Novartis' Delpacibart Etedesiran Phase III Trial Misses Primary Endpoint for DM1

Novartis announced that its global Phase III HARBOR study for delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 (DM1) did not meet its primary endpoint. The trial failed to show a statistically significant improvement in hand myotonia. However, secondary endpoints and exploratory analyses did indicate some evidence of clinical activity. DM1 is a progressive neuromuscular disease currently lacking approved treatment options, making this outcome a setback for patients.