PepGen, a biotech company developing innovative oligonucleotide therapies for severe neuromuscular and neurological diseases, has received approval from the UK regulator (MHRA) to launch the Phase 2 CONNECT2-EDO51 trial. This trial will evaluate PGN-EDO51, a potential treatment for Duchenne muscular dystrophy (DMD) patients who can benefit from exon 51 skipping.
“We are excited to take the next step forward in our development of PGN-EDO51, which we believe to be a potentially transformative investigational candidate for people living with DMD and are pleased the MHRA authorized our CTA. We believe this study, together with the data generated in our ongoing CONNECT1-EDO51 trial, could potentially support accelerated approval of EDO51, subject to alignment with regulators,” said James McArthur, Ph.D., President and CEO of PepGen. “We are grateful to continue our work with the DMD community to develop this therapy.”
The CONNECT2-EDO51 trial remains a multinational, randomised, double-blind, placebo-controlled, multiple ascending dose (MAD) study. It aims to enrol approximately 20 ambulatory and non-ambulatory boys and young men, at least six years old, diagnosed with DMD amenable to exon 51 skipping. Participants will be assigned treatment through randomization, receiving either PGN-EDO51 or a placebo for a treatment period of 24 weeks. Doses will be administered every four weeks, with an initial escalation from 5 mg/kg to 10 mg/kg, potentially reaching higher levels based on safety data from the preceding participant cohorts within the trial. A baseline muscle biopsy will be acquired from each participant, followed by another at week 25. The primary objectives of the trial are to evaluate exon skipping efficiency, dystrophin protein production, and the overall safety and tolerability of PGN-EDO51. All participants will be offered the opportunity to participate in an open-label extension.
Would you like to know more about Duchenne muscular dystrophy? Increase your knowledge and understanding of Duchenne with our bite-sized science video series.
Section 1 – Facts about Duchenne muscular dystrophy
Section 2 – Signs and Symptoms of Duchenne muscular dystrophy
Section 3 – Diagnosis of Duchenne muscular dystrophy
Section 4 – Crucial Genetic Terminology
Section 5 – Genetics – Blueprint of Duchenne muscular dystrophy
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