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PepGen Announces Clearance by Health Canada of CTA for PGN-EDO51 to Begin the Phase 2 Clinical Trial, CONNECT1-EDO51, for the Treatment of Duchenne Muscular Dystrophy
- CONNECT1-EDO51 will assess dystrophin levels, exon skipping data and safety data following multiple doses of PGN-EDO51 in patients with mutations amenable
About this update from Pepgen Inc.
[{"type":"text","content":"- CONNECT1-EDO51 will assess dystrophin levels, exon skipping data and safety data following multiple doses of PGN-EDO51 in patients with mutations amenable to skipping exon 51 - - Initial data readout is expected mid-2024 - BOSTON, May 18, 2023 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the company 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. The Company expects to begin dosing patients with PGN-EDO51 in 2H 2023. “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,” said James McArthur, Ph.D., President and CEO of PepGen. “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.” “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,” said Michelle Mellion, M.D., SVP and Head of Clinical Development at PepGen. “At these dose levels, the majority of treatment emergent adverse events (TEAE) were assessed as mild and resolved without any interv...