Novartis announced that the pelacarsen phase III Lp(a)HORIZON trial, a pioneering cardiovascular outcomes study, did not meet its primary endpoint of reducing the risk of cardiovascular events, a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization, compared to placebo¹. Lower lipoprotein (a), Lp(a), levels were achieved with pelacarsen in this study population, which was receiving guideline-directed treatments, including lipid lowering and antihypertensive therapies^1,2.

The findings provide important new evidence on the relationship between Lp(a) lowering and cardiovascular outcomes. Elevated Lp(a) is an inherited cardiovascular risk factor affecting approximately one in five people worldwide with no approved targeted treatment^1,3.

"Lp(a)HORIZON was a pioneering cardiovascular outcomes trial designed to answer one of the most important unanswered questions in cardiovascular medicine by evaluating whether lowering Lp(a) can reduce residual cardiovascular risk beyond optimized, guideline-directed care, when other major cardiovascular risk factors are already being managed," said Shreeram Aradhye, President, Development and Chief Medical Officer, Novartis. "Although lower Lp(a) levels were observed with pelacarsen, the findings did not demonstrate that this translated into reduced cardiovascular risk in the overall study population.

These are not the results we hoped for, but they provide important evidence that advances scientific understanding of the relationship between Lp(a) lowering and cardiovascular outcomes and may help inform future approaches to cardiovascular risk management. We thank the more than 8,000 trial participants and investigators who made this study possible, and remain committed to driving cardiovascular innovation."