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PepGen reports 46.5% splicing correction in DM1 trial as FDA partial hold persists

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

Phase 2 FREEDOM2-DM1 data for PGN-EDODM1 show no serious adverse events at 10 mg/kg, but the March 2026 FDA partial clinical hold is still unresolved.

PepGen reported topline Phase 2 FREEDOM2-DM1 results for PGN-EDODM1 in myotonic dystrophy type 1 on 10 November 2026, alongside its third-quarter financial results. The 10 mg/kg cohort showed mean splicing correction of 46.5%, and no serious adverse events were reported.

A data safety monitoring board (DSMB) has cleared the 12.5 mg/kg cohort, which is fully enrolled. The company's update did not change the regulatory position: the FDA partial clinical hold imposed in March 2026 remains in place.

Separately, the FDA selected the programme in October 2026 for its CMC Development and Readiness Pilot. This is a chemistry, manufacturing and controls initiative, and the selection points to regulatory attention on the product's manufacturing readiness as well as its clinical data.

PepGen said cash of $104.8M is expected to fund operations into 2028.

What it means for your team

  • The partial clinical hold is still unresolved, so the timing of any wider dosing or progression to later-stage development remains tied to FDA decisions.
  • DSMB clearance of the fully enrolled 12.5 mg/kg cohort adds further dose-level safety and splicing data, which regulators will weigh in assessing the hold.
  • Inclusion in the FDA CMC Development and Readiness Pilot is relevant for quality and CMC teams tracking early engagement on manufacturing readiness.
  • The cash runway into 2028 reduces near-term financing risk for the programme while the hold is addressed.

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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