PepGen has announced that its Phase 2 trial of PGN-EDODM1 for myotonic dystrophy type 1 (DM1) will progress to the next stage following a positive safety review.
Phase 2 trial of PGN-EDODM1 for DM1 advances
11 August 2026
After reviewing safety data from participants in the study, the Data and Safety Monitoring Board (DSMB) recommended that the trial continue. The DSMB also supported progression of the open-label extension study, where participants can continue receiving PGN-EDODM1 after completing the main trial.
According to PepGen, 16 participants have enrolled in the extension study so far.
PGN-EDODM1 is designed to target the underlying cause of DM1 by preventing toxic RNA from disrupting normal cell function.
PepGen expects to report further results from the trial later this year, with additional data expected in 2027.
PGN-EDODM1 remains an experimental treatment and further research is needed to determine its safety and effectiveness.
