PepGen (NASDAQ:PEPG) has announced initial positive data from the five and ten mg/kg dose cohorts in its ongoing FREEDOM-DM1 Phase 1 study investigating PGN-EDODM1 in myotonic dystrophy type 1 (DM1).
PeoGen reports that these results include a robust splicing correction of 29.1% following a single dose of PGN-EDODM1 at ten mg/kg.
James McArthur, Ph.D., president and CEO of PepGen, said, “These results far exceed our expectations for splicing correction following a single dose of PGN-EDODM1. Mis-splicing is the underlying cause of DM1 pathology, and we believe the mean splicing correction observed at 28 days following a single dose of PGN-EDODM1 at ten mg/kg in the FREEDOM clinical trial surpasses those reported to date in multi-dose clinical trials of up to nine months in duration in patients with DM1. We believe this is a strong indicator of our EDO technology’s potential to deliver therapeutic oligonucleotides to the nucleus and PGN-EDODM1’s potential to address the underlying cause of disease.”
Dr. McArthur added, “We believe these results provide initial validation of PGN-EDODM1’s ability to selectively bind the pathogenic CUG-repeat DMPK RNA and we look forward to evaluating PGN-EDODM1 with more doses over longer time periods in our FREEDOM2-DM1 multiple ascending dose study. Based on these initial results, we aim to build on the significant correction of mis-splicing observed in this single-dose study to potentially provide improved functional benefit for patients who currently have no available approved therapeutic options.”
