Cholesterol drug pelacarsen fails to prevent heart attacks in major trial

Key Points:

• The experimental drug pelacarsen, designed to lower lipoprotein(a) [Lp(a)] levels, showed no reduction in heart attacks, strokes, cardiovascular deaths, or related procedures in a large clinical trial involving over 8,300 participants.
• Despite pelacarsen reducing Lp(a) levels by 78%, the trial results, presented at the American Heart Association meeting and published in the New England Journal of Medicine, revealed no cardiovascular health benefits, surprising many researchers.
• Lp(a) is a genetic form of cholesterol linked to heart disease risk, affecting about 20% of Americans, and current treatments like diet and exercise do not affect its levels, which has driven interest in drug therapies.
• Experts suggest the drug may have been administered too late in life or that other aggressive treatments participants received could have masked potential benefits; however, Lp(a) remains a valuable marker for cardiovascular risk assessment.
• While no further studies on pelacarsen are planned, other Lp(a)-lowering drugs using different technologies are in development, and researchers remain hopeful about finding effective treatments in the future.