Novartis announces Phase III HARBOR study for myotonic dystrophy type 1 did not meet its primary endpoint

Novartis reported that its global Phase III HARBOR study, evaluating delpacibart etedesiran (del-desiran) for myotonic dystrophy type 1 (DM1), did not achieve its primary endpoint of statistically significant improvement in video hand opening time (vHOT). Despite this, evidence of clinical activity was observed in secondary endpoints and exploratory analyses. DM1 is a progressive neuromuscular disease with no approved treatments, and Novartis is currently evaluating the full dataset to determine the future development path for del-desiran.