PepGen's DM1 drug shows myotonia gains at 10 mg/kg; higher-dose data due H1 2027

PepGen has given investors and prospective partners a first dose-level view of its lead myotonic dystrophy type 1 (DM1) candidate, PGN-EDODM1. On 10 November 2026 it reported results from the 10 mg/kg cohort of the Phase 2 FREEDOM2-DM1 study. The company has not disclosed financial terms, partnering plans or timelines beyond the next readout, so the commercial implications remain unquantified.

Muscle biopsies showed that, on average, 46.5% of the targeted splicing abnormality was corrected after four monthly doses. Splicing disruption is the molecular hallmark of DM1. On the clinical side, myotonia, the delayed muscle relaxation typical of the disease, eased by 52.7% at Week 16 on a video-based hand-opening measure. PepGen also said grip strength and mobility assessments improved, but the material available gives no figures for them.

On safety, the company reported no serious or severe adverse events, with mild renal events seen in the cohort. Details of those events, such as how many patients were affected, were not available. Neither was the number of patients in the cohort, or how the functional endpoints were analysed. All of the figures are company-reported topline data rather than a full dataset.

A 12.5 mg/kg cohort is expected to report in the first half of 2027. As context, DM1 is a rare inherited neuromuscular disease, so the second dose level, and any renal findings alongside it, will help show how the programme's efficacy and safety profile develops.