Novartis’ del-desiran fails Phase III DM1 primary endpoint

  • Novartis reported that del-desiran failed HARBOR’s primary DM1 endpoint against placebo.
  • Novartis shares fell more than 3% after a separate cholesterol-drug trial failure.
  • Novartis reaffirmed 5-6% annual sales growth guidance for 2025-2030.

Novartis said its global Phase III HARBOR study of del-desiran in myotonic dystrophy type 1 (DM1) did not achieve a statistically significant improvement versus placebo on video hand opening time (vHOT), its primary endpoint measuring hand myotonia. The announcement followed a more than 3% decline in Novartis shares the previous day after its cholesterol drug failed in a closely watched study. Novartis said it would review the complete HARBOR dataset and consult health authorities on del-desiran’s development path. The company observed evidence of clinical activity in secondary endpoints and exploratory analyses, with safety generally consistent with earlier data. Del-desiran entered Novartis’ pipeline through its $12 billion acquisition of Avidity Biosciences. Novartis reaffirmed its forecast for sales to grow at a compound annual rate of 5-6% from 2025 through 2030.

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