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Pelacarsen Heart Drug Fails Despite 78% Cholesterol CutHealth
A landmark trial has delivered a sobering result for heart medicine: a drug that cut a risky cholesterol marker by 78% did not prevent a single extra heart attack or stroke.
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A much-anticipated heart drug has failed the test that mattered most. Pelacarsen, an experimental medicine from Novartis, cut levels of a genetically inherited cholesterol particle by about 78%, yet it did not prevent heart attacks, strokes or cardiovascular deaths, according to results presented on Saturday at the American Heart Association meeting in Chicago and published in the New England Journal of Medicine.
The trial, known as Lp(a)HORIZON, enrolled 8,323 patients around the world who had high levels of lipoprotein(a) — usually shortened to Lp(a) — and established heart disease. Half received a monthly 70-milligram injection of pelacarsen and half a placebo, and they were followed for a median of 4.7 years. At 12 months, patients on the drug showed an estimated 77.7% greater reduction in Lp(a) than those on placebo. But major cardiovascular events occurred in 12.3% of the pelacarsen group and 12.1% of the placebo group — effectively no difference.
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A result described as completely neutral
Dr Steven Nissen of the Cleveland Clinic presented the results. Novartis had already announced in September that the trial had missed its main goal, but doctors and investors had been waiting for the full details. A separate analysis published in NEJM Evidence, which looked at patients with the largest Lp(a) reductions and those who stayed in the trial, also found no benefit. About 20.6% of patients stopped pelacarsen because of side effects, compared with 7.5% on placebo. As one cardiologist at the meeting put it, the drug did nothing: the result was completely neutral.
The outcome is a setback for a long-held idea in preventive cardiology. Lp(a) is a largely inherited particle that raises the risk of heart disease, and it is estimated to affect about one in five people worldwide. There are currently no approved treatments for high Lp(a). Researchers had hoped that lowering it sharply would translate into fewer heart attacks. The trial suggests that changing a blood marker, even dramatically, does not automatically protect patients.
Doctors urged caution before writing off Lp(a) altogether. Dr Donald Lloyd-Jones, a professor of cardiology at Boston University, said he would not recommend abandoning Lp(a) as a cause of heart disease or as a treatment target until there is much more data in different patient groups. Attention now turns to other drugs in development, including Amgen’s olpasiran and Eli Lilly’s lepodisiran, which use a different technology and have lowered Lp(a) by about 95% in earlier studies. Their large trial results are expected in early 2028 and 2029.
The same meeting delivered a second disappointment: a Novo Nordisk drug designed to reduce heart inflammation also hit its biological target but failed to prevent major cardiovascular events. For patients and doctors, the message from Chicago is clear — promising laboratory numbers are not the same as fewer heart attacks, and only large, long trials can tell the difference.
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