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Another Heart Drug Fails, Shocking Cardiologists

Novartis announced that pelacarsen failed to achieve its primary outcome in the Lp(a)HORIZON trial. While the drug successfully lowered lipoprotein(a), it did not reduce cardiovascular events in patients with established disease and elevated Lp(a). This represents the first cardiovascular outcomes trial for an agent designed to lower Lp(a), a protein particle associated with higher heart risk in roughly one in five people. There are currently no approved therapies to lower Lp(a), which is primarily determined by genetics.

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What changed

Novartis officially confirmed the Lp(a)HORIZON trial failed its primary outcome.

Live updates

  1. Pelacarsen Fails to Reduce Cardiovascular Events in Phase III Trial

    Novartis announced that pelacarsen failed to achieve its primary outcome in the Lp(a)HORIZON trial. While the drug successfully lowered lipoprotein(a), it did not reduce cardiovascular events in patients with established disease and elevated Lp(a). This represents the first cardiovascular outcomes trial for an agent designed to lower Lp(a), a protein particle associated with higher heart risk in roughly one in five people. There are currently no approved therapies to lower Lp(a), which is primarily determined by genetics.

    Why it matters

    Cardiologists viewed this drug as a potential breakthrough for a genetic risk factor that remains untreatable. The failure raises questions about whether lowering Lp(a) alone is sufficient to improve patient outcomes. This occurs amid a broader trend where over two-thirds of 2024 FDA-approved drugs relied on a single study.

    What is confirmed

    • The Lp(a)HORIZON trial of pelacarsen failed to achieve its primary outcome.
    • Pelacarsen successfully lowered levels of the Lp(a) protein particle.
    • High levels of Lp(a) occur in about one in five people and are associated with elevated risk for CV events.
    • No therapies are currently approved to lower Lp(a).

    What to watch next

    • Analysis of secondary endpoints from the Lp(a)HORIZON trial
    • Novartis statements on the future development of pelacarsen
    Sources used for this update (7)
    1. economictimes.indiatimes.com — Persistent systems Q4 results
    2. www.yahoo.com — I Thought Cheating Was Unforgivable. Then A Friend's Affair Made Me Question Everything.
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    6. www.statnews.com — Pharmalittle: We’re reading about another Novartis trial failure, a Novo Nordisk setback, and more
    7. www.healio.com — ‘Not the results we hoped for’: Lp(a)-lowering drug pelacarsen fails to reduce CV events
    confidence 100%
  2. Novartis Drug Failure Questions New Heart Medicines

    An experimental cardiovascular drug developed by Novartis and Ionis called pelacarsen failed its Phase III Lp(a)HORIZON trial. The late-stage study targeted patients with established cardiovascular disease and elevated Lp(a). Although the treatment successfully lowered levels of the Lp(a) protein particle, it failed to protect heart health or meet its primary endpoint. This unexpected outcome has surprised cardiologists and raised broader questions about the future of this emerging class of heart medications.

    Why it matters

    The unsuccessful trial represents a major setback for researchers pursuing novel approaches to lower cardiovascular risk through targeted protein reduction. Pelacarsen was evaluated in a large clinical trial designed to treat patients suffering from elevated Lp(a) and established heart disease. The topline results confirmed the drug missed its necessary goals, challenging existing assumptions about how lowering this specific particle impacts patient survival and health.

    What is confirmed

    • Novartis announced that its experimental cardiovascular drug pelacarsen failed to meet its primary endpoint in the Lp(a)HORIZON Phase III trial.
    • The study targeted patients with established cardiovascular disease and elevated Lp(a).
    • Pelacarsen was developed in partnership with Ionis.
    • Pelacarsen successfully lowered levels of a protein particle known as Lp(a) in a large clinical trial.
    • The drug did not protect heart health despite lowering Lp(a) levels, surprising cardiologists and raising questions about an emerging class of heart medicines.

    What to watch next

    • Further data releases or analyses from the Lp(a)HORIZON Phase III trial by Novartis and Ionis
    • Decisions by researchers and developers regarding the future path of emerging Lp(a)-lowering cardiovascular therapies
    Sources used for this update (4)
    1. www.dailymail.com — How to ditch your blood pressure pills: Fed up with side effects or worried about the dangers? These are the simple changes that could help you get off the medication f…
    2. finance.yahoo.com — Novartis, Ionis drug failure spurs questions about an emerging class of heart medicines
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    4. medicalxpress.com — Immediate coronary angiography fails to boost survival after cardiac arrest, trial finds
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  3. Novartis Pelacarsen Fails Phase III Heart Disease Trial

    Novartis announced that its experimental cardiovascular drug pelacarsen failed to meet its primary endpoint in the Lp(a)HORIZON Phase III trial. The study targeted patients with established cardiovascular disease and elevated Lp(a). The failure of this late-stage trial has surprised cardiologists and represents a setback for a new approach to treating heart disease. The drug was developed in partnership with Ionis. Novartis released the topline results confirming the drug did not achieve the necessary goals for the pivotal study.

    Why it matters

    Pelacarsen was designed to lower Lp(a), a specific type of lipoprotein linked to heart disease. This trial aimed to prove that reducing these levels could prevent cardiovascular events. The failure challenges the current experimental strategy for this specific heart disease pathway.

    What is confirmed

    • Novartis announced that pelacarsen missed its primary endpoint in the Lp(a)HORIZON Phase III trial.
    • The trial included patients with established cardiovascular disease and elevated Lp(a).
    • Ionis is a key partner in the development of the experimental heart drug.

    What to watch next

    • Detailed data release from the Lp(a)HORIZON trial
    • Novartis and Ionis official statement on the future of the pelacarsen program
    Sources used for this update (11)
    1. The New York Times — Another Heart Drug Fails, Shocking Cardiologists
    2. Novartis — Novartis announces Lp(a)HORIZON Phase III topline results for pelacarsen in patients with elevated Lp(a) and established cardiovascular disease (CVD)
    3. reuters.com — Ionis, Novartis' key experimental heart drug fails in late-stage trial
    4. STAT — Novartis’ experimental cardiovascular drug fails a pivotal study
    5. Bloomberg.com — Novartis Drug Fails Key Test for New Heart Disease Approach
    6. WSJ — Novartis Says Pelacarsen Heart Disease Drug Missed Primary Endpoint
    7. GlobeNewswire — Novartis announces Lp(a)HORIZON Phase III topline results
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