BOSTON — PepGen reported a narrower second-quarter loss as the biotechnology company advanced its Phase 2 trial of PGN-EDODM1 in patients with myotonic dystrophy type 1.
The company posted a net loss of $17.8 million, or 26 cents per share, for the quarter ended June 30, compared with a loss of $23.1 million, or 70 cents per share, a year earlier.
Research and development expenses declined to $12.5 million from $18.4 million. General and administrative expenses increased to $6.4 million from $5.5 million.
PepGen ended the quarter with $117.2 million in cash, cash equivalents and marketable securities. The company expects its existing resources to fund operations into the fourth quarter of 2027.
The company said the 10-milligram-per-kilogram cohort in its Phase 2 FREEDOM2-DM1 trial is fully enrolled, with seven of eight participants having completed dosing. Data from the cohort are expected in November.
An independent data and safety monitoring board reviewed available safety findings and recommended advancing the study to its highest planned dose of 12.5 milligrams per kilogram.
The board also approved increasing the dose in the trial’s open-label extension from 5 milligrams per kilogram to 10 milligrams per kilogram.
“During the second quarter, our team maintained a strong focus on execution as we advanced FREEDOM2 and continued preparing for our next clinical data readout,” said James McArthur, PhD, President and Chief Executive Officer of PepGen. “The DSMB’s recommendation to dose escalate in both FREEDOM2 and the open-label extension reflects the encouraging safety profile of PGN-EDODM1 following several months of repeat dosing. We look forward to initiating enrollment in the 12.5 mg/kg cohort shortly and sharing data from the 10 mg/kg cohort in November. We remain confident that repeat dosing has the potential to build on the clinical foundation established in the FREEDOM single-ascending dose trial as we continue evaluating the safety and benefits of PGN-EDODM1 for individuals living with DM1.”
Results from the 12.5-milligram-per-kilogram cohort are expected in the first half of 2027. PepGen said it plans to meet with regulators to discuss a potential registrational program after reviewing results from the two higher-dose cohorts.
The company expects to provide an update from the open-label extension by early January. Six of the eight participants from the 5-milligram-per-kilogram FREEDOM2 cohort elected to enter the extension, bringing total enrollment to 16 participants.
PGN-EDODM1 is an experimental oligonucleotide therapy designed to address the underlying RNA-splicing abnormalities associated with myotonic dystrophy type 1.
The candidate uses PepGen’s Enhanced Delivery Oligonucleotide platform to deliver an oligonucleotide intended to bind to expanded CUG repeats in DMPK transcripts and release MBNL1, a protein involved in normal RNA splicing.
PepGen believes the approach could correct downstream mis-splicing while allowing the DMPK transcripts to continue performing their normal cellular function.
The U.S. Food and Drug Administration has granted PGN-EDODM1 orphan drug and fast-track designations. The European Medicines Agency has also granted the candidate orphan designation.
PepGen presented clinical and natural-history findings from the program at the International Myotonic Dystrophy Consortium meeting in May.
