Novartis heart drug failure leaves doctors seeking evidence for genetic cholesterol target
The Phase 3 drug cut Lp(a) by 80% but did not significantly lower death, heart attack, stroke or revascularization risk, Novartis said.
- Novartis and Ionis Pharmaceuticals reported in early September that their Phase 3 pelacarsen trial failed to meet its primary goal of reducing cardiovascular risk, despite lowering Lp levels by 80%.
- Participants concurrently used LDL-lowering statins and PCSK9s, which may have masked efficacy. Dr. Robert Rosenson, director of metabolism and lipids at Mount Sinai Fuster Heart Hospital, cited potential patient dropouts, noting "One possibility is that maybe there were dropouts. They never studied that dose all at once."
- Study investigator Parag Joshi expressed interest in seeing specific heart attack and revascularization rates. The trial failure triggered a 16% share price drop for Novartis, signaling market concern over the unexpected negative result.
- Researchers presented full results on Saturday in Chicago at the AHA annual scientific meeting, where the 80% Lp reduction failed to translate into lower heart attack or stroke risks.
- Competitors Amgen and Eli Lilly are developing therapies showing 95% Lp reduction in earlier trials, with Phase 3 results expected in early 2028 and 2029 respectively. Their studies differ by enrolling only patients with higher baseline Lp levels than the Novartis trial.
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Novartis heart drug failure leaves doctors seeking evidence for genetic cholesterol target
The recent failure of a Novartis experimental heart drug in a large trial raised questions among cardiologists and investors about the widely held idea that reducing levels of a genetically set cholesterol carrier called lipoprotein(a) can improve heart health.
This cholesterol drug promised to be a game-changer. Why did it flop in the clinic — and is there still hope?
Nature, Published online: 03 November 2026; doi:10.1038/d41586-026-03504-2Physicians were excited about several therapies that slash levels of lipoprotein(a) — an important cardiovascular risk factor. One major trial failed to show a benefit, and now scientists are scrambling to work out what that means.
Cholesterol Treatment Breakthrough: Why It Failed in Clinical Trials and Whether Hope Remains
In early September, pharmaceutical company Novartis reported that pelacarsen, a drug once expected to become a blockbuster, had failed to reduce the risk of heart disease or stroke in people with established cardiovascular disease. The result from the late-stage Lp(a)HORIZON trial surprised cardiologists because pelacarsen sharply lowers lipoprotein(a), or Lp(a), a genetically determined cholesterol-carrying particle [...]
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