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Ionis, Novartis’ Key Experimental Heart Drug Fails in Late-Stage Trial

The drug lowered lipoprotein(a) levels, but the 8,323-patient study did not reduce major cardiovascular events, the companies said.

  • On Friday, Novartis and partner Ionis Pharmaceuticals announced that pelacarsen failed the Phase 3 HORIZON trial, missing its primary goal of reducing cardiovascular death or emergencies compared to placebo.
  • The trial targeted lipoprotein, a genetic cholesterol risk factor affecting approximately 20% of people worldwide that contributes to plaque buildup and inflammation in arteries, leading to cardiovascular events.
  • Although pelacarsen substantially lowered lipoprotein levels, Ionis CEO Brett Monia said it did not translate to cardiovascular risk reduction, while Novartis chief medical officer Shreeram Aradhye called the results "important evidence" for scientific understanding.
  • Shares of Novartis declined 5%, while Ionis stock fell 12% in aftermarket trading, as investors reacted to the failure of the 8,323-patient cardiovascular outcomes trial.
  • Citi analyst Geoff Meacham noted this failure lowers confidence in the drug class, increasing pressure on competitors like Amgen's olpasiran and Eli Lilly's lepodisiran, which remain in late-stage development.
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Ionis, Novartis' key experimental heart drug fails in late-stage trial

·London, United Kingdom
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One of the world's largest pharmaceutical companies, Switzerland-based Novartis, admitted on Friday that an experimental cardiovascular disease drug it is developing did not reduce the risk of serious heart attacks and strokes in patients with a genetic risk factor.

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Reuters broke the news in London, United Kingdom on Friday, September 4, 2026.
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