STAT+: PepGen says early trial for muscular dystrophy drug looks promising

The biotech PepGen said Tuesday that its experimental drug had shown, by one key measure, the most promising early data yet in the race to treat a muscle disorder that affects over 100,000 people in the U.S. and Europe. PepGen is a latecomer to the fight to treat myotonic dystrophy type 1 (DM1), a condition that typically robs patients of muscle control and strength beginning around adolescence. Tuesday’s data come from six patients, who were treated for 12 weeks. Dyne Therapeutics is close to completing a pivotal trial for a DM1 drug. Novartis just announced that a DM1 drug it acquired in its $12 billion buyout of Avidity Biosciences failed to improve a key measure of muscle control in a Phase 3 trial, casting a pall over the field. STAT+ Exclusive Story Already have an account? Log in This article is exclusive to STAT+ subscribers Unlock this article — plus in-depth analysis, newsletters, premium events, and news alerts. Already have an account? Log in View All Plans To read the rest of this story subscribe to STAT+. Subscribe