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Getting the latest healthcare news for you
Getting the latest healthcare news for you
A highly anticipated trial testing pelacarsen, a drug designed to lower lipoprotein(a), failed to reduce major cardiovascular events compared to placebo. The Lp(a)HORIZON trial — the first-ever CV outcomes trial for an Lp(a)-lowering agent — showed the drug did reduce Lp(a) levels, but that didn't translate into fewer heart attacks, strokes, or CV deaths. The results leave big questions about whether Lp(a) is a true modifiable risk factor.
Novartis announced that pelacarsen, an antisense oligonucleotide designed to lower lipoprotein(a) [Lp(a)], failed to meet its primary endpoint in the Lp(a)HORIZON phase 3 trial — the first-ever cardiovascular outcomes trial for an Lp(a)-lowering agent. While patients on pelacarsen did achieve meaningfully lower Lp(a) levels, this did not translate into a statistically significant reduction in CV death, nonfatal MI, nonfatal stroke, or urgent coronary revascularization compared to placebo.
The trial enrolled patients with elevated Lp(a) and established cardiovascular disease who were already on guideline-directed medical therapy, including lipid-lowering and blood pressure treatments. Experts note the excellent baseline risk factor control in the trial may have made it harder to detect an incremental benefit from pelacarsen — a challenge common in well-managed trial populations.
Key Takeaways:
Why it matters: Elevated Lp(a) affects roughly 1 in 5 people and has long been considered a promising therapeutic target. This trial's failure is a significant setback, but experts say it raises — rather than closes — important scientific questions about Lp(a)'s role in cardiovascular risk and the future of this drug class.
