- PepGen can start a Phase 1 study in the U.S. of a therapy for a rare form of muscular dystrophy after the Food and Drug Administration lifted a clinical hold on the program.
- The trial will include sites in both the U.S. and Canada, where regulators cleared the treatment for human testing in September. PepGen said Thursday it expects to release proof-of-concept data from the study, dubbed Freedom-DM1, next year
- The oligonucleotide-based therapy is designed to treat patients with myotonic dystrophy type 1, or DM1. The life-shortening condition causes stiff or contracted muscles, general muscle weakness and cardiac and respiratory symptoms. It is estimated to affect about 40,000 people in the U.S. and 70,000 in the European Union.
The FDA has shown caution with companies developing RNA treatments for neuromuscular conditions. Dyne Therapeutics, Sarepta Therapeutics, Avidity Biosciences and Entrada Therapeutics have all faced delays in their work amid partial or full clinical holds.
PepGen appears to have resolved its issues with regulators relatively quickly, which is an encouraging sign for the program, Stifel analyst Paul Matteis wrote in a note to investors. He called the stock “very cheap,” given that the company is on track to provide proof-of-concept data not only for the DM1 drug but also for another experimental therapy for Duchenne muscular dystrophy next year.
Shares of PepGen were little changed in early trading Thursday. The company’s stock had soared above $17 in March of this year before the clinical hold was announced in May. PepGen had $147 million in cash as of June 30. The company reiterated Thursday that it expects to have enough money to fund operations into 2025.