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Novartis announced that its global Phase III HARBOR study for delpacibart etedesiran (del-desiran) in myotonic…
NVSNovartis AG
Novartis announced that its global Phase III HARBOR study for delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 failed to meet the primary endpoint of video hand opening time versus placebo. The company observed evidence of clinical activity in secondary endpoints and is evaluating the full dataset with health authorities to determine the future development path. Novartis maintains its five-year sales CAGR guidance of 5-6% for 2025-2030. The filing also highlighted positive developments elsewhere in the neuromuscular pipeline, including FDA priority review designation for delpacibart zotadirsen in Duchenne muscular dystrophy and planned regulatory discussions for delpacibart braxlosiran in facioscapulohumeral muscular dystrophy based on Phase I/II biomarker data. Novartis is an innovative medicines company developing pharmaceuticals to treat serious diseases.