Delpacibart Etedesiran (del-desiran) - (HARBOR)

September 8, 2026: Novartis announced that the Phase III HARBOR study of del-desiran in myotonic dystrophy type 1 (DM1) failed its primary endpoint, showing no statistically significant improvement versus placebo on video hand opening time (vHOT). HARBOR is a global, randomized, double-blind, placebo-controlled 54-week study in about 150 patients. Novartis said del-desiran showed evidence of clinical activity in secondary endpoints and exploratory analyses, and safety was generally consistent with prior data. The company is analyzing the full dataset and plans to engage health authorities to determine the program’s next development path.

The release also highlighted other neuromuscular catalysts from the AOC pipeline acquired through Avidity Biosciences: delpacibart zotadirsen (del-zota) in Duchenne muscular dystrophy patients amenable to exon 44 skipping (DMD44) has been filed for accelerated approval and received FDA priority review; delpacibart braxlosiran (del-brax) in facioscapulohumeral muscular dystrophy (FSHD) is headed to a planned FDA meeting on next steps following positive Phase I/II biomarker data.

Novartis also said it maintains its 2025-2030 guidance for 5-6% five-year sales CAGR.