PepGen Inc. (NASDAQ:PEPG) reported third-quarter 2026 financial results alongside new Phase 2 clinical data for PGN-EDODM1, its investigational treatment for myotonic dystrophy type 1 (DM1). The biotechnology company ended September with $104.8 million in cash, cash equivalents and marketable securities, which it expects will fund operations into 2028.
The company also announced results from the 10 mg/kg dose cohort of its ongoing FREEDOM2-DM1 study, reporting improvements in RNA splicing correction and several functional assessments.
Phase 2 Trial Reports 46.5% Mean Splicing Correction
PepGen reported mean splicing correction of 46.5% in the 10 mg/kg cohort, which it described as the highest reported in a multiple-dose DM1 study.
Five of six treated participants demonstrated splicing improvements exceeding 20%, while four recorded improvements above 50% at Week 13.
The company also reported improvements across assessments of hand opening time, grip strength, walking speed and mobility.
PGN-EDODM1 was generally well tolerated at this dose, with no serious or severe adverse events, treatment discontinuations or dose-limiting toxicities reported.
Higher-Dose Results Expected in First Half of 2027
Following a safety review, an independent monitoring board recommended progression to the 12.5 mg/kg cohort, which is now fully enrolled.
Results from this cohort are expected during the first half of 2027.
PepGen also plans to report additional safety and clinical data from its FREEDOM open-label extension study in January 2027.
The company continues to address an FDA partial clinical hold imposed in March 2026 following nonclinical findings in mice. A potential end-of-Phase 2 regulatory meeting is targeted for the second half of 2027, subject to removal of the hold.
Third-Quarter Net Loss Widens to $19.4 Million
PepGen reported a third-quarter net loss of $19.4 million, or $0.28 per share, compared with $18.0 million, or $0.52 per share, in the corresponding period of 2025.
Research and development expenses increased to $14.5 million from $13.4 million, while general and administrative expenses rose to $5.9 million from $5.2 million.
Cash, cash equivalents and marketable securities totalled $104.8 million at September 30, 2026.
Chief Executive Officer James McArthur said the company had advanced its clinical programme while maintaining sufficient funding for its planned operations.
PepGen expects further clinical updates in January and the first half of 2027 as it continues evaluating PGN-EDODM1 for DM1, a rare genetic disease associated with progressive muscle impairment and other systemic complications.