Novartis announced that its late-stage HARBOR study evaluating del-desiran in people with myotonic dystrophy type 1 (DM1) failed to meet the primary endpoint, causing shares to plunge. The study did not show a statistically significant improvement over placebo on the primary endpoint of video hand opening time, though evidence of clinical activity was seen in secondary endpoints. This marks the second pipeline setback for Novartis in five days, following the failure of pelacarsen in a late-stage study. Del-desiran is one of three AOC therapies acquired through the Avidity Biosciences deal, and Novartis is evaluating the full dataset to determine next steps. The company faces a major patent cliff in 2026 as generic competition affects key drugs, and these failures dampen investor sentiment.
Novartis AGDel-desiran failed late-stage DM1 study, missing primary endpoint.
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