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Ionis partner Novartis announces Lp(a)HORIZON Phase 3 topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD)

Friday, September 4, 2026 BiopharmaWatch Research 1 min read
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Key Takeaway: Ionis Pharmaceuticals' partner Novartis reported that the pelacarsen Phase 3 Lp(a)HORIZON study failed to achieve its primary goal of reducing cardiovascular event risks. The study aimed to evaluate the drug's effectiveness in patients with elevated Lp(a) and established cardiovascular disease but did not show significant benefits compared to placebo. This outcome is consistent with previous research findings.

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CONCERNS & RISKS

  • Pelacarsen did not meet its primary endpoint in the Phase 3 study.
  • The trial aimed to reduce cardiovascular event risks but failed to do so.
  • This outcome raises concerns about the drug's efficacy in treating elevated Lp(a).

BiopharmaWatch Analysis

From our catalyst data and publicly available data · not financial advice
Best trade, last catalyst
+18%
120-day peak, hindsight
Typical move
4%
average across 12 past catalysts
Lead asset
ISIS-GCGRRx- Dose Level 1
Phase 2 · Type 2 Diabetes Mellitus

Full Press Release Details

CARLSBAD, Calif.--(BUSINESS WIRE)--Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) partner Novartis today announced that the pelacarsen Phase 3 Lp(a)HORIZON study, a pioneering cardiovascular outcomes trial, did not meet its primary endpoint of reducing the risk of cardiovascular events, a composite of cardiovascular death, non-fatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization, compared to placebo. Consistent with previous studies, substan

Frequently Asked Questions

What was the primary endpoint of the Lp(a)HORIZON study?

The primary endpoint was to reduce the risk of cardiovascular events compared to placebo.

Did pelacarsen meet its primary endpoint?

No, pelacarsen did not meet its primary endpoint in the Phase 3 study.

What type of patients were involved in the study?

The study involved patients with elevated Lp(a) and established cardiovascular disease.

What cardiovascular events were assessed in the trial?

The trial assessed cardiovascular death, non-fatal myocardial infarction, and non-fatal stroke.

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Tags: IONS Viatris Investor Conferences Healthcare pelacarsen Phase 3 Lp(a)HORIZON cardiovascular disease Novartis
Last updated: Sep 4, 2026

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Lead asset
ISIS-GCGRRx- Dose Level 1Phase 2
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