Clinical TrialMay 21, 2026, 07:32 AM
PepGen Updates PGN-EDODM1 Clinical Data; FREEDOM2 5mg/kg Shows Splicing Correction
AI Summary
PepGen Inc. updated its Corporate Presentation for the 15th International Myotonic Dystrophy Consortium, highlighting promising clinical data for PGN-EDODM1 in Myotonic Dystrophy Type 1 (DM1). The FREEDOM single-ascending dose study demonstrated dose-dependent splicing correction, with 87.5% of participants showing improvement. The FREEDOM2 multiple-ascending dose 5 mg/kg cohort showed a favorable safety profile and mean splicing correction of 7.3%, with promising vHOT trends. The company anticipates reporting 10 mg/kg clinical results in 2H 2026 and 12.5 mg/kg results in 2027, supported by a cash runway into 2H 2027.
Key Highlights
- PepGen updated its Corporate Presentation for the 15th International Myotonic Dystrophy Consortium.
- PGN-EDODM1 demonstrated dose-dependent splicing correction in FREEDOM SAD trial, with 87.5% of participants showing improvement.
- FREEDOM SAD trial showed >20% splicing correction in a majority of DM1 patients at ">=10 mg/kg.
- FREEDOM2 5 mg/kg MAD cohort exhibited a favorable safety profile with all AEs mild or moderate.
- FREEDOM2 5 mg/kg MAD cohort showed mean splicing correction of 7.3% (22.9% excluding one outlier).
- Promising vHOT trends were observed in the PGN-EDODM1 treated group in FREEDOM2 5 mg/kg.
- Company expects FREEDOM2 10 mg/kg clinical results in 2H 2026 and 12.5 mg/kg results in 2027.
- PepGen maintains a strong cash runway into 2H 2027, through the FREEDOM2 12.5 mg/kg MAD readout.
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