Novartis, Ionis' experimental heart drug misses key goal in closely watched trial

Live updates

1. 22d ago

   Novartis and Ionis Pelacarsen Trial Misses Cardiovascular Goal

   Novartis and Ionis Pharmaceuticals reported that their Phase 3 Lp(a)HORIZON trial for the experimental drug pelacarsen missed its primary goal. While the therapy sharply lowered lipoprotein(a) protein levels in more than 8,000 patients, it failed to produce a statistically significant reduction in major cardiovascular events such as heart attacks, strokes, and cardiovascular deaths. The setback bruised the case for treatment classes built on genetic risk signals and triggered immediate financial losses, causing Ionis shares to drop 13.1% to $50 and Novartis shares to fall over three percent.

   Why it matters

   Pelacarsen is an antisense-based drug targeting lipoprotein(a) and was evaluated in a large-scale outcomes study involving over 8,300 subjects with cardiovascular disease and elevated Lp(a) levels. The failure disconnects biomarker reduction from actual clinical event reduction, raising hard questions about what DNA evidence can predict for heart disease. This unexpected result carries broader implications for the Lp(a) drug race and other developers relying on genetic targets.

   What is confirmed

   • The Phase 3 Lp(a)HORIZON trial for pelacarsen failed to meet its primary goal of reducing major cardiovascular events.
   • Pelacarsen successfully lowered lipoprotein(a) protein levels in a trial involving more than 8,000 patients.
   • The trial failure caused Ionis shares to fall 13.1% to $50 and Novartis shares to drop over three percent.
   • Ionis Pharmaceuticals and Roche stated that an interim phase 3 study analysis for sefaxersen met its primary goal in adults with primary IgA nephropathy.

   What to watch next

   • Further data releases or analyses from the Lp(a)HORIZON trial investigators
   • Strategic adjustments by companies developing competing treatments in the Lp(a) drug race
   Sources used for this update (10)

   1. Longevity.Technology — Novartis’ Lp(a) drug misses key cardiovascular goal
   2. www.marketscreener.com — Ionis Pharmaceuticals, Roche Say Kidney Disease Drug Candidate Meets Main Goal in Phase 3 Study
   3. finance.yahoo.com — Failure of Novartis’ Pelacarsen Could Reshape the Lp(a) Race ...
   4. nexi.fund — Pelacarsen Deeply Lowered Lp (a). Cardiovascular Events ...
   5. www.el-fondo.com — Novartis Faces Phase 3 Setback in Pelacarsen Trial
   6. imedic.health — Pelacarsen Heart Trial: What Does the Lp (a) Setback | iMedic
   7. www.insidermonkey.com — Failure of Novartis’ Pelacarsen Could Reshape the Lp(a) Race ...
   8. biotechmorning.com — Genetics Said These Heart Drugs Should Work. The Patients ...
   9. pharmaphorum.com — Dosing prompts Novartis to tap Ionis for second Lp(a) drug
   10. shicheng-guo.github.io — Clinical Trial Updates — Shicheng Guo, Ph.D.
   confidence 100%
2. 34d ago

   Pelacarsen Phase 3 trial fails to reduce major cardiovascular events

   Novartis and Ionis Pharmaceuticals reported that the Phase 3 Lp(a)HORIZON trial for pelacarsen failed to meet its primary goal. While the experimental drug successfully lowered Lp(a) protein levels, it did not reduce the incidence of strokes, heart attacks, or cardiovascular deaths among more than 8,000 patients. The failure triggered a sharp decline in stock prices for both companies, with Ionis shares falling 13.1% to $50 and Novartis shares dropping over three percent on Monday. The results disappointed cardiologists who viewed the therapy as a potential tool for preventing heart attacks.

   Why it matters

   Lp(a) is a specific type of cholesterol that current medications struggle to target. This trial sought to prove that lowering these levels directly improves patient outcomes. The setback increases scrutiny of Novartis CEO Vas Narasimhan's pipeline and acquisition plans.

   What is confirmed

   • The Phase 3 Lp(a)HORIZON trial of pelacarsen failed to reduce major cardiovascular events.
   • Pelacarsen successfully lowered Lp(a) protein levels.
   • The trial included over 8,000 patients.
   • Ionis Pharmaceuticals shares fell 13.1% to $50 following the announcement.
   • Novartis shares dropped more than three percent on Monday.

   What to watch next

   • Official statements from Novartis regarding future pipeline adjustments
   • Analysis of the Lp(a)HORIZON data to determine why protein reduction did not prevent events
   Sources used for this update (2)

   1. www.insidermonkey.com — Novartis (NVS) and Ionis (IONS)’ Big Cholesterol Bet Comes Up Short in Late-Stage Trial
   2. finance.yahoo.com — Ionis Pharmaceuticals (IONS) Dropped, What Is Behind The Latest Attention?
   confidence 100%
3. 37d ago

   Novartis and Ionis heart drug pelacarsen fails Phase 3 trial

   Pelacarsen failed its primary endpoint in a Phase 3 trial involving over 8,000 patients. The drug lowered Lp(a) protein levels but did not reduce heart attacks, strokes, or cardiovascular deaths. Novartis announced the results, which disappointed cardiologists who expected the therapy to be a major tool against heart attacks. Following the news, Ionis Pharmaceuticals shares fell 13.1% to $50 and Novartis shares dropped more than three percent on Monday. The failure puts pressure on Novartis CEO Vas Narasimhan regarding pipeline and acquisition strategies.

   Why it matters

   Pelacarsen was developed as a targeted therapy to lower specific proteins linked to cardiovascular risk. Its failure leaves a gap in the pipeline for Novartis and Ionis. Analysts continue to track the financial impact on Ionis, which holds an average overweight rating.

   What is confirmed

   • Pelacarsen failed its primary endpoint in a Phase 3 trial of over 8,000 patients.
   • The drug lowered Lp(a) protein levels but failed to reduce cardiovascular deaths, strokes, or heart attacks.
   • Ionis Pharmaceuticals shares dropped 13.1% to $50 on Monday.
   • Novartis shares fell more than three percent on Monday.

   Still unconfirmed

   • BofA adjusted its price target on Ionis Pharmaceuticals to $91 from $93.
   • FactSet analysts report a mean price target of $84.24 and an average overweight rating for Ionis Pharmaceuticals.

   What to watch next

   • Official statements from Novartis regarding the future of the pelacarsen program
   • Further analyst price target adjustments 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.marketscreener.com — HSBC Adjusts Price Target on Eli Lilly and Co. to $940 From $850, Keeps Reduce Rating
   4. www.marketscreener.com — Eli Lilly Looks to Channel GLP-1 Revenue Into Women's Health
   confidence 90%
4. 45d ago

   Pelacarsen trial failure hits Novartis and Ionis shares

   Novartis and Ionis Pharmaceuticals experimental heart drug pelacarsen failed its primary endpoint in a Phase 3 trial. While the therapy lowered Lp(a) protein levels in over 8,000 patients, it failed to reduce cardiovascular deaths, strokes, or heart attacks. Ionis Pharmaceuticals shares dropped 13.1% to $50, while Novartis shares fell more than three percent on Monday. The result creates pipeline challenges for Novartis and increases pressure on CEO Vas Narasimhan regarding the company's acquisition strategy and overall pipeline.

   Why it matters

   Pelacarsen was viewed as a potential blockbuster treatment for cardiovascular disease. This failure follows other drug trial setbacks for Novartis. The outcome may also cast doubt on similar efforts by competitors Amgen and Lilly.

   What is confirmed

   • Pelacarsen lowered Lp(a) levels in more than 8,000 patients but did not reduce the risk of heart attacks, strokes, or cardiovascular death.
   • Ionis Pharmaceuticals shares fell 13.1% to $50.
   • Novartis shares dropped more than three percent on Monday.

   Still unconfirmed

   • The trial failure raises doubts for similar programs at Amgen and Lilly.
   • The setbacks impact the acquisition strategy of Novartis.

   What to watch next

   • Upcoming data from Novartis muscle-wasting therapy
   • Official response from CEO Vas Narasimhan regarding pipeline strategy
   Sources used for this update (4)

   1. finance.yahoo.com — Novartis pelacarsen Lp(a) trial fails, raising doubts for Amgen, Lilly
   2. finance.yahoo.com — Ionis Pharmaceuticals Shares Fall After Pelacarsen Phase 3 Trial Misses Primary Endpoint
   3. www.globalbankingandfinance.com — Novartis twin drug trial setbacks pile pressure on pipeline, CEO
   4. www.marketscreener.com — Cloverleaf Bio announced that it has received $33 million in funding from a group of investors
   confidence 90%
5. 46d ago

   Novartis and Ionis Heart Drug Pelacarsen Fails Late-Stage Trial

   Novartis and Ionis Pharmaceuticals face investor pressure after their experimental cardiovascular therapy, pelacarsen, missed its primary endpoint in a late-stage trial. Although the treatment successfully reduced levels of a protein particle known as Lp(a), the study did not demonstrate a significant reduction in cardiovascular events. The setback triggered a drop in Novartis shares of more than three percent on Monday. The failure deals a blow to expectations for the drug as a potential blockbuster treatment and heightens pressure on upcoming data from a muscle-wasting therapy.

   Why it matters

   Pelacarsen was designed to lower cardiovascular risk by targeting lipoprotein(a). The failure of the late-stage trial disrupts expectations for the Swiss drugmaker's pipeline and creates immediate hurdles for upcoming drug data evaluations. Separately, in unrelated pharmaceutical developments, Novo Nordisk reported positive late-stage trial results for semaglutide in younger patients.

   What is confirmed

   • Novartis shares fell more than 3% on Monday after its cholesterol drug failed in a closely watched study.
   • Novartis announced that pelacarsen, its investigational cardiovascular therapy targeting lipoprotein(a), failed to meet the primary endpoint.
   • The drug aimed to lower cardiovascular risk by reducing levels of a protein particle called Lp(a).
   • Novo Nordisk stated that 40.4% of children aged 6 to under 12 treated with semaglutide were no longer classified as having obesity after 68 weeks.

   Still unconfirmed

   • Investors viewed pelacarsen as a blockbuster treatment prior to the trial failure.

   What to watch next

   • Upcoming data releases from Novartis regarding its acquired muscle-wasting therapy
   • Further market response to the failed pelacarsen late-stage cardiovascular outcome study
   Sources used for this update (8)

   1. pharma.economictimes.indiatimes.com — Novartis, Ionis' experimental heart drug misses key goal in closely watched trial
   2. pharma.economictimes.indiatimes.com — News From Reuters
   3. www.ksl.com — Novartis shares down as failed cholesterol drug study increases pipeline pressure
   4. www.ksl.com — Novo Nordisk says semaglutide cut obesity rates in young children in trial
   5. finance.yahoo.com — Novartis Late-Stage Cardiovascular Outcome Study Fails, Stock Down
   6. finance.biggo.com — Novartis Heart Drug Pelacarsen Fails Late-Stage Trial, Sending Shares Down
   7. www.ksl.com — Waves of layoffs are sweeping across Utah. Here's what workers losing their jobs should know
   8. www.ksl.com — Switzerland's Sandoz pivots to biosimilars, expects rapid mid-term growth
   confidence 95%
6. 48d ago

   Novartis, Ionis' heart drug fails in late-stage trial

   Novartis and Ionis Pharmaceuticals' experimental heart drug, pelacarsen, failed to meet its primary goal in a closely watched late-stage trial. The drug aimed to lower cardiovascular risk by reducing levels of a protein particle called Lp(a). Despite achieving this, the trial did not show a significant reduction in cardiovascular events. This development has significant implications for cardiovascular drug research.

   Why it matters

   The failure of pelacarsen is a setback for Novartis and Ionis, as well as for the emerging class of heart medicines targeting Lp(a). Cardiovascular disease remains a major public health concern, and the development of effective treatments is crucial. The trial's results were unexpected, as previous research suggested that lowering Lp(a) levels could help reduce cardiovascular risk.

   What is confirmed

   • Novartis and Ionis Pharmaceuticals' experimental heart drug, pelacarsen, failed to meet its primary goal in a late-stage trial.
   • The drug aimed to lower cardiovascular risk by reducing levels of a protein particle called Lp(a).
   • Despite achieving this, the trial did not show a significant reduction in cardiovascular events.
   • The failure of pelacarsen has significant implications for cardiovascular drug research.

   What to watch next

   • Further analysis of the trial data to understand why pelacarsen failed to meet its primary goal
   • Decisions on the future development of pelacarsen and similar drugs
   • Impact on Novartis, Ionis, and Amgen's stock prices and future research plans
   Sources used for this update (8)

   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. STAT — Novartis’ experimental cardiovascular drug fails a pivotal study
   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?
   6. fiercebiotech.com — Novartis Lp(a) drug fails to lower cardiovascular risk in closely watched ph. 3 trial
   7. finance.yahoo.com — Novartis, Ionis drug failure spurs questions about an emerging class of heart medicines
   8. srnnews.com — US phases out $45M annual support for Namibia HIV program
   confidence 100%