Novartis cholesterol drug pelacarsen fails to cut heart disease risk in phase 3 trial

Pelacarsen, a drug from pharmaceutical major Novartis, has failed to deliver the expected results in a key phase 3 trial. The outcome is a major setback to efforts to reduce the risk of heart disease by lowering a particular type of cholesterol in the body. The trial did not prove a real clinical benefit from the drug, and disappointment is being voiced in heart disease research.

Pelacarsen was developed to lower levels of lipoprotein(a), or Lp(a), a substance linked to cholesterol. High levels of it in the body raise the risk of heart attack, stroke and heart valve diseases. Notably, Lp(a) levels depend largely on heredity, so they cannot easily be brought down through regular exercise, changes in diet or weight loss. About one in every five people in the world is believed to have a high level of Lp(a).

LDL, commonly known as bad cholesterol, can be reduced with drugs such as statins. But no widely accepted treatment has so far been available to lower Lp(a). For this reason, the medical community was watching pelacarsen closely. Earlier tests had shown that the drug could lower Lp(a) levels by more than 80 per cent. Even so, the result has once again made it clear that lowering cholesterol levels and actually lowering the risk of heart disease are two different things.

Meanwhile, drug companies such as Amgen and Eli Lilly are also researching drugs that lower Lp(a). The results of their trials will now be important. Genetic research has indicated that Lp(a) may not merely signal the risk of heart disease but could also be a factor that causes the disease. Finding out whether lowering its level truly reduces cases of heart disease and stroke has become a major challenge for medical research.