PepGen Q1 2026 (May 14): FREEDOM2-DM1 5 mg/kg PGN-EDODM1 Cohort Topline — 7.3% Mean Splicing Correction in n=6 Myotonic Dystrophy Patients
PepGen reported Q1 2026 results on May 14 with topline data from the Phase 2 FREEDOM2-DM1 5 mg/kg multiple-ascending-dose cohort. PGN-EDODM1 — the company's peptide-conjugated phosphorodiamidate morpholino oligomer for myotonic dystrophy type 1 (DM1) — produced a mean splicing correction of 7.3% in 6 treated patients vs 6.8% in 2 placebo patients, with no serious adverse events and mainly mild treatment-emergent adverse events. The 10 mg/kg cohort is fully enrolled with data expected H2 2026; the 12.5 mg/kg cohort readout is expected in 2027. PepGen received regulatory clearance to initiate FREEDOM2 sites in South Korea, Australia, and New Zealand to add to existing sites in Canada and the UK. Q1 net loss of $17.8M (vs $30.2M a year earlier); $132.3M cash supports operations through the 12.5 mg/kg MAD readout into 2H 2027. The DM1 program is PepGen's lead clinical asset following the April DMD program discontinuation; it competes with Vertex/Entrada's VX-670 cyclic peptide-oligonucleotide (GALILEO Phase 1/2, H2 2026 readout).