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PepGen Presents Clinical and Nonclinical Data at the 2023 Annual Muscular Dystrophy Association Clinical and Scientific Conference
- In NHP, four monthly doses of 20 mg/kg of PGN-EDO51 resulted in 34.9% exon skipped transcripts in biceps; a 14-fold increase over the 2.5% exon 51 skipped
About this update from Pepgen Inc.
[{"type":"text","content":"- In NHP, four monthly doses of 20 mg/kg of PGN-EDO51 resulted in 34.9% exon skipped transcripts in biceps; a 14-fold increase over the 2.5% exon 51 skipped transcripts observed after a single dose – - Following on the EDO51 Phase 1 study, we anticipate initiating in the first half of 2023 the CONNECT1-EDO51 Study in Duchenne muscular dystrophy (DMD) patients, a Phase 2 open-label multiple ascending dose (MAD) study in Canada. We expect to report top-line tolerability, exon skipping and dystrophin data in 2024 – - In parallel, we anticipate initiating in the second half of 2023 the CONNECT2-EDO51 Study in DMD patients, a Phase 2 global, randomized placebo-controlled MAD study that is designed to potentially provide an accelerated path to approval for EDO51 – - In vitro studies of the clinical candidate PGN-EDODM1 showed a dose-dependent 54% reduction in toxic nuclear foci per nuclei, liberated MBNL1 from toxic foci and produced >68% correction of downstream transcript mis-splicing events in myotonic dystrophy type 1 (DM1) patient myoblasts – - We anticipate initiating in the first half of 2023 the FREEDOM-DM1 Study in DM1 patients, a Phase 1 global, placebo-controlled randomized single ascending dose (SAD) study for EDODM1 – BOSTON, March 22, 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, will be presenting today 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 (mouse equivalent of PGN-EDO51) resulted in 52.5% exon 23 skipping and dystrophin production of 22.5% that was sustained for up to four weeks. Exon skipping increased considerably with repeat dosing of 30 mg/kg PGN-EDO23 once every four weeks for a total of four doses achieving 91.5% exon 23 skipping measured by RT-PCT and production of 82.3% normal levels of dystrophin in biceps. Even more encouragi...