Merck Data at ACC.25 Highlight Innovative Advancements and Commitment to Improving Outcomes for People Living with Cardiovascular Disease

Rhea-AI Summary

Merck (MRK) announced new clinical and outcomes research data presentations at the American College of Cardiology's Annual Scientific Session (ACC.25) in Chicago, March 29-31. The highlight is the Phase 3 ZENITH trial results for WINREVAIR™ (sotatercept-csrk) in treating pulmonary arterial hypertension (PAH) patients, which concluded early due to overwhelming efficacy.

The company will present four outcomes research presentations on cholesterol management, including real-world data on healthcare utilization costs for dyslipidemia treatment and LDL-C measurement patterns. Research focuses on barriers to achieving LDL-C goals, noting that nearly two-thirds of individuals don't reach their targets. The presentations include a systematic review of residual atherosclerotic cardiovascular disease risk in statin users and a model estimating the impact of lipid-lowering therapy treatment patterns.

Positive

• ZENITH Phase 3 trial concluded early due to overwhelming efficacy
• Expanding research portfolio in cardiovascular disease management
• Strong clinical trial results supporting WINREVAIR's effectiveness in PAH treatment

Negative

• Nearly two-thirds of patients fail to reach LDL-C goals in current treatment approaches

Insights

Financial Analyst

Merck's announcement about the Phase 3 ZENITH trial for WINREVAIR™ represents a significant clinical development with positive commercial implications. The trial, evaluating WINREVAIR in high-risk pulmonary arterial hypertension (PAH) patients with WHO functional class III or IV, was concluded early due to "overwhelming efficacy" - a rare occurrence in clinical trials that signals exceptionally positive results.

This development is particularly meaningful as WINREVAIR (sotatercept-csrk) received FDA approval just recently in March 2024. The ZENITH data could potentially expand the drug's label to include the most severe PAH patients, representing a critical commercial opportunity in a high-unmet-need population. While PAH affects relatively few patients, treatments command premium pricing due to orphan drug status, making this a potentially valuable addition to Merck's revenue stream.

The company's cardiovascular franchise may also benefit from the four presentations on cholesterol management, particularly as these highlight substantial treatment gaps - with nearly 66% of patients failing to reach LDL-C goals. This research could support Merck's existing or pipeline products in the larger cardiovascular market.

While the press release doesn't provide the actual efficacy data (to be presented March 29-31), the early trial termination language suggests potentially practice-changing results that could strengthen Merck's position in the specialized PAH treatment landscape.

Healthcare Specialist

The early termination of Merck's ZENITH trial due to "overwhelming efficacy" represents a potential medical breakthrough for the most vulnerable PAH patients. WHO functional class III and IV patients face mortality rates as high as 30% within three years despite current therapies, making any significant efficacy improvement genuinely lifesaving.

WINREVAIR's mechanism as an activin signaling inhibitor represents a novel therapeutic approach in PAH. Unlike traditional vasodilators, it targets the underlying vascular remodeling process, potentially modifying disease progression rather than just managing symptoms. The fact that this approach showed such compelling results in the highest-risk patients suggests potential disease-modifying properties that existing PAH therapies lack.

The clinical significance extends beyond PAH, as this activin signaling pathway has implications across multiple cardiopulmonary conditions. Success here might validate this mechanism for exploration in related diseases, expanding the potential therapeutic applications.

The cholesterol management research also addresses a critical healthcare challenge - the persistent treatment gap in lipid management despite clear guidelines and available therapies. This research may highlight systemic barriers to effective cholesterol management that impact cardiovascular outcomes nationwide.

While we await the specific data at ACC.25, the language suggests potentially practice-changing results that could establish WINREVAIR as a cornerstone therapy for high-risk PAH patients who currently have effective options.

Data from Phase 3 ZENITH trial evaluating WINREVAIR™ (sotatercept-csrk) when added to background therapy in adults with pulmonary arterial hypertension (PAH, Group 1 PH) WHO* functional class (FC) III or IV at high risk of mortality to be featured as a late-breaking presentation

New research highlights gaps and barriers for patients to achieve low-density lipoprotein cholesterol (LDL-C) goals and the impact on patient outcomes and healthcare utilization, underscoring the urgency to treat to LDL-C goals

RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that new clinical and outcomes research data will be presented at the American College of Cardiology’s Annual Scientific Session and Expo (ACC.25) in Chicago from March 29-31. Data being shared at the meeting highlights how Merck is advancing research focused on improving outcomes for even more patients living with cardiovascular disease, through the evaluation of approved treatments, as well as data supporting opportunities for improved disease management in a real-world setting.

Merck will present results from the Phase 3 ZENITH trial evaluating WINREVAIR™ (sotatercept-csrk) when added to background therapy in adults with pulmonary arterial hypertension (PAH, Group 1 PH) WHO* functional class (FC) III or IV at high risk of mortality. These results will be shared as an oral presentation in a late-breaking session on Monday, March 31 during the Clinical and Investigative Horizons I session from 10:00-11:00 a.m. ET/9:00-10:00 a.m. CT (LBA402-16).

“Merck has a long legacy of making an impact in cardiovascular disease, and since we introduced our first therapy more than 60 years ago, we have remained committed to advancing innovative research to better understand and help improve the lives of patients with cardiovascular disease,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “We are particularly thrilled to share research from the Phase 3 ZENITH trial evaluating WINREVAIR in PAH as a late-breaking presentation. ZENITH is the first study in PAH in which the interim analysis led to an early conclusion due to overwhelming efficacy and adds to the growing body of data evaluating WINREVAIR in a broad spectrum of patients with PAH.”

Four outcomes research presentations focused on cholesterol management from Merck will also be shared during the meeting. These include two presentations evaluating real-world data: the first highlights healthcare utilization and costs among patients treated for dyslipidemia in a U.S. integrated delivery system and the second provides insights into low-density lipoprotein (LDL-C) measurement patterns. Two additional presentations include a model to estimate the impact of treatment patterns for adults on lipid lowering therapy (LLT), as well as a systematic literature review and meta-analysis of residual atherosclerotic cardiovascular disease (ASCVD) risk among those treated with statin therapy.

“Our commitment to improving the lives of patients includes driving analyses of observational data to provide valuable insights into the barriers to achieving LDL-C goals in order to manage atherosclerotic cardiovascular disease,” said Dr. Bjorn Oddens, senior vice president and head of value and implementation, Merck Research Laboratories. “Low-density lipoprotein cholesterol is an established and modifiable risk factor for the development of atherosclerotic cardiovascular disease, a disease which accounts for 85 percent of cardiovascular deaths, but nearly two-thirds of individuals do not reach their LDL-C goals. Research presented at ACC aims to provide the cardiovascular community with insights into existing gaps in management, the impact on patient outcomes and healthcare systems and opportunities for improvement, with the ultimate goal of helping more patients better manage their LDL-C.”

Key data from Merck to be presented at ACC.25:

• First presentation of results from the Phase 3 ZENITH trial evaluating WINREVAIR when added to background therapy in patients with PAH FC III or IV at high risk of mortality (Abstract #LBA402-16; Clinical and Investigative Horizons I);
• A systematic review and meta-analysis evaluating the residual ASCVD risk in statin users in the real-world setting (Abstract #1062-36; Cardiovascular Disease Prevention 03);
• Real-world data evaluating the frequency of LDL-C measurement and its association with patient characteristics among adults prescribed LLT in the U.S. (Abstract #1062-19; Cardiovascular Disease Prevention 03);
• Presentation of a model to estimate the benefits of non-statin LLT use in terms of clinical (LDL-C and ASCVD event reduction) and economic (cost reduction) impact on the U.S. health system (Abstract #977-03; Costs of Cardiovascular Preventive Care: Implications Across Stakeholders);
• Poster presentation estimating the association between healthcare resource use and treatment patterns among adults on LLT across various levels of risk in a large U.S. health system (Abstract #977-15; Costs of Cardiovascular Preventive Care: Implications Across Stakeholders).

Details on key abstracts for Merck:

PAH
Efficacy and safety of sotatercept in high-risk patients with pulmonary arterial hypertension: results from ZENITH Phase 3 trial. M. Humbert.	Abstract #LBA402-16; Clinical and Investigative Horizons I on Monday, March 31, 10:00-11:00 a.m. ET/9:00-10:00 a.m. CT
Burden of pulmonary arterial hypertension (PAH) on women across regions: multinational patient qualitative study. I. Preston.	Abstract #66; Pulmonary Vascular Disease 07 on Sunday, March 30, 11:30 a.m.-12:30 p.m. ET/10:30-11:30 a.m. CT

Atherosclerotic Cardiovascular Disease
Clinical and economic burden of adding ezetimibe therapy in patients with atherosclerotic cardiovascular disease. T. Galvain.	Abstract #977-03; Costs of Cardiovascular Preventive Care: Implications Across Stakeholders on Sunday, March 30, 3:00-5:00 p.m. ET/2:00-4:00 p.m. CT
Healthcare resource use (HCRU) and costs among adults on lipid lowering therapy (LLT) with and at risk for atherosclerotic cardiovascular disease (ASCVD) events in a US integrated delivery system. A. Victores.	Abstract #977-15; Costs of Cardiovascular Preventive Care: Implications Across Stakeholders on Sunday, March 30, 3:00-5:00 p.m. ET/2:00-4:00 p.m. CT
Frequency of LDL-C measurement among patients prescribed lipid lowering therapy (LLT) in the US. A. Victores.	Abstract #1062-19; Cardiovascular Disease Prevention 03 on Saturday, March 29, 1:30-2:30 p.m. ET/12:30-1:30 p.m. CT
Residual atherosclerotic cardiovascular disease risk in statin users – a systematic review and meta-analysis. A.H. Watanabe.	Abstract #1062-36; Cardiovascular Disease Prevention 03 on Saturday, March 29, 1:30-2:30 p.m. ET/12:30-1:30 p.m. CT

Heart Failure
Exploring barriers to optimization of medical therapy and the role of checklist-based decision support in heart failure. B. Montelaro.	Abstract #1132-177; Heart Failure and Cardiomyopathies 06 on Sunday, March 30, 10:00-11:00 a.m. ET/9:00-10:00 a.m. CT
Worsening heart failure events are associated with new initiation of guideline directed medical therapy among patients with heart failure with reduced ejection fraction. A. Ambrosy.	Abstract #1215-146; Heart Failure and Cardiomyopathies 10 On Sunday, March 30, 4:00-5:00 p.m. ET/3:00-4:00 p.m. CT

*World Health Organization

About WINREVAIR^™ (sotatercept-csrk) for injection, for subcutaneous use, 45 mg, 60 mg

WINREVAIR is FDA-approved for the treatment of adults with pulmonary arterial hypertension (PAH, Group 1 PH) to increase exercise capacity, improve WHO functional class (FC) and reduce the risk of clinical worsening events. WINREVAIR is the first activin signaling inhibitor therapy approved to treat PAH. WINREVAIR improves the balance between pro-proliferative and anti-proliferative signaling to modulate vascular proliferation. In preclinical models, WINREVAIR induced cellular changes that were associated with thinner vessel walls, partial reversal of right ventricular remodeling, and improved hemodynamics.

WINREVAIR is the subject of a licensing agreement with Bristol Myers Squibb.

Selected Safety Information for WINREVAIR in the U.S.

WINREVAIR may increase hemoglobin (Hgb). Severe erythrocytosis may increase the risk of thromboembolic events or hyperviscosity syndrome. Monitor Hgb before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter, to determine if dose adjustments are required.

WINREVAIR may decrease platelet count. Severe thrombocytopenia may increase the risk of bleeding. Thrombocytopenia occurred more frequently in patients also receiving prostacyclin infusion. Do not initiate treatment if platelet count is <50,000/mm3. Monitor platelets before each dose for the first 5 doses, or longer if values are unstable, and periodically thereafter to determine whether dose adjustments are required.

In clinical studies, serious bleeding (e.g., gastrointestinal, intracranial hemorrhage) was reported in 4% of patients taking WINREVAIR and 1% of patients taking placebo. Patients with serious bleeding were more likely to be on prostacyclin background therapy and/or antithrombotic agents, or have low platelet counts. Advise patients about signs and symptoms of blood loss. Do not administer WINREVAIR if the patient is experiencing serious bleeding.

WINREVAIR may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use an effective method of contraception during treatment with WINREVAIR and for at least 4 months after the final dose. Pregnancy testing is recommended for females of reproductive potential before starting WINREVAIR treatment.

Based on findings in animals, WINREVAIR may impair female and male fertility. Advise patients on the potential effects on fertility.

The most common adverse reactions occurring in the phase 3 clinical trial (≥10% for WINREVAIR and at least 5% more than placebo) were headache (24.5% vs 17.5%), epistaxis (22.1% vs 1.9%), rash (20.2% vs 8.1%), telangiectasia (16.6% vs 4.4%), diarrhea (15.3% vs 10.0%), dizziness (14.7% vs 6.2%), and erythema (13.5% vs 3.1%).

Because of the potential for serious adverse reactions in the breastfed child, advise patients that breastfeeding is not recommended during treatment with WINREVAIR, and for 4 months after the final dose.

Merck’s focus on cardiovascular disease

Merck has a long history of making an impact in cardiovascular disease. More than 60 years ago, we introduced our first cardiovascular therapy – and our scientific efforts to understand cardiovascular-related disorders have continued. Cardiovascular disease continues to be one of the most serious health challenges of the 21st century. Approximately 19 million people across the globe die every year, and in the United States one person dies every 36 seconds from cardiovascular disease.

Advancements in the treatment of cardiovascular disease can make a critical difference for patients around the world. At Merck, we strive for scientific excellence and innovation in all stages of research, from discovery through approval and life cycle management. We work with experts throughout the cardiovascular and pulmonary community to advance research that can help improve the lives of patients globally.

About Merck

At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking Statement of Merck & Co., Inc., Rahway, N.J., USA

This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

Please see Prescribing Information for WINREVAIR (sotatercept-csrk) at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_pi.pdf , Patient Information for WINREVAIR at http://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ppi.pdf , and Instructions for Use for WINREVAIR (1-vial kit, 2-vial kit) at https://www.merck.com/product/usa/pi_circulars/w/winrevair/winrevair_ifu_1-vial_2-vial_kits.pdf.

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Source: Merck & Co., Inc.

FAQ

What are the key findings from Merck's ZENITH trial for WINREVAIR presented at ACC.25?

The Phase 3 ZENITH trial for WINREVAIR in PAH patients was concluded early due to overwhelming efficacy when added to background therapy in adults with WHO functional class III or IV at high risk of mortality.

How many research presentations is MRK delivering on cholesterol management at ACC.25?

Merck is presenting four outcomes research presentations focused on cholesterol management, including real-world data analysis and systematic reviews.

What percentage of patients fail to reach their LDL-C goals according to Merck's research?

According to the research, nearly two-thirds of individuals do not reach their LDL-C (low-density lipoprotein cholesterol) goals.

When and where is Merck presenting the ZENITH trial results at ACC.25?

Merck will present the ZENITH trial results on March 31 during the Clinical and Investigative Horizons I session from 10:00-11:00 a.m. ET in Chicago.