FDA Lifts Hold on PepGen Study

FDA has lifted a clinical hold against PepGen that will allow the company to begin the Phase 1 FREEDOM-DM1 study of PGN-EDODM1 in patients with myotonic dystrophy Type 1 in the U.S. The company says the condition (also known as Steinert’s disease) is a progressively disabling, life-shortening genetic disorder.

“Our novel PGN-EDODM1 approach targets the toxic RNA species responsible for the disease, and the strength of our Enhanced Delivery Oligonucleotide (EDO) safety preclinical package has enabled us to launch this study in both the U.S. and internationally at doses that we believe could provide a clinically meaningful benefit to patients,” a company release says. “We are very pleased after review of our existing safety data that the FDA agreed with our proposed starting dose of 5 mg/kg, moving up to 10 mg/kg and 20 mg/kg.”

PGN-EDODM1 is designed to deliver a peptide-conjugated antisense oligonucleotide to restore cellular function, PepGen says.