Novartis Says Pelacarsen Heart Disease Drug Missed Primary Endpoint

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1. 3d ago

   Novartis Pelacarsen Phase 3 Trial Fails to Meet Primary Endpoint

   Novartis halted the Lp(a)FRONTIERS PEARL Phase 3 study after stopping the Lp(a)HORIZON trial due to a missed composite cardiovascular primary endpoint. Although pelacarsen successfully reduced lipoprotein(a) levels, it failed to achieve a significant reduction in heart attacks, strokes, and major cardiovascular events among patients with acute coronary syndrome. This setback follows the discontinuation of an iptacopan mid-stage lupus trial and a failed muscle-disorder treatment trial. Additionally, another Novartis drug, the IL-6 inhibitor ziltivekimab, failed to lower cardiovascular events in its own Phase 3 trials.

   Why it matters

   These trial failures deal a substantial blow to the cardiovascular drug pipeline at Novartis. The inability of pelacarsen to translate biomarker reductions into clinical event prevention raises questions about targeting lipoprotein(a) for heart disease. And the simultaneous failures of multiple late-stage clinical candidates compound the clinical and strategic challenges facing the company.

   What is confirmed

   • Novartis stopped the Lp(a)FRONTIERS PEARL Phase 3 study of pelacarsen in patients with acute coronary syndrome.
   • The Lp(a)HORIZON Phase 3 trial missed its composite cardiovascular primary endpoint.
   • Pelacarsen lowered lipoprotein(a) levels but failed to significantly reduce heart attacks, strokes, and major cardiovascular events.
   • The IL-6 inhibitor ziltivekimab also failed to reduce cardiovascular events in Phase 3 trials.
   • Novartis discontinued a mid-stage lupus trial for iptacopan and a muscle-disorder treatment trial.

   What to watch next

   • Comprehensive data presentation from the halted Lp(a) trials at an upcoming medical meeting
   • Further pipeline portfolio adjustments announced by Novartis management
   Sources used for this update (2)

   1. www.marketbeat.com — Top Biotech Stocks To Research - October 14th | MarketBeat
   2. pubmed.ncbi.nlm.nih.gov — Lp(a)-Reducing Agent Pelacarsen Misses the Mark in Phase 3 ...
   confidence 100%
2. 6d ago

   Novartis Halts Acute Coronary Syndrome Study After Pelacarsen Trial Failure

   Novartis stopped the Lp(a)FRONTIERS PEARL Phase 3 study of pelacarsen in patients with acute coronary syndrome. This follows the September 4, 2026 announcement that the Lp(a)HORIZON Phase 3 trial missed its composite cardiovascular primary endpoint. While pelacarsen lowered lipoprotein(a) levels, it failed to significantly reduce heart attacks, strokes, and major cardiovascular events. Another Novartis drug, the IL-6 inhibitor ziltivekimab, also failed to reduce cardiovascular events in Phase 3 trials. These setbacks coincide with the discontinuation of a mid-stage lupus trial for iptacopan and a failed muscle-disorder treatment trial.

   Why it matters

   Targeting Lp(a) is a strategy to reduce atherosclerotic events in high-risk patients. The failure of both an antisense oligonucleotide and an IL-6 inhibitor suggests that lowering Lp(a) alone may be insufficient for cardiovascular protection. Multiple pipeline failures are now impacting investor confidence in Novartis acquisitions.

   What is confirmed

   • The Phase 3 Lp(a)HORIZON trial of pelacarsen missed its composite cardiovascular primary endpoint.
   • Novartis reported the Lp(a)HORIZON trial results on September 4, 2026.
   • Ziltivekimab failed to reduce cardiovascular events in Phase 3 trials.
   • Novartis stopped the Phase 3 Lp(a)FRONTIERS PEARL study of pelacarsen in patients with acute coronary syndrome.
   • Novartis discontinued a mid-stage lupus trial for iptacopan.

   Still unconfirmed

   • A clinical trial for a muscle-disorder treatment failed, causing Novartis shares to fall sharply.

   What to watch next

   • Company guidance on the future of the acquired drug pipeline.
   Sources used for this update (7)

   1. swissobserver.ch — Novartis shares plunge after latest clinical-trial setback
   2. www.pharmacytimes.com — Pelacarsen, Ziltivekimab Miss CV End Points: What to Know
   3. trial.medpath.com — Pelacarsen and Ziltivekimab Miss Cardiovascular End Points in ...
   4. www.trialticker.com — Novartis Pharmaceuticals stops Phase 3 ACS study Lp (a ...
   5. www.simianx.ai — pelacarsen · NCT07625306 registry update | SimianX AI
   6. www.europesays.com — Novartis Discontinues Mid-Stage Lupus Trial In Latest ...
   7. finance.yahoo.com — Novartis Discontinues Mid-Stage Lupus Trial In Latest Pipeline Setback
   confidence 95%
3. 13d ago

   Novartis' Pelacarsen Misses Primary Endpoint in Phase 3 Trial

   Novartis' pelacarsen drug failed to produce a statistically significant reduction in strokes, heart attacks, and major cardiovascular events despite lowering lipoprotein(a) levels in over 8,000 patients. The Phase 3 Lp(a)HORIZON trial missed its primary endpoint, suggesting that lowering Lp(a) alone may not be sufficient to reduce atherosclerotic events. This result has implications for the development of treatments targeting Lp(a).

   Why it matters

   The trial's outcome has significant implications for the pharmaceutical industry, particularly in the development of cardiovascular treatments. Pelacarsen was developed by Novartis and Ionis Pharmaceuticals to target lipoprotein(a), a protein associated with an increased risk of cardiovascular disease. The failure of pelacarsen and another trial, ZEUS, has raised questions about the Lp(a) hypothesis.

   What is confirmed

   • Pelacarsen significantly lowered lipoprotein(a) levels in over 8,000 patients.
   • The Phase 3 Lp(a)HORIZON trial missed its primary endpoint of reducing strokes, heart attacks, and major cardiovascular events.
   • The failure of pelacarsen and ziltivekimab trials has raised questions about the Lp(a) hypothesis.
   • Experts believe it is too early to give up on the Lp(a) hypothesis.

   What to watch next

   • Further analysis of Lp(a)HORIZON trial data
   • Development of other Lp(a) targeting treatments
   • Regulatory decisions on pelacarsen
   Sources used for this update (3)

   1. ecgwaves.com — ZEUS and Lp(a)HORIZON: why ziltivekimab and pelacarsen failed
   2. www.medscape.com — Experts Say It’s Too Early to Ditch the Lp(a) Hypothesis - Today on Medscape
   3. ctv.veeva.com — Lp (a) Lowering Study of Pelacarsen (TQJ230) in US Black ...
   confidence 90%
4. 18d ago

   Novartis Pelacarsen Fails Phase 3 Heart Disease Trial

   Novartis and Ionis Pharmaceuticals report that pelacarsen missed its primary endpoint in the Phase 3 Lp(a)HORIZON trial. Despite significantly lowering lipoprotein(a) levels in more than 8,000 patients, the drug failed to produce a statistically significant reduction in strokes, heart attacks, and major cardiovascular events. This result suggests that lowering Lp(a) alone may not be sufficient to reduce atherosclerotic events. Meanwhile, partner Ionis Pharmaceuticals reported positive interim results for sefaxersen, an experimental kidney disease drug developed with Roche for adults with primary IgA nephropathy.

   Why it matters

   Lipoprotein(a) is a known risk factor for atherosclerotic cardiovascular disease. The failure of pelacarsen impacts the broader competitive race for Lp(a) treatments, which includes candidates from companies like Amgen. Novartis had previously invested 60 million dollars in a next-generation follow-up drug with Ionis.

   What is confirmed

   • Pelacarsen failed to produce a statistically significant reduction in major cardiovascular events in the Phase 3 Lp(a)HORIZON trial.
   • The Lp(a)HORIZON study included more than 8,000 patients.
   • Pelacarsen successfully lowered lipoprotein(a) levels.
   • Ionis Pharmaceuticals reported positive prespecified interim results from the Phase 3 IMAgINATION study for sefaxersen.

   Still unconfirmed

   • Lp(a) lowering alone may not be enough to reduce cardiovascular death or atherosclerotic events.
   • The failure of pelacarsen could reshape the Lp(a) development race for CRISPR and Ionis.

   What to watch next

   • Phase 3 trial results for Amgen's olpasiran candidate.
   • Development progress of the next-generation Lp(a) drug Novartis is developing with Ionis.
   Sources used for this update (10)

   1. finance.yahoo.com — Failure of Novartis’ Pelacarsen Could Reshape the Lp(a) Race for CRISPR and Ionis
   2. pharmaphorum.com — Tenax trounced by phase 3 heart failure readout | pharmaphorum
   3. imedic.health — Pelacarsen Heart Trial: What Does the Lp (a) Setback | iMedic
   4. stockanalysis.com — AMGN06
   5. hemostasistoday.com — Franck Perraudin: Lp (a) Lowering Alone May Not Be Enough
   6. pauldthompsonmd.substack.com — Reducing Lipoprotein a Failed to Reduce Atherosclerotic Events
   7. pharmaphorum.com — Dosing prompts Novartis to tap Ionis for second Lp(a) drug
   8. www.ajmc.com — Jorgen Vestbo, MD
   9. pharmaphorum.com — Chasing Novartis, Amgen ushers Lp (a) drug into phase 3
   10. ctv.veeva.com — Lp(a) Lowering Study of Pelacarsen (TQJ230) With Background ...
   confidence 95%
5. 30d ago

   Pelacarsen Fails Phase 3 Heart Disease Trial; Ionis Reports Kidney Drug Success

   Novartis and Ionis Pharmaceuticals reported that pelacarsen failed its Phase 3 Lp(a)HORIZON trial primary endpoint. The drug failed to reduce strokes, heart attacks, and related cardiovascular events despite lowering Lp(a) levels in a study of over 8,000 patients. While Novartis faces a string of clinical setbacks, partner Ionis Pharmaceuticals announced positive prespecified interim results from the Phase 3 IMAgINATION study for sefaxersen, an experimental kidney disease drug developed with Roche for adults with primary IgA nephropathy.

   Why it matters

   The pelacarsen failure is part of a wider period of volatility for Novartis, which recently paused eight trials for rapcabtagene autoleucel following three patient deaths. These combined clinical losses have put pressure on the company's $12 billion Avidity agreement.

   What is confirmed

   • Pelacarsen failed to reduce heart attacks, strokes and related cardiovascular events in the Lp(a)HORIZON trial.
   • The Lp(a)HORIZON trial enrolled more than 8,000 patients.
   • Pelacarsen succeeded in lowering Lp(a) levels.
   • Sefaxersen met its primary goal in a Phase 3 interim analysis for adults with primary IgA nephropathy.

   What to watch next

   • Final results of the Phase 3 IMAgINATION study for sefaxersen
   • Regulatory response to the Lp(a)HORIZON trial data
   • Updates on the paused rapcabtagene autoleucel trials
   Sources used for this update (3)

   1. finance.yahoo.com — Novartis (NVS) and Ionis (IONS)’ Big Cholesterol Bet Comes Up Short in Late-Stage Trial
   2. www.marketscreener.com — Ionis Pharmaceuticals, Roche Say Kidney Disease Drug Candidate Meets Main Goal in Phase 3 Study
   3. www.marketscreener.com — Ionis Pharmaceuticals, Inc. and Roche Holding Ltd. Report Positive Prespecified Interim Results from Phase 3 Imagination Study Evaluating Investigational Sefaxersen in Adults ...
   confidence 100%
6. 37d ago

   Novartis Shares Fall After Three Clinical Trial Setbacks

   Novartis shares declined following three clinical failures within eight days. On September 4, Novartis and Ionis Pharmaceuticals announced that pelacarsen failed its Phase 3 Lp(a)HORIZON trial primary endpoint of reducing cardiovascular event risk. Additionally, del-desiran missed Phase 3 goals. Two days prior to the pelacarsen announcement, Novartis paused eight trials for rapcabtagene autoleucel (rap-cel) after three patients died from immune effector cell-associated hemophagocytic syndrome. These setbacks raise questions regarding the company's $12 billion Avidity deal.

   Why it matters

   The company has pursued a growth strategy based on acquisitions and deals. The failure of pelacarsen follows a pattern of volatility that previously affected both Novartis and Amgen stock.

   What is confirmed

   • Pelacarsen failed to meet the primary endpoint of reducing cardiovascular event risk in the Phase 3 Lp(a)HORIZON trial.
   • Novartis paused eight trials for rapcabtagene autoleucel after three patients died from immune effector cell-associated hemophagocytic syndrome.
   • Del-desiran missed its Phase 3 goals.
   • Novartis and Ionis Pharmaceuticals acknowledged the pelacarsen trial failure on September 4.

   Still unconfirmed

   • The recent trial failures raise doubts over the $12 billion Avidity deal.

   What to watch next

   • Updates on the status of the Avidity deal
   • Further data on rapcabtagene autoleucel patient deaths
   Sources used for this update (2)

   1. www.ad-hoc-news.de — Novartis Faces a Reckoning: Three Trial Failures in Eight Days Test the Limits of Its Deal-Driven Growth Story
   2. www.genengnews.com — StockWatch: Novartis Loses More than Market Value After Phase III Failure
   confidence 100%
7. 42d ago

   Novartis Pelacarsen Fails Phase 3 Lp(a)HORIZON Trial

   Novartis announced that its experimental heart disease drug pelacarsen failed to meet its primary endpoint in the Phase 3 Lp(a)HORIZON trial. The drug successfully lowered lipoprotein(a) levels but failed to produce a statistically significant reduction in major cardiovascular events, heart attacks, strokes, or cardiovascular deaths among more than 8,000 patients with established cardiovascular disease. While the result disappointed cardiologists, Bank of America stated the failure does not break the Novartis investment thesis. The setback previously contributed to stock declines for Novartis and its peer Amgen.

   Why it matters

   Pelacarsen targeted lipoprotein(a) to prevent heart attacks in high-risk patients. This failure follows a separate late-stage study loss for Novartis's neuromuscular therapy del-desiran. Analysts are now evaluating how this result affects other companies developing similar therapies.

   What is confirmed

   • Pelacarsen lowered lipoprotein(a) levels but did not reduce major cardiovascular events in the Phase 3 Lp(a)HORIZON trial.
   • The trial included more than 8,000 patients.
   • The drug failed to significantly reduce strokes, heart attacks, or cardiovascular deaths.

   Still unconfirmed

   • Bank of America believes the failed study creates limited damage to the Novartis investment thesis.
   • Ionis Pharmaceuticals has an average overweight rating and a mean price target of $84.24 according to FactSet analysts.
   • Bank of America adjusted its price target for Ionis Pharmaceuticals to $91 from $93.

   What to watch next

   • Clinical trial results from Amgen or Ionis for competing lipoprotein(a) therapies
   • Further analyst revisions of price targets for Ionis Pharmaceuticals
   Sources used for this update (4)

   1. www.marketscreener.com — BofA Adjusts Price Target on Ionis Pharmaceuticals to $91 From $93
   2. medicalxpress.com — Cardiologists expected this new drug to prevent heart attacks—so why did it fail?
   3. www.thecardiologyadvisor.com — Pelacarsen Misses CV Risk Endpoint in Lp(a)HORIZON Trial
   4. finance.yahoo.com — BofA Sees Limited Damage from Novartis (NVS)’ Pelacarsen Miss While Citi Looks Towards Ionis (IONS)
   confidence 95%
8. 44d ago

   Novartis Pelacarsen Fails Phase III Trial

   Novartis announced that its experimental heart disease drug pelacarsen missed its primary Phase III endpoint in a trial of more than 8,000 patients. Although the drug successfully lowered lipoprotein(a) levels, it failed to significantly reduce major cardiovascular events, cardiovascular deaths, heart attacks, or strokes. The setback triggered a stock decline for Novartis and pulled industry peer Amgen down on Wall Street due to investor fears over competing products in development. The Swiss drugmaker suffered a second clinical setback days later when its neuromuscular disease therapy del-desiran failed a separate late-stage study.

   Why it matters

   The consecutive late-stage trial failures highlight the high risks and capital demands involved in developing innovative biopharmaceutical products. Market analysts noted that the failure of pelacarsen has also sparked investor anxiety regarding similar competing pipeline assets. This includes Amgen's experimental therapy olpasiran, which targets the same patient pathway.

   What is confirmed

   • Novartis stated that pelacarsen did not significantly reduce major cardiovascular events in a trial of 8,323 patients.
   • Amgen shares plunged on Wall Street following the rival drug failure due to fears over competing products in development.

   Still unconfirmed

   • Novartis's consecutive late-stage trial failures could signal broader clinical risks for similar therapies in development across the industry.

   What to watch next

   • Data readouts and clinical updates for competing products in development, such as Amgen's olpasiran.
   • Further market reactions from biopharmaceutical industry peers following the back-to-back trial failures.
   Sources used for this update (5)

   1. allsci.com — Novartis’s pelacarsen misses Phase III endpoint despite deep Lp(a) lowering
   2. www.marketscreener.com — Amgen caught in the crossfire of Novartis' failure
   3. eu.36kr.com — Lessons from Novartis's two consecutive Phase III trial failures
   4. finance.yahoo.com — Amgen's Olpasiran May Still Show Heart-Risk Reduction Despite Novartis Miss, UBS Says
   5. www.findarticles.com — Novartis Pelacarsen Misses Phase III Cardiovascular Endpoint
   confidence 100%
9. 45d ago

   Novartis Drug Trial Failures Spark Doubts

   Novartis shares fell after the company announced consecutive late-stage clinical trial failures for two experimental therapies. The experimental heart disease drug pelacarsen successfully lowered lipoprotein(a) levels in more than 8,000 patients but failed to reduce cardiovascular deaths, heart attacks, or strokes. Days later, the Swiss drugmaker reported a second setback when its neuromuscular disease therapy del-desiran failed a late-stage study without delivering a statistically significant improvement. These back-to-back pipeline blows also triggered drops for industry peers such as Amgen.

   Why it matters

   The failure of pelacarsen raises broad industry questions regarding the efficacy of therapies targeting lipoprotein(a) to prevent heart disease. These clinical disappointments deliver a severe financial and strategic challenge to Novartis as it absorbs consecutive R&D setbacks within days. The results also impact competitors like Amgen and Lilly that are developing treatments in similar therapeutic spaces.

   What is confirmed

   • Novartis announced on Tuesday that its experimental drug del-desiran for a muscle-wasting disorder failed a late-stage study.
   • The pelacarsen trial enrolled more than 8,000 patients.
   • Pelacarsen lowered Lp(a) levels but did not reduce the risk of heart attacks, strokes, or cardiovascular death.
   • Amgen shares dropped following the disappointing trial results.

   What to watch next

   • Further disclosures from Novartis regarding the future development path of pelacarsen and del-desiran
   • Market performance and pipeline adjustments by competitor drugmakers such as Amgen and Lilly
   Sources used for this update (6)

   1. www.briefs.co — Novartis Heart Drug Misses Final Test, Jolting Hopes for a New Heart-Disease Approach
   2. wdez.com — Novartis sinks as muscle wasting drug setback delivers fresh pipeline blow
   3. finance.yahoo.com — Novartis pelacarsen Lp(a) trial fails, raising doubts for Amgen, Lilly
   4. pharmaphorum.com — Novartis falls sharply on another major R&D setback
   5. www.marketscreener.com — Novartis reports another clinical setback
   6. www.marketscreener.com — Novartis faces fresh blow to pipeline with del-desiran drug setback
   confidence 100%
10. 46d ago

    Novartis Pelacarsen Fails Phase 3 Trial for Cardiovascular Events

    Novartis shares fell more than 3% on Monday after the company announced that its experimental drug pelacarsen failed to meet the primary endpoint in the Lp(a)HORIZON trial. The late-stage study enrolled over 8,000 patients with lipoprotein(a) levels of 70mg/dL or above. Despite successfully lowering this genetic risk factor, the drug did not reduce the risk of strokes, heart attacks, or other major adverse cardiovascular events. The result raises industry concerns regarding the efficacy of therapies targeting Lp(a) to prevent heart disease.

    Why it matters

    Pelacarsen was designed to lower genetically driven cardiovascular risk factors. This failure marks the first Phase 3 outcomes trial for an Lp(a)-lowering drug. The outcome puts pressure on the Novartis pipeline and affects competitors developing similar heart disease treatments.

    What is confirmed

    • Novartis shares dropped 3% on Monday following the trial results.
    • The Lp(a)HORIZON trial enrolled more than 8,000 patients with elevated levels of lipoprotein(a).
    • Pelacarsen failed to demonstrate a reduction in the risk of heart attacks, strokes, and other serious cardiovascular events.
    • The trial population consisted of patients with Lp(a) of 70mg/dL or above.
    • Pelacarsen successfully lowered the genetically driven cardiovascular risk factor despite missing the primary endpoint.

    What to watch next

    • Analysis of specific patient subgroups within the Lp(a)HORIZON data
    • Regulatory responses from the FDA or EMA regarding Lp(a)-targeting therapies
    • Clinical trial updates from rivals developing competing cardiovascular drugs
    Sources used for this update (6)

    1. www.yahoo.com — Novartis’ pelacarsen fails to meet primary endpoint in Lp(a)HORIZON trial
    2. finance.yahoo.com — Novartis Shares Fall After Pelacarsen Misses Primary Goal in Late-Stage Heart Trial
    3. www.globalbankingandfinance.com — Novartis shares down as failed cholesterol drug study increases pipeline pressure
    4. blockonomi.com — Novartis (NVS) Stock Drops 3% as Pelacarsen Heart Drug Disappoints in Key Trial
    5. european-biotechnology.com — Novartis’ Lp(a) drug misses Phase 3 endpoint
    6. finance.yahoo.com — Amgen Shares Fall as Novartis' Pelacarsen Misses Phase III Study Goal
    confidence 100%
11. 46d ago

    Novartis Heart Disease Drug Pelacarsen Misses Primary Endpoint

    Novartis announced that its experimental heart disease drug, Pelacarsen, failed to meet its primary endpoint in a late-stage clinical trial. The study focused on cholesterol and was closely watched by the medical community. Following the announcement, after-hours stock prices for Novartis, Ionis, and Amgen declined. The failure has surprised cardiologists, as the drug was considered a key experimental treatment for heart disease.

    Why it matters

    Pelacarsen was developed as a potential treatment to lower cholesterol and reduce cardiovascular risk. Its failure removes a high-profile candidate from the pipeline of late-stage heart disease therapies.

    What is confirmed

    • The experimental heart disease drug Pelacarsen missed its primary endpoint.
    • The Pelacarsen study was a late-stage trial focused on cholesterol.
    • Stocks for Novartis, Ionis, and Amgen dropped in after-hours trading following the news.

    What to watch next

    • Detailed data release from the clinical trial
    • Official statements from Ionis regarding the partnership
    • Regulatory responses to the trial failure
    Sources used for this update (5)

    1. The New York Times — Another Heart Drug Fails, Shocking Cardiologists
    2. reuters.com — Ionis, Novartis' key experimental heart drug fails in late-stage trial
    3. WSJ — Novartis Says Pelacarsen Heart Disease Drug Missed Primary Endpoint
    4. WSJ — Novartis Says Closely Watched Cholesterol Study Failed to Meet Goal
    5. Yahoo Finance — NVS, IONS, AMGN Stocks Drop After-Hours — What’s Driving The Selloff?
    confidence 100%