PepGen to hold trials to assess safety and efficacy of repeated PGN-EDO51 doses

PepGen will be presenting nonclinical and clinical data of its Enhanced Oligonucleotide Delivery EDO platform at the Muscular Dystrophy Association MDA Clinical and Scientific Conference in Dallas, Texas.PepGen’s preclinical data of PGN-EDO51, the company’s lead product candidate for the treatment of people living DMD whose mutations are amenable to an exon 51 skipping approach, showed in the mdx mouse model that a single, 30 mg/kg dose of PGN-EDO23 resulted in 52.5% exon 23 skipping and dystrophin production of 22.5% that was sustained for up to four weeks…"Our preclinical and Phase 1 clinical data that is being presented at MDA highlight the observed ability of PepGen’s EDO technology platform to drive encouraging levels of exon 51 skipped transcripts following a single-dose in NHPs and in humans. Our repeat-dose NHP data we will present here further support the potential stacking of exon 51 skipped transcripts with monthly PGN-EDO51 dosing," stated Jaya Goyal, PhD, Executive Vice President, Research and Preclinical Development at PepGen. "We look forward to sharing our PGN-EDO51 clinical development plan in the near future." Based on the encouraging nonclinical data, PepGen is planning to initiate two clinical trials to assess the safety and efficacy of repeat doses of PGN-EDO51 in young men living with DMD. CONNECT1-EDO51 is a Phase 2 open-label MAD clinical trial to be initiated in Canada in the first half of 2023 that is expected to report dystrophin protein data in 2024. Additionally, CONNECT2-EDO51, a Phase 2, global, randomized placebo-controlled clinical trial is expected to be initiated in the second half of 2023 to support a potential accelerated approval pathway, subject to alignment with regulators. The corresponding poster presentation and slides referenced during the oral presentations are available on the Events and Presentations page in the Investor Relations section of the company’s website.