STAT+: Novartis’ experimental cardiovascular drug fails a pivotal study — SkimNews

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- Novartis said Friday that pelacarsen failed the Phase 3 HORIZON trial, missing its goal of reducing cardiovascular death, heart attacks, or stroke versus placebo.
- Pelacarsen had previously demonstrated it could drastically lower Lp(a), a lipid-and-protein particle that at elevated concentrations increases the risk of severe heart problems.
- The HORIZON trial specifically tested whether lowering Lp(a) would translate into fewer real-world cardiovascular events — a hypothesis the study did not confirm.
- The failure is a blow not only to Novartis but to broader drug-development efforts aimed at Lp(a) as a modifiable heart disease risk factor.
- Novartis disclosed the Phase 3 results Friday, calling off what had been a closely watched attempt to prove Lp(a) reduction prevents cardiovascular emergencies.
Why it matters: Pelacarsen was the most advanced drug designed to prove that lowering Lp(a) prevents heart attacks and strokes, and its Phase 3 failure removes the leading proof-of-concept for the entire Lp(a) drug class. Novartis now absorbs a high-profile pipeline setback while competitors working on similar Lp(a)-targeting therapies lose their clearest benchmark for whether the mechanism translates into clinical benefit.
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