Key Highlights

  • Third quarter loss per share was USD 0.28 against an estimated USD 0.27 loss, with no Revenue reported.
  • Cash, equivalents and marketable securities were USD 104.8 million, and the accumulated Deficit was USD 416.1 million.
  • Nine-month net loss narrowed to USD 55.0 million from USD 71.3 million in the prior-year period.
  • Data from one FREEDOM2 cohort are scheduled for the first half of 2027, pending removal of a partial hold.

PepGen (NASDAQ:PEPG), a clinical-stage company developing treatments for myotonic dystrophy type 1, reported a third quarter 2026 loss of USD 0.28 per share, slightly wider than the USD 0.27 estimated loss. The company reported no revenue. Net loss for the nine months was USD 55.0 million, down from USD 71.3 million a year earlier.

General and administrative expenses rose USD 0.7 million to USD 5.9 million in the quarter. Cash, cash equivalents and marketable securities totaled USD 104.8 million at September 30, held in a checking account and U.S. treasury-backed Money Market funds. The accumulated deficit was USD 416.1 million.

PGN-EDODM1 Clinical Status

The company is developing its lead candidate for myotonic dystrophy type 1 using a mechanism and delivery approach it describes as different from more advanced programs. The filing states that it is conducting additional nonclinical mouse studies and remains in discussions with the FDA to respond to feedback, and it refers to a partial clinical hold in the United States.

In the Phase 1 FREEDOM study, adults received a single dose and were assessed at day 28 and week 16. Data from the 5 mg/kg, 10 mg/kg and 15 mg/kg cohorts were announced in 2025 showing the candidate was generally well tolerated, with drug-related adverse events mild or moderate and a dose-dependent mean splicing correction.

The company recently reported topline results from the 10 mg/kg cohort of the FREEDOM2 study, in which the candidate was generally well tolerated, with no serious adverse events and no signs of cumulative toxicity, and a dose-dependent splicing correction of CLCN1, a muscle chloride channel believed to drive myotonia, was observed.

Next Data and Regulatory Designations

Data from the 5 mg/kg cohort of FREEDOM2, which is fully enrolled, are scheduled for the first half of 2027. In the open-label extension, 19 participants have enrolled, with 100 doses given at 5 mg/kg and over 35 at 10 mg/kg. Data from FREEDOM2 and the extension are intended to support an end-of-Phase 2 meeting with the FDA in the second half of 2027, pending removal of the partial clinical hold in the United States.

The FDA has granted Orphan Drug designation and Fast Track Designation for the candidate in myotonic dystrophy type 1, and the European Medicines Agency has granted orphan medicinal product designation.

What to Follow

Resolution of the partial clinical hold, the first half of 2027 data and quarterly spending against the USD 104.8 million balance are the items to follow. The filing's legal proceedings item states that the company believes the final outcome of ordinary course matters is not anticipated to have a material adverse effect.