Deldesiran (delpacibart etedesiran, AOC 1001) is an investigational RNA therapeutic candidate for myotonic dystrophy type 1 (DM1). It is being evaluated in ongoing clinical studies and has received multiple FDA expedited development designations. The therapy remains unapproved.
Novartis beat second-quarter profit expectations as Kisqali, Kesimpta and Leqvio offset a 50% plunge in Entresto sales. The Swiss drugmaker maintained its full-year guidance and awaits late-stage data on three experimental drugs.
Avidity Biosciences announced promising Phase 1/2 trial results for its myotonic dystrophy type 1 drug delpacibart etedesiran, showing 40% reduction in DMPK mRNA and functional improvements. The therapy has received multiple regulatory designations and is now in Phase 3 testing with data expected in late 2026. The company's stock has surged 128% over the past year despite reporting a net loss of approximately $550 million.
The FDA has placed a partial hold on U.S. enrollment in a Phase 2 trial of del-desiran for myotonic dystrophy type 1, citing preclinical mouse data showing blood pressure drops not seen in humans, following a serious adverse event in earlier testing.
Phase 1/2 MARINA trial results published in NEJM show del-desiran achieved approximately 40% reduction in toxic DMPK mRNA in muscle tissue and improvements in myotonia and functional measures in myotonic dystrophy type 1 patients, with acceptable safety profile.
