PepGen Pauses DMD Trial in UK, Awaits Data from Canadian Study

PepGen, a biotech company focusing on muscular dystrophy therapies, has announced a temporary halt to the UK arm of its phase 2 clinical trial for Duchenne muscular dystrophy (DMD). This decision comes as the company navigates regulatory challenges and awaits crucial data from an ongoing study in Canada.

Trial Pauses and Regulatory Hurdles

The CONNECT2-EDO51 trial, designed to evaluate PepGen's exon-skipping therapy PGN-EDO51 over a 25-week period in DMD patients, has faced setbacks on multiple fronts. In December, the U.S. Food and Drug Administration (FDA) placed the trial on hold, prompting PepGen to reassess its strategy. The company subsequently revealed that two patients in the 10-mg/kg cohort had experienced asymptomatic magnesium deficiency, although their levels returned to baseline after oral supplementation.

Now, PepGen has opted to pause the UK arm of the study while it awaits results from the CONNECT1 trial in Canada. This parallel study has fully enrolled its first two cohorts, with data expected in the third quarter of this year.

Strategic Decision-Making and Future Plans

James McArthur, Ph.D., CEO of PepGen, explained the rationale behind the pause: "With our 10 mg/kg cohort of CONNECT1 study fully enrolled and data expected later this year, we decided to pause CONNECT2 until we are able to review results from the 10 mg/kg cohort in patients with DMD. This will allow us to gather additional safety data, assess the impact of this dose of PGN-EDO51 on dystrophin levels, and potentially improve the design of CONNECT2."

While the DMD trial faces delays, PepGen is advancing studies of another therapy, PGN-EDODM1, for myotonic dystrophy type 1. A phase 1 trial of this antisense oligonucleotide therapy, which previously faced its own FDA hold in 2023, has recently shown what the company describes as "encouraging initial clinical data."

Regulatory Oversight and Safety Concerns

Health Canada has been actively involved in ensuring PepGen addresses safety concerns related to its trials. The company maintains that no new safety issues have been observed in its ongoing studies. However, the decision to pause the UK trial underscores the complex regulatory landscape and the importance of comprehensive safety data in drug development for rare diseases.

As PepGen navigates these challenges, the pharmaceutical industry watches closely, recognizing the potential impact on future therapies for muscular dystrophies and the broader field of genetic medicine.