While PepGen works to persuade the FDA to let it launch a phase 2 trial of its Duchenne muscular dystrophy (DMD) therapy in the U.S., the biotech has decided to pause the U.K. arm of the study.
The CONNECT2-EDO51 trial, which is designed to test PepGen’s PGN-EDO51 exon-skipping therapy over 25 weeks in patients with DMD, was placed on hold by the FDA in December. The following month, PepGen disclosed that two patients in the 10-mg/kg cohort had reported asymptomatic magnesium deficiency, although their magnesium levels had since returned to baseline levels after receiving oral magnesium supplements.
Now, PepGen has decided to pause the U.K. arm of the study while it considers any lessons from a separate phase 2 study, dubbed CONNECT1, that’s ongoing in Canada. The first two cohorts of the Canadian study have been fully enrolled, with a readout expected in the third quarter.
Health Canada has previously been in touch with PepGen to ensure it addressed the regulator’s safety concerns. The company said today that it hasn’t observed any new safety issues in its trials.
“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,” PepGen CEO James McArthur, Ph.D., said in the March 4 release.
“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,” McArthur continued.
PepGen will also “rapidly advance” studies of its antisense oligonucleotide therapy PGN-EDODM1 in myotonic dystrophy type 1. A phase 1 trial of the therapy endured its own FDA hold back in 2023 but has since gone on to post what PepGen described last week as “encouraging initial clinical data.”