Novartis reported on Sept. 4, 2026 that its experimental cardiovascular therapy pelacarsen failed to reduce the risk of cardiovascular death and emergency events such as heart attack or stroke in the Phase 3 HORIZON trial when compared with placebo. The result is a significant setback for the company’s program targeting elevated levels of Lp(a), a lipid–protein particle associated with higher risk of serious cardiovascular disease.
The company’s announcement followed earlier clinical work in which pelacarsen demonstrated the ability to markedly lower Lp(a) levels. Those biomarker reductions, however, did not translate into the hoped-for benefit on hard clinical endpoints in this pivotal study.
Drug developers and clinicians have been watching therapies that target Lp(a) closely because elevated levels of the particle are linked to an increased likelihood of major heart problems. Pelacarsen had been among the most closely followed candidates after early-stage studies showed strong reductions in Lp(a).
Novartis’ disclosure that the Phase 3 HORIZON trial did not demonstrate a reduction in cardiovascular death or emergency events means that lowering the biomarker alone did not prove sufficient, at least in this trial, to improve those clinical outcomes. The finding will likely prompt reappraisal across companies pursuing Lp(a)-lowering strategies and among researchers studying the relationship between the biomarker and cardiovascular events.
The STAT report notes that other large biopharma firms, including Amgen and Eli Lilly, are developing therapies designed to lower Lp(a). The failure of pelacarsen in HORIZON does not eliminate interest in the pathway, but it does raise questions about whether alternative agents, dosing strategies, trial designs, patient selection, or longer follow-up will be required to show clinical benefit.
The STAT article provides the core outcome: pelacarsen did not reduce the risk of cardiovascular death or emergency cardiovascular events versus placebo in the Phase 3 HORIZON trial. It also reiterates that pelacarsen had reduced Lp(a) in earlier studies.
The STAT piece did not report additional trial details in its public posting. Specifics such as the trial’s enrollment size, participant characteristics, length of follow-up, numerical effect sizes, statistical confidence measures, subgroup results, safety data, or Novartis’ immediate plans for the program were not included in the excerpt available from the source.
Cardiovascular disease remains a leading cause of death globally, and finding new ways to reduce the risk of heart attacks, strokes, and cardiovascular death is a central goal of many development programs. Lp(a) has been considered a promising target because of its association with risk, and several companies invested in therapies aimed at lowering its levels.
A negative pivotal trial such as HORIZON affects multiple stakeholders: it can influence clinical practice expectations, investor assessments of programs that target Lp(a), regulatory considerations for related drugs, and the design of future studies investigating the pathway. It also highlights the distinction between changing a biomarker and demonstrating a meaningful reduction in clinical events.
The STAT piece did not describe Novartis’ next steps for the pelacarsen program, any planned analyses of the HORIZON data, or whether the company will pursue additional studies or regulatory dialogue. It also did not provide details on safety findings from the trial or whether any subgroups experienced benefit. Those details will be important for understanding whether the HORIZON outcome closes the book on pelacarsen and whether lessons from the trial will inform other Lp(a) programs.
Observers will look for further communications from Novartis, full data releases, and peer-reviewed publication of the HORIZON results to learn more about the trial’s design, outcomes, and implications. Other companies working on Lp(a)-lowering therapies will likely interpret these results in the context of their own development plans, and regulators and clinicians will await more comprehensive data before drawing broader conclusions.
This coverage is based on a STAT News report by Andrew Joseph published Sept. 4, 2026. The STAT article provided the key outcome from Novartis’ announcement but did not include the fuller set of trial data or Novartis’ stated next steps in the public excerpt.
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