Novartis said its global Phase 3 HARBOR study of del-desiran in myotonic dystrophy type 1 did not meet the primary endpoint of video hand opening time (vHOT) versus placebo. The company reported evidence of clinical activity in secondary endpoints and exploratory analyses, with safety findings described as generally consistent with previously reported data. Novartis is now evaluating the full HARBOR dataset and will engage with health authorities to determine the most appropriate development path for the antibody-oligonucleotide conjugate. Separately, Novartis is maintaining momentum in related neuromuscular programs, including delpacibart zotadirsen in DMD44, which is under accelerated approval-related pathways.
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