FDA Puts PepGen's Trial for Myotonic Dystrophy Treatment on Hold
By Kelly Cloonan
PepGen said the Food and Drug Administration is pausing a trial evaluating its potential treatment for myotonic dystrophy.
The biotechnology company said Wednesday the FDA put the Phase 2 trial on hold due to questions related to previously submitted preclinical pharmacology and toxicology studies.
The hold did not cite questions regarding blinded clinical data from the Phase 1 study, which was previously submitted to the FDA to initiate the Phase 2 study in the U.S., the company said.
The company said it plans to submit additional analyses to the FDA and will work to address its questions as quickly as possible.
The trial will continue in Canada and the U.K. It was also recently cleared to open in New Zealand, Australia and South Korea, PepGen said.
The company expects to report data from one cohort of the trial in the first quarter, and from a cohort with a higher dose in the second half of 2026. The trial is evaluating PGN-EDODM1 in patients with myotonic dystrophy type 1.
Write to Kelly Cloonan at kelly.cloonan@wsj.com
(END) Dow Jones Newswires
March 04, 2026 17:28 ET (22:28 GMT)
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