PepGen Inc. announced topline results from the 5 mg/kg cohort of its Phase 2 FREEDOM2-DM1 trial, showing PGN-EDODM1 was generally well-tolerated with favorable splicing and vHOT data, supporting the ongoing 10 mg/kg cohort.
Key takeaways
PGN-EDODM1 at 5 mg/kg was generally well-tolerated with all adverse events mild or moderate and no serious adverse events reported.
Mean splicing correction was 7.3% for the treatment group versus 6.8% for placebo; excluding one outlier, the treatment group mean was 22.9%.
Promising trends were observed in video hand opening test (vHOT) in the PGN-EDODM1 treated group.
The company is on track to report clinical data from the 10 mg/kg multiple dose cohort in the second half of 2026.
The company expects its cash to fund operations into the second half of 2027.