BOSTON - PepGen Inc. (NASDAQ:PEPG), a clinical-stage biotechnology company, has received authorization from the UK Medicines & Healthcare products Regulatory Agency (MHRA) to begin the CONNECT2-EDO51 Phase 2 clinical trial for its Duchenne muscular dystrophy (DMD) treatment, PGN-EDO51.
The trial is set to enroll approximately 20 boys and young men who are six years or older and have DMD amenable to an exon 51-skipping approach.
The CONNECT2-EDO51 trial is a multinational, randomized, double-blind, placebo-controlled study that will administer seven doses of either PGN-EDO51 or a placebo at four-week intervals over 24 weeks. The trial aims to evaluate the safety, tolerability, and efficacy of the drug, including its ability to induce exon skipping and dystrophin production, which is crucial for muscle function.
PGN-EDO51 uses PepGen's proprietary Enhanced Delivery Oligonucleotide (EDO) technology to potentially restore the production of a truncated but functional dystrophin protein. This approach targets the root cause of DMD, a genetic disorder characterized by progressive muscle weakness that usually leads to loss of mobility, cardiac issues, and respiratory difficulties.
In preclinical studies, PGN-EDO51 has demonstrated promising results, showing higher levels of exon 51 skipping in non-human primates compared to other molecules. Additionally, in a Phase 1 study with healthy volunteers, PGN-EDO51 exhibited a six-fold increase in mean exon 51 skipping compared to a naked oligonucleotide after a single dose.
James McArthur, Ph.D., President, and CEO of PepGen, expressed optimism about the potential of PGN-EDO51 to offer a transformative treatment for those living with DMD. The company is also conducting the CONNECT1-EDO51 trial, and together, these studies could potentially support accelerated approval of the drug, subject to alignment with regulators.
PepGen's focus is on advancing oligonucleotide therapies to address severe neuromuscular and neurological diseases. While the company has forward-looking statements regarding the anticipated benefits of PGN-EDO51 and its EDO platform, it acknowledges the risks and uncertainties inherent in clinical development.
This news article is based on a press release statement from PepGen Inc.
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