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PepGen Inc. Presents PGN-EDODM1 Preclinical Data Supporting the Company’s Enhanced Delivery Oligonucleotide Platform and PGN-EDODM1 Program at Two Medical Conferences
- EDO platform observed to drive 25-fold higher level of oligonucleotide delivery to myotube nuclei compared to “naked” oligonucleotide - - EDO technology
About this update from Pepgen Inc.
[{"type":"text","content":"- EDO platform observed to drive 25-fold higher level of oligonucleotide delivery to myotube nuclei compared to “naked” oligonucleotide - - EDO technology enabled delivery of therapeutic oligonucleotide to 72% of muscle nuclei in non-human primates - - PGN-EDODM1 corrected 99% of mis-splicing and reversed 99% of myotonia following multiple doses in Myotonic Dystrophy Type 1 (DM1) murine model - BOSTON, Sept. 06, 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 key highlights from the upcoming presentation of preclinical non-human primate (NHP) data supporting PepGen’s proprietary Enhanced Delivery Oligonucleotide (EDO) platform at the 6th Ottawa International Conference on Neuromuscular Disease and Biology (NMD) being held on September 7-9, 2023 in Ottawa, ON, Canada, and at the 2023 Myotonic Dystrophy Foundation (MDF) Annual Conference being held on September 7-9, 2023 in Washington, D.C. In a poster presentation titled “PGN-EDODM1 Nonclinical Data Demonstrate Mechanistic and Meaningful Activity for Potential Treatment of Myotonic Dystrophy Type 1” at MDF and “FREEDOM-DM1: Nonclinical Data Support the Phase 1 Study Design to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PGN-EDODM1 in Adults with Myotonic Dystrophy Type 1 (DM1)” at NMD, PepGen reported that the EDO technology enables up to 25-fold higher level of nuclear delivery of oligonucleotides in vitro to myotubes compared to “naked” unconjugated oligonucleotides. Additionally, the presentation reported that EDO technology was also shown to improve in vivo nuclear delivery, with 72% of skeletal muscle nuclei in NHPs positive for oligonucleotide following two doses at 30mg/kg. PepGen previously demonstrated that a single dose of 30 mg/kg of PGN-EDODM1 corrected myotonia in the DM1 HSALR mouse model as measured by electrophysiology. In the study reported here, a single dose of 30 mg/kg of PGN-EDODM1 reversed 76% of myotonia following a single dose, as measured by pinch test and corrected 68% of mis-splicing. Following this single-dose, 6 nM of PGN-EDODM1 was quantitated in muscle 28 days following dosing. With the EDO platform in humans, Pep...