other material
confidence high
sentiment positive
materiality 0.75
PepGen reports positive Phase 2 FREEDOM2 data for PGN-EDODM1 in DM1; splicing correction 22.9% ex-outlier
PepGen Inc.
- 5 mg/kg cohort (n=8): mean splicing correction 7.3% overall, 22.9% excluding outlier; placebo 6.8%.
- No serious adverse events; all TEAEs mild/moderate; nausea most common; no renal toxicity.
- Middle finger vHOT showed improvement trend vs placebo worsening; returned to baseline at Week 16.
- 10 mg/kg cohort >50% enrolled; data expected 2H 2026; cash sufficient into 2H 2027.
- Mean muscle PGN-EDODM1 concentration 158 ng/g in 5 of 6 treated patients (one pending).