PepGen has received a No Objection Letter (NOL) for its Clinical Trial Application (CTA) from Health Canada for its Phase 2 CONNECT1-EDO51 study to initiate an open-label, multiple ascending dose (MAD) clinical trial of PGN-EDO51 in patients with Duchenne muscular dystrophy (DMD) amenable to an exon 51 skipping approach.

“Today is the next step in our development of PGN-EDO51, a potentially transformative treatment candidate for people living with DMD. Building upon the encouraging safety, tolerability, and exon skipping data from our Phase 1 healthy volunteer (HV) study reported last year showing promising tolerability and exon-skipping activity, we are pleased to continue our work with the DMD community to develop a therapy that we hope will produce meaningful levels of a functional, skipped dystrophin protein.

In addition to our most advanced program, PGN-EDO51, we eagerly anticipate providing the community with updates on our progress to develop transformational therapies for myotonic dystrophy type one (DM1), and other exon skippable mutations for people living with DMD, including 53, 45 and 44. We are leveraging the power of our EDO technology to work to change the future for people living with these devastating diseases.”

James McArthur, Ph.D., President and CEO of PepGen

“PGN-EDO51 exhibited the highest levels of oligonucleotide delivery and exon 51 skipping in a clinical study following a single dose of 5, 10 and 15 mg/kg in healthy volunteers when compared to publicly available clinical data for other exon 51 skipping approaches.

At these dose levels, the majority of treatment emergent adverse events (TEAE) were assessed as mild and resolved without any intervention. Looking ahead, and based on our nonclinical data, we believe CONNECT1-EDO51 may support a differentiated profile for PGN-EDO51 relative to other investigational and approved therapies based on previously observed meaningful and durable data on dystrophin production, as well as clinical assessments. In summary, we believe PGN-EDO51 offers the hope of a more complete correction of the disease pathology and look forward to sharing updates on the progress of our planned trial.”

Michelle Mellion, M.D., SVP and Head of Clinical Development at PepGen

PepGen plans to evaluate PGN-EDO51 in approximately 3 cohorts of ambulatory and non-ambulatory boys and young men in the CONNECT1-EDO51 study, an open-label, MAD clinical trial starting at 5 mg/kg, with plans to escalate to 10 mg/kg and potentially other doses following Drug Safety Monitoring Board (DSMB) review. The Phase 2 study will evaluate the safety, tolerability, dystrophin levels, pharmacokinetics and clinical assessments.

Defeat Duchenne Canada held a webinar with PepGen on May 18, 2023. Watch the recording and learn more about this exciting announcement: