and Improvements Across Multiple Functional Measures

PEPG
  • PepGen Inc. reported positive topline results from the 10 mg/kg cohort of the Phase 2 FREEDOM2-DM1 trial for its investigational therapy PGN-EDODM1 in patients with myotonic dystrophy type 1.
  • Mean splicing correction in muscle reached 46.5% at Week 13, with five of six participants exceeding 20% correction and four exceeding 50% correction.
  • Video Hand Opening Time, an objective measure of myotonia, improved by 3.9 seconds (52.7%) from baseline at Week 16.
  • Functional improvements were observed in hand grip strength (16.2%), Timed Up and Go assessments (8.5%), and the 10-Meter Walk/Run Test (6.8%) at Week 16.
  • PGN-EDODM1 was generally well tolerated at 10 mg/kg, with no serious adverse events, severe adverse events, or dose-limiting toxicities reported.
  • Treatment-emergent adverse events were mild or moderate, with the most common being nausea; renal events were transient \& mild without evidence of cumulative toxicity.
  • Patient-reported outcomes were inconclusive at Week 16, although the Patient Global Impression of Change showed a favorable directional trend versus placebo.
  • The 12.5 mg/kg cohort of FREEDOM2-DM1 is fully enrolled, with clinical results expected in the first half of 2027.
  • PepGen plans to report interim clinical data for the 5 mg/kg dose \& safety data for the 10 mg/kg dose from the open-label extension study in January 2027.
  • A conference call to discuss these results was scheduled for 8:00 a.m. ET on the day of the announcement.