Novartis Says Delpacibart Etedesiran Missed Primary Endpoint in Late-stage Neuromuscular Disease Study

01:24 AM EDT, 09/08/2026 (MT Newswires) -- Novartis (NOVN.SW) said Tuesday that delpacibart etedesiran, or del-desiran, failed to meet the primary endpoint of a phase 3 trial in myotonic dystrophy type 1, a progressive neuromuscular disease. In the late-stage Harbor study, del-desiran did not demonstrate a statistically significant improvement in video-assessed hand opening time, compared with placebo. Meanwhile, the drug candidate showed clinical activity in secondary endpoints and exploratory analyses. The safety profile of del-desiran was generally consistent with previously reported data. Novartis plans to evaluate the full clinical data and coordinate with health agencies to decide on the "most appropriate" development steps for del-desiran.