Novartis heart drug failure leaves doctors seeking evidence for genetic cholesterol target
NVS•Novartis said its Phase 3 trial of pelacarsen did not meet its main goal of reducing cardiovascular risk, contributing to a 16% share-price drop. Doctors are awaiting detailed results to assess whether lowering Lp(a) can prevent heart attacks and other events.
1. Trial misses main goal
Novartis’s experimental heart drug pelacarsen failed to significantly reduce the risk of a composite of death, heart attack, stroke and procedures to restore blood flow. The drug lowered Lp(a) levels by as much as 80% in earlier research, but that did not translate into a lower risk of heart attack and stroke in the trial.
2. Results under scrutiny
Full trial details are due to be presented Saturday at the American Heart Association’s annual scientific meeting in Chicago. Cardiologists say they will look at whether the study narrowly missed statistical significance, showed no impact or found a negative effect, and will focus on heart attack and revascularization rates. The trial tested a monthly 80-milligram dose; doctors also raised questions about patient dropouts, the study’s composite endpoint and use of PCSK9 drugs during the trial.
3. Other drugs in development
Amgen’s olpasiran and Eli Lilly’s lepodisiran have lowered Lp(a) levels by around 95% in studies, with Phase 3 results expected in early 2028 and 2029, respectively. Those studies differ from Novartis’s trial, including by enrolling patients with higher Lp(a) levels.
