Cholesterol drug setback casts doubt over multibillion-dollar race

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Novartis announced the failure of its experimental drug pelacarsen in a late-stage clinical trial. Developed jointly with Ionis Pharmaceuticals, the treatment reduced levels of lipoprotein(a), a particularly harmful form of cholesterol, but failed to significantly improve cardiovascular outcomes. This is the first major clinical setback in the race to develop treatments that lower Lp(a), affecting roughly one in five people worldwide, with analysts modeling peak annual sales of 4 to 5 billion dollars. Amgen and Eli Lilly, which are developing competing drugs using different technologies, also saw their shares decline. Analysts now say the Lp(a) hypothesis is weakened, but further data is needed to determine whether the failure reflects pelacarsen’s mechanism, trial design, or a broader challenge to the hypothesis that lowering Lp(a) can reduce heart attacks and strokes.

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