PepGen has announced that an independent Data and Safety Monitoring Board (DSMB) has approved the progression to the highest dosage cohort in its Phase II FREEDOM2-DM1 study of PGN-EDODM1.
The trial, which evaluates the therapy on participants with myotonic dystrophy type 1 (DM1), will now proceed to the 12.5mg/kg dosage level without any alterations to the study protocol.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
The decision also includes a dose increase in the open-label extension (OLE) study to 10mg/kg from 5mg/kg.
Safety analysis of the fully enrolled 10mg/kg cohort and the OLE prompted these recommendations.
Currently, six out of eight participants from the 5mg/kg FREEDOM2 cohort have opted to join the OLE study, increasing total enrolment to 16 participants.
PGN-EDODM1 has been generally well-tolerated in both the FREEDOM2 study and the OLE, with no serious adverse events or dose-limiting toxicities noted.
Repeated doses at 5mg/kg and 10mg/kg have shown no signs of cumulative toxicity, and no participants have discontinued treatment in these studies.
PepGen aims to report findings from the 10mg/kg FREEDOM2 cohort in November, while data from the upcoming 12.5mg/kg cohort is expected in the first half of 2027.
PepGen president and CEO James McArthur said: “The DSMB’s recommendation to advance FREEDOM2 into the highest planned dose level in the study and escalate dosing in the OLE supports the encouraging safety profile of PGN-EDODM1 following multiple months of treatment.
“In the FREEDOM2 study, seven of eight participants in the 10mg/kg cohort have now completed dosing. We look forward to reporting additional safety, splicing and functional data from FREEDOM2 as we continue to evaluate the potential of PGN-EDODM1 to address the root cause of disease across multiple organ systems.”
The company plans to discuss further development plans with regulators in an end of Phase II meeting. An update from the OLE study is anticipated by early January.
PGN-EDODM1 has received both orphan drug and fast track designations from the US Food and Drug Administration for treating DM1. Additionally, the European Medicines Agency has recently awarded orphan designation for the treatment.
In September 2025, PepGen’s stock increased by more than 120% after its investigational DM1 candidate reported the highest mean splicing correction in a Phase I trial.
