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PepGen Inc. Announces Clearance of CTA by Health Canada to Begin the FREEDOM-DM1 Phase 1 Study of PGN-EDODM1 in Patients with Myotonic Dystrophy Type 1
The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to PGN-EDODM1 for the treatment of Myotonic Dystrophy Type 1 (DM1) BOSTON, Sept.
About this update from Pepgen Inc.
[{"type":"text","content":"The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to PGN-EDODM1 for the treatment of Myotonic Dystrophy Type 1 (DM1)\nBOSTON, 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 it has received a No Objection Letter (NOL) from Health Canada for its Clinical Trial Application (CTA) to initiate the FREEDOM-DM1 Phase 1 study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). In addition, the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to PGN-EDODM1 for the treatment of myotonic dystrophy type 1. The FREEDOM-DM1 clinical trial is a randomized, placebo-controlled, single ascending dose (SAD) study, intended to enroll approximately 24 adult patients with DM1 to evaluate the safety and tolerability of PGN-EDODM1. In addition to safety, oligonucleotide muscle concentrations and PGN-EDODM1 correction of mis-splicing of transcripts, clinical functional outcomes impacted in DM1 will also be assessed 28 days and at later time points following a single dose of PGN-EDODM1. The approved dose escalation from the initial 5 mg/kg dose cohort will be to 10 mg/kg and then to 20 mg/kg. The decision to escalate to the next dose will be determined based on clinical data from the prior dose cohorts “We are very pleased to have received Health Canada's clearance of PepGen’s latest CTA for PGN-EDODM1, which allows PepGen to continue striving toward developing truly transformative medicines that have the potential to bring clinically meaningful outcomes to individuals with neuromuscular and neurological diseases. We expect to report initial results from this study in 2024,” said James McArthur, Ph.D., President and CEO of PepGen. “People living with DM1 have a significant unmet medical need with currently no approved disease-modifying therapies, and the initiation of this trial plus the FDA’s granting of orphan drug designation for PGN-EDODM1 signifies another important step toward addressing that need.” Under the Orphan Drug Act, the FDA may grant orphan drug designation to a drug product intended to treat a rare disease or condition, which is generally a disease or co...