A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study of PGN-EDO51 With a Long-Term Extension in Participants With Duchenne Muscular Dystrophy Amenable to Exon 51-Skipping Treatment (CONNECT2-EDO51)
Latest Information Update: 30 Jun 2025
At a glance
- Drugs PGN-EDO51 (Primary)
- Indications Duchenne muscular dystrophy
- Focus Adverse reactions; Registrational; Therapeutic Use
- Acronyms CONNECT2-EDO51
- Sponsors PEPGENE
Most Recent Events
- 24 Jun 2025 Status changed from suspended to withdrawn prior to enrolment because Global PGN-EDO51 development voluntarily discontinued by Sponsor.
- 08 May 2025 According to a Pepgen Corporation media release, dystrophin production data from 10 mg/kg cohort is expected in Q3 2025.
- 18 Mar 2025 Planned End Date changed from 30 Apr 2029 to 30 Apr 2030.