Business
PepGen Inc. Announces FDA has Lifted the Clinical Hold on its Investigational New Drug Application for FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 for Myotonic Dystrophy Type 1 (DM1)
– Lifting of FDA hold allows FREEDOM-DM1 study to launch in the U.S. with target dose levels of 5 mg/kg, 10 mg/kg and 20 mg/kg – – Safety, transcript splicing
About this update from Pepgen Inc.
[{"type":"text","content":"– Lifting of FDA hold allows FREEDOM-DM1 study to launch in the U.S. with target dose levels of 5 mg/kg, 10 mg/kg and 20 mg/kg – – Safety, transcript splicing and clinical outcome measures data at 5 mg/kg PGN-EDODM1 dose level in patients from FREEDOM-DM1 clinical study expected in 2024 – – Continue to expect safety, muscle exon skipping and dystrophin data at 5 mg/kg PGN-EDO51 dose level in patients from CONNECT1-EDO51 clinical study in mid-2024 – – Cash runway expected to fund operations into 2025 – BOSTON, Oct. 12, 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 U.S. Food and Drug Administration (FDA) has lifted the full clinical hold and cleared the Company’s Investigational New Drug Application (IND) to initiate the FREEDOM-DM1 Phase 1 study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1) in the U.S. “We have worked closely with the FDA to resolve their questions expeditiously and are pleased that the clinical hold on our DM1 program in the United States has been lifted. Our novel PGN-EDODM1 approach targets the toxic RNA species responsible for the disease, and the strength of our Enhanced Delivery Oligonucleotide (EDO) safety preclinical package has enabled us to launch this study in both the U.S. and internationally at doses that we believe could provide a clinically meaningful benefit to patients. We are very pleased after review of our existing safety data that the FDA agreed with our proposed starting dose of 5 mg/kg, moving up to 10 mg/kg and 20 mg/kg,” said James McArthur, Ph.D., President and CEO of PepGen. As previously communicated, the Company opened FREEDOM-DM1 in Canada in September of this year. FREEDOM-DM1 is a randomized, double-blind, placebo-controlled, single ascending dose (SAD) study, designed to assess PGN-EDODM1 safety and tolerability, correction of mis-splicing of transcripts, and clinical functional outcome measures. Sites in both the U.S. and Canada will evaluate PGN-EDODM1 in 3 cohorts of 5 mg/kg, 10 mg/kg, and 20 mg/kg dose levels. The decision to advance to the next dose level will be contingent upon the evaluation of safety data derived from previous dose coh...