Lp(a): Why biotech is betting on the “diabolical” molecule

The “diabolical” molecule is lipoprotein(a), or Lp(a), an inherited blood particle associated with heart attacks, strokes and aortic-valve disease.

Lp(a) is largely genetically determined, so diet and exercise have limited effect on lowering it. Its production in the liver makes it a promising target for RNA-based medicines, including antisense oligonucleotides and siRNA therapies.

An estimated 20% of people worldwide may have Lp(a) levels associated with elevated cardiovascular risk, creating a potentially enormous treatment market.

Novartis and Ionis Pharmaceuticals’ antisense drug pelacarsen is the leading near-term test for the field. Its phase 3 Horizon trial is studying 8,323 people with cardiovascular disease and high Lp(a), seeking to show fewer repeat cardiac events—not merely lower biomarker levels.

Results were expected within months of the April 2026 report. If positive, pelacarsen could potentially be approved as early as the following year and stimulate major investment in Lp(a)-lowering drugs.

Amgen and Eli Lilly are developing competing approaches, while AstraZeneca and Thalia Therapeutics are pursuing additional Lp(a)-focused strategies.

The opportunity is substantial but not guaranteed:
researchers still need definitive evidence that reducing Lp(a) directly lowers cardiovascular events, and the market would have to overcome competition, pricing and treatment-selection questions.