PepGen Received Safety Board Backing for Higher Myotonic Dystrophy Doses
PepGen said seven of eight participants had completed dosing in its 10 mg/kg trial group and expects results in November.
PepGen Inc. (PEPG), a clinical-stage biotechnology company, received an independent safety board's recommendation to test its myotonic dystrophy treatment at the highest planned dose in its Phase 2 study. The development carried its PGN-EDODM1 program beyond the initial dosing reported in March and full enrollment announced in May, toward another escalation in the repeat-dose trial.
The progression centered on FREEDOM2, a randomized, placebo-controlled study in myotonic dystrophy type 1, or DM1. In March, four of eight participants in the 10 mg/kg group had received up to two doses; by May, enrollment was complete. The August update placed that group near the end of dosing, while PepGen said it was preparing to begin enrollment at the next dose shortly.
The independent Data and Safety Monitoring Board recommended moving into the 12.5 mg/kg group after reviewing available safety data from the ongoing trial and its open-label extension. That was the third and highest dose group. Chief Executive James McArthur said the recommendation reflected an encouraging safety profile following several months of repeat dosing; the company had not yet reported the middle-dose group's results.
PepGen expects the 10 mg/kg results in November, specifying a month within its previous second-half 2026 forecast. The company also expects results from the highest-dose group in the first half of 2027. Those reports concern repeated administration, following the earlier FREEDOM trial that evaluated single doses of the drug. McArthur said repeat dosing had the potential to build on that earlier clinical work.
The safety board also approved increasing the open-label extension dose from 5 mg/kg to 10 mg/kg. Enrollment in that study totaled 16 participants, including six participants from FREEDOM2's initial dose group. PepGen expects an extension-study update by early January, adding another scheduled report alongside the main trial's dose-group results.
The company described PGN-EDODM1 as an oligonucleotide treatment designed to restore the normal function of MBNL1, a protein involved in RNA splicing. Its approach targeted abnormal repeat sequences in DMPK transcripts that trap the protein. PepGen said it believed the approach may allow those transcripts to continue their normal cellular function while releasing MBNL1, without relying on transcript degradation. The drug remained investigational and had not been approved in any country.
The clinical progress followed a regulatory constraint disclosed in the preceding updates. In May, the U.S. Food and Drug Administration placed FREEDOM2 on partial clinical hold over questions concerning previously submitted preclinical pharmacology and toxicology studies. The study's timing had not been affected. PepGen's August presentation continued to identify release of that hold among regulatory risks.
Research and development spending fell to $12.5 million in the second quarter from $18.4 million a year earlier. General and administrative expenses were $6.4 million, bringing total operating expenses to approximately $18.9 million. The research-spending reduction was not attributed to a specific operating change. Research remained the larger of the company's two reported expense categories.
The quarterly net loss narrowed to $17.8 million from $23.1 million a year earlier. The loss was $0.26 a share, and interest income totaled approximately $1.1 million. PepGen had about 69.3 million shares outstanding at the end of June.
Cash, cash equivalents and marketable securities stood at $117.2 million on June 30. Based on currently planned operations, that balance is sufficient to fund operations into the final three months of 2027, a more specific forecast than the previous second-half 2027 wording. Pending results from FREEDOM2's two higher-dose groups, the company plans an end-of-Phase-2 meeting with regulators to discuss its proposed registrational program.
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