Novartis pelacarsen Phase 3 Lp(a)HORIZON trial misses cardiovascular endpoint
On September 4, 2026, Novartis reported that its Phase 3 Lp(a)HORIZON study of pelacarsen did not reduce a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization versus placebo, so the primary endpoint was not met. Pelacarsen nevertheless substantially lowered lipoprotein(a) levels.
Direct September 4, 2026 disclosures from Novartis and Ionis report that the Phase 3 Lp(a)HORIZON trial of pelacarsen failed its prespecified composite cardiovascular endpoint versus placebo despite substantially lowering Lp(a). This is new, material, forward-looking clinical information that is likely to reduce expectations for pelacarsen’s development and commercial value; the reported share-price move is excluded under the anti-price-move rule. The announcement date is explicit, but no exact real-world event time is provided, so 00:00 UTC is used as a date-level estimate. NVS and IONS are approved as the direct sponsor/licensor and development partner, while AMGN is approved because its directly competing Phase 3 Lp(a) outcomes program creates a clear competitive and target-validation read-through; less direct competitor and CDMO exposures are excluded.
Novartis said a heart disease drug did not meet its primary endpoint in a Phase 3 study.
The company on Friday said that the drug, pelacarsen, did not reduce the risk of cardiovascular events--including deaths, heart attacks, and strokes--compared to a placebo.
The drug did succeed in lowering lipoprotein(a) levels, Novartis said.
Late on Friday, Novartis said its drug pelacarsen did not cut heart attack and stroke risk in a large, late-stage study of patients with high Lp(a), an inherited, cholesterol-related risk factor with no approved targeted treatments.
Analysts had modeled peak annual sales between $3 billion and $6 billion for pelacarsen.