PepGen Inc., a clinical-stage biotech company that focuses on creating advanced oligonucleotide therapies for severe neuromuscular and neurological diseases, recently shared positive clinical results from its FREEDOM-DM1 Phase 1 trial. The data from the 15 mg/kg dose group in their single ascending dose study showed an average splicing correction of 53.7 percent in patients with myotonic dystrophy type 1. This level of correction is much higher than what has been reported before for this condition.

Health Technology Insights: TIME Names CancerIQ as One of World’s Top HealthTech Companies

Paul Streck, M.D., M.B.A., Executive Vice President of Research and Development at PepGen, expressed excitement about the results. He explained that since mis-splicing causes DM1, achieving high splicing correction has the potential to address the root molecular problems and may lead to functional improvements such as reduced myotonia and muscle weakness in future studies with repeated doses.

Health Technology Insights: Oculis to Present Positive Phase 2 ACUITY Trial Results at ECTRIMS 2025

James McArthur, Ph.D., President and CEO of PepGen, noted that these findings build on earlier encouraging results seen in lower dose groups at 5 mg/kg and 10 mg/kg. He highlighted that every patient in the 15 mg/kg group showed measurable splicing correction after treatment. McArthur also mentioned that data from the ongoing multiple ascending dose study, FREEDOM2, are expected in early 2026.

In the 15 mg/kg group of eight patients, six were evaluated for splicing correction 28 days after receiving the treatment, and all showed improvement. Muscle tissue concentrations of the drug increased with higher doses across the 5 mg/kg, 10 mg/kg, and 15 mg/kg groups. Safety observations indicated that PGN-EDODM1 was generally well-tolerated, with no serious adverse events linked to the treatment. Most side effects were mild or moderate and temporary. There were no significant electrolyte disturbances, and any kidney-related effects were short-term and resolved without any intervention. One mild elevation in a kidney biomarker met dose-limiting toxicity criteria but quickly resolved, and the patient continued in the study.

PepGen plans to report results from the 5 mg/kg cohort of the FREEDOM2 multiple ascending dose study in the first quarter of 2026 and will start dosing the 10 mg/kg group during the same time period.

Health Technology Insights: XRHealth Launches XR CareCart VR Rehab Station for Clinics

To participate in our interviews, please write to our HealthTech Media Room at sudipto@intentamplify.com