PepGen has announced that they have successfully dosed the first participant in their Phase 2 CONNECT1-EDO51 study, which will evaluate the safety and tolerability of PGN-EDO51, the company’s next-generation conjugated-PMO exon skipping therapy for the treatment of Duchenne muscular dystrophy patients amendable to exon 51 skipping.
Press Release Highlights:
- Preliminary data from the 5 mg/kg PGN-EDO51 dose level in the CONNECT1-EDO51 Phase 2 trial are expected in mid-2024, including initial safety, exon 51 skipping, and dystrophin protein production data.
- Phase 1 results following a single dose of 10 mg/kg of PGN-EDO51 in healthy volunteers demonstrated peak exon 51 skipping of 3.8%, with 100% of subjects demonstrating exon skipping.
“We are pleased to have dosed the first patient in our CONNECT1-EDO51 clinical trial, which marks another milestone in our commitment to developing therapies with the potential to truly improve the lives of people living with DMD. Based on the levels of exon skipping achieved following a single dose of PGN-EDO51 in our Phase 1 healthy volunteer trial, we are looking forward to our initial planned data readout in DMD patients at the 5 mg/kg PGN-EDO51 dose level for CONNECT1-EDO51 in the middle of 2024.”James McArthur, Ph.D., President and CEO of PepGen
The trial is expected to enroll 10 participants who are at least eight years of age and amenable to an exon 51 skipping approach. In addition to safety, oligonucleotide muscle concentrations, exon skipping and dystrophin protein production in muscle will be assessed at week 12 following four monthly doses of PGN-EDO51.
Clinical trials are being conducted in Canada. If interested in learning more, please contact [email protected]. Defeat Duchenne Canada will keep you up to date with the progress as the trial moves forward.