Therapy Areas: Cardiovascular
Ionis partner Novartis reports pelacarsen Phase 3 trial misses primary endpoint
7 September 2026 -

RNA therapy company Ionis Pharmaceuticals Inc (Nasdaq: IONS) disclosed on Monday that its partner Novartis reported topline results from the Phase 3 Lp(a)HORIZON study and said pelacarsen did not meet its primary endpoint of reducing cardiovascular events in patients with elevated lipoprotein(a) [Lp(a)] and established cardiovascular disease.

The trial evaluated a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke and urgent coronary revascularisation requiring hospitalisation compared with placebo. Pelacarsen substantially reduced Lp(a) levels, consistent with previous studies, and demonstrated an acceptable safety profile.

Lp(a) is an inherited cardiovascular risk factor affecting approximately one in five people worldwide, with no approved targeted treatment. Ionis discovered and conducted the early development of pelacarsen before Novartis exercised an option in February 2019 to license development and commercialisation rights.

Ionis said Lp(a)HORIZON enrolled 8,323 patients with elevated Lp(a) and established cardiovascular disease in a global, randomised, placebo-controlled Phase 3 cardiovascular outcomes trial. Full results will be presented at an upcoming medical congress.

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