Clinical trialsCompany release

PepGen reports 46.5% mean splicing correction in 10 mg/kg myotonic dystrophy cohort

🇺🇸 PepGen Inc. (PEPG) · Tue 10 Nov 2026

Topline Phase 2 data for PGN-EDODM1 in myotonic dystrophy type 1 show improved myotonia and a generally well-tolerated profile, with higher-dose results due in H1 2027.

PepGen has announced positive topline results from the 10 mg/kg cohort of FREEDOM2-DM1, its Phase 2 trial of PGN-EDODM1 in myotonic dystrophy type 1 (DM1). The company reported the data on 10 November 2026.

After four monthly doses, mean splicing correction in muscle reached 46.5%. Myotonia, measured by video Hand Opening Time, improved by 52.7% at Week 16. PepGen also reported gains in grip strength and mobility tests, although the summary available does not give figures for those measures.

On safety, PepGen described PGN-EDODM1 as generally well tolerated, with no serious or severe adverse events. Mild renal events did occur. The details of those events were not available in the material reviewed.

Results from the 12.5 mg/kg cohort are expected in the first half of 2027.

What it means for your team

  • The mild renal events are worth watching: renal safety is likely to draw attention as higher-dose data emerge from the 12.5 mg/kg cohort.
  • The data are topline company-reported results; full datasets, including cohort size and the handling of the functional endpoints, were not available in the material reviewed.
  • Regulatory and market-access teams tracking DM1 should note the 12.5 mg/kg readout in H1 2027 as the next data point.

Company's press release (SEC filing): PepGen Inc. ↗

Article written by VigiEye from the official source (PepGen Inc.). Claims are the company's own. AI-assisted; check the source before acting.

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