PPMD is disappointed to learn that PepGen Inc. has received a full clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application to initiate the CONNECT2-EDO51 clinical trial in patients with Duchenne in the United States. PGN-EDO51 is an PMO-exon skipping therapy bound to a peptide which improves uptake into muscle cells, and targets those amenable to exon 51 skipping. CONNECT2 is the company’s Phase 2 multinational, double-blind placebo-controlled, multiple ascending dose, 25-week clinical trial of PGN-EDO51 in people living with Duchenne.

While the study is open and recruiting in the United Kingdom, no participants have been recruited or dosed in the U.S. According to PepGen, the FDA indicated it will provide an official clinical hold letter to the company within 30 days, and PepGen has indicated that it will work closely with the FDA to resolve questions on the application and initiate CONNECT2 in the U.S. as soon as possible.

CONNECT1-EDO51, PepGen’s multiple ascending dose study of PGN-EDO51, is ongoing in Canada. The company reports that it has completed enrollment of the 10 mg/kg dose cohort, and all four patients in this cohort have received at least one dose.

This news is disheartening and PPMD will share updates with the community as soon as they become available.

Read PepGen’s press release here.