View source for FDA Pauses PepGen’s DMD Drug Trial In The US

<br>The therapy was being evaluated in ascending doses in the 25-week,  Read more multinational, double-blind, placebo-controlled CONNECT2-EDO51 trial. According to a PepGen news release, the decision was tied to questions arising during the regulatory review and does not impact ongoing studies outside the US, such as the mid-stage trial in the United Kingdom and the open-label CONNECT1-EDO51 trial enrolling in Canada. PepGen expected to open the phase 2 CONNECT2-EDO51 trial in the US by end of year. PepGen did not mention a specific reason for the decision; the FDA is expected to issue an official clinical hold letter to the company within 30 days of the notice. "We intend to work closely with the FDA to address their questions on our application to initiate CONNECT2 as expeditiously as possible," said Paul Streck, MD, MBA, executive vice president and head of research and development at PepGen. PGN-EDO51 is an investigational therapy that has not been approved for use in the US or any other country, though it was previously granted Orphan Drug and Rare Pediatric Disease designations, reflecting the unmet need in this patient population.<br><br><br><br>The therapy is designed to skip exon 51 of the dystrophin gene, which is mutated in approximately 13% of patients with DMD. Earlier this year, PepGen shared results from the first dose cohort (5 mg/kg) of the ongoing phase 2 CONNECT1-EDO51 study.2 The data revealed that PGN-EDO51 achieved greater levels of exon skipping and showed comparable or higher increases in total and muscle-adjusted dystrophin production compared with outcomes from earlier studies of other oligonucleotide therapies at similar PMO dose levels. This clinical hold marks a familiar challenge for PepGen. In May 2023, the company’s phase 1 trial of PGN-EDODM1 for myotonic dystrophy type 1 also faced a US regulatory hold. DMD is a rare genetic disorder that causes progressive muscle degeneration and weakness, affecting roughly 1 in 3600 male births worldwide.5 Over time, the condition leads to loss of mobility, respiratory complications, and heart issues. While there is no cure, treatments like physical therapy, medications, and emerging therapies aim to slow progression and improve quality of life. Newer therapies like PGN-EDO51 that target exon skipping are considered promising pathways to slow disease progression by restoring partial dystrophin function. 1. PepGen announces clinical hold in the U.S. IND application to initiate CONNECT2-EDO51 phase 2 study of PGN-EDO51 for Duchenne muscular dystrophy. 2. PepGen announces positive data from low-dose cohort of PGN-EDO51 in ongoing CONNECT1-EDO51 phase 2 clinical trial for treatment of Duchenne muscular dystrophy. 3. PepGen reports third quarter 2024 financial results and recent corporate highlights. 4. 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). 5. Duchenne muscular dystrophy (DMD).<br><br><br><br>If you’re serious about weightlifting, you’ve probably heard the term "progressive overload" before. But what exactly is it? Progressive overload is the steady increase of stress placed on your muscles during exercise over time. You can achieve this through various methods, including increasing weight, reps, or sets. Mind you; this method is not for the faint of heart. As the name suggests, it [https://www.britannica.com/search?query=demands demands] more and more from your muscles. The line that sits between what is within the limit and beyond the limit is extended. Curious? Keep reading to find out more about it. What is Progressive Overload? 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