other_material
confidence high
sentiment negative
materiality 0.85
PepGen discontinues DMD program after PGN-EDO51 misses dystrophin target; shifts focus to DM1
PepGen Inc.
- 10 mg/kg PGN-EDO51: total dystrophin only 0.59% of normal (mean increase 0.36%); exon skipping +3.5%.
- Company voluntarily discontinuing PGN-EDO51 and winding down all DMD research & development.
- DM1 program prioritized; FREEDOM-DM1 15 mg/kg data expected H2 2025, FREEDOM2-DM1 5 mg/kg data Q1 2026.
- PGN-EDO51 safety favorable; all treatment-related AEs mild, no serious adverse events.
- Prior DM1 data (10 mg/kg single dose) showed 29% mean mis-splicing correction at safety update.
item 7.01item 8.01item 9.01