Corneal Ulcer Market Research Report 2025-2035 | Overview and Ecosystem, Epidemiology, Key Trends, Impact Analysis, Regulatory Landscape, Pipeline Analysis, Market Dynamics – ResearchAndMarkets.com




Corneal Ulcer Market Research Report 2025-2035 | Overview and Ecosystem, Epidemiology, Key Trends, Impact Analysis, Regulatory Landscape, Pipeline Analysis, Market Dynamics – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Corneal Ulcer Market – A Global and Regional Analysis: Focus on Drug Class and Region – Analysis and Forecast, 2025-2035” report has been added to ResearchAndMarkets.com’s offering.

The corneal ulcer treatment market is poised for significant growth, driven by advancements in novel therapies, including regenerative treatments and targeted drug delivery systems. As awareness of corneal ulcers continues to rise, particularly in regions with high rates of contact lens usage and microbial infections, demand for effective treatments is expected to grow.

Additionally, improved healthcare infrastructure, especially in emerging markets, will enhance accessibility to advanced care. The growing focus on personalized medicine, combined with favourable reimbursement policies, will further support market expansion. With continued patient education and the development of innovative, more effective therapies, the corneal ulcer treatment market is well-positioned to address the rising need for specialized and targeted treatment options.

Growth in the corneal ulcer treatment market is supported by the increasing recognition of the condition’s impact on patients’ quality of life, especially due to its potential to lead to blindness if untreated. The market is evolving as healthcare providers and pharmaceutical companies focus on early diagnosis and intervention, offering a broader range of treatment options to improve patient outcomes and reduce recurrence of infections. Key therapeutic categories include topical antibiotics, antivirals, antifungals, corticosteroids, and regenerative therapies such as platelet-rich plasma (PRP) and recombinant human nerve growth factors, which are gaining attention for their potential to accelerate corneal healing and improve recovery rates.

Improved healthcare infrastructure in emerging markets, rising awareness among patients, and enhanced access to treatment are significant contributors to market growth. Furthermore, favourable reimbursement policies in developed regions are enabling greater access to advanced treatments, which further drives market expansion. The ongoing development of more targeted therapies and personalized treatment approaches, especially for patients with chronic or recurrent corneal ulcers, is expected to open new opportunities for market growth.

Advancements in drug delivery technologies, such as sustained-release ocular implants, nanoparticle-based therapies, and innovative eye drops, are playing a crucial role in expanding the therapeutic landscape for corneal ulcers. These innovations are designed to improve drug bioavailability, enhance treatment adherence, and minimize side effects, which are key challenges in the current treatment regimen. Additionally, personalized medicine, which takes into account individual patient profiles and genetic factors, is poised to revolutionize the management of corneal ulcers by offering more precise and effective treatment options.

Despite the promising growth prospects, the corneal ulcer market faces several challenges, including the high cost of advanced treatments, limited availability of specialized ophthalmologists, and inconsistent patient adherence to long-term treatment regimens. Moreover, the complex nature of microbial infections and the emergence of antibiotic-resistant pathogens complicate treatment efforts and may limit the effectiveness of current therapies. Regulatory hurdles and lengthy approval timelines for new treatments may also delay the availability of breakthrough therapies, further impacting market dynamics.

The competitive landscape of the corneal ulcer treatment market is characterized by the active involvement of leading pharmaceutical companies, biotechnology firms, and research institutions. Strategic partnerships, mergers, and acquisitions are common as stakeholders seek to enhance their product portfolios and accelerate research into more effective treatments. Investments in research and development, particularly in regenerative therapies, drug delivery systems, and novel antimicrobial agents, will play a key role in shaping the future of the market, aiming to improve clinical efficacy and provide better patient-centric care.

Looking forward, the global corneal ulcer treatment market is poised to continue its growth, driven by the rising incidence of corneal infections, advancements in treatment modalities, and a growing emphasis on patient education and disease prevention. The integration of digital health technologies, such as mobile apps for tracking treatment progress and remote consultations, is expected to improve treatment adherence and facilitate better disease management. With continued focus on personalized medicine and the development of more innovative therapeutic options, the corneal ulcer market is positioned to enhance patient outcomes and quality of life, providing a brighter future for those affected by this debilitating condition worldwide.

Competitive Landscape

• AbbVie Inc. (Allergan plc)
• Baxter International Inc.
• Bayer AG
• Dr. Reddy’s Laboratories Ltd.
• Eli Lilly and Company
• Merck & Co., Inc.
• Novartis AG
• Pfizer Inc.
• Sun Pharmaceutical Industries Ltd.
• Teva Pharmaceutical Industries Ltd.

Key Topics Covered:

1. Global Corneal Ulcer Market: Industry Analysis

• 1.1 Market Overview and Ecosystem
• 1.2 Epidemiological Analysis
• 1.3 Key Market Trends
• 1.3.1 Impact Analysis
• 1.4 Regulatory Landscape
• 1.5 Pipeline Analysis
• 1.6 Market Dynamics
• 1.6.1 Overview
• 1.6.2 Market Drivers
• 1.6.3 Market Restraints
• 1.6.4 Market Opportunities

2. Global Corneal Ulcer Market (by Drug Class), Value ($million), 2023-2035

• 2.1 Antibiotics
• 2.2 Antifungals
• 2.3 Antivirals
• 2.4 Corticosteroids
• 2.5 Others

3. Global Corneal Ulcer Market (by Region), Value ($Million), 2023-2035

• 3.1 North America
• 3.1.1 Market Dynamics
• 3.1.2 Market Sizing and Forecast
• 3.1.3 North America Corneal Ulcer Market, by Country ($Million), 2023-2035
• 3.1.3.1 U.S.
• 3.1.3.2 Canada
• 3.2 Europe
• 3.2.1 Market Dynamics
• 3.2.2 Market Sizing and Forecast
• 3.2.3 Europe Corneal Ulcer Market, by Country ($Million), 2023-2035
• 3.2.3.1 U.K.
• 3.2.3.2 France
• 3.2.3.3 Germany
• 3.2.3.4 Italy
• 3.2.3.5 Spain
• 3.2.3.6 Rest-of-Europe
• 3.3 Asia-Pacific
• 3.3.1 Market Dynamics
• 3.3.2 Market Sizing and Forecast
• 3.3.3 Asia-Pacific Corneal Ulcer Market, by Country ($Million), 2023-2035
• 3.3.3.1 Japan
• 3.3.3.2 China
• 3.3.3.3 India
• 3.3.3.4 Australia
• 3.3.3.5 South Korea
• 3.3.3.6 Rest-of-Asia-Pacific
• 3.4 Rest-of-the-World
• 3.4.1 Market Dynamics
• 3.4.2 Market Sizing and Forecast Rest-of-the-World Corneal Ulcer Market, by Type ($Million), 2023-2035
• 3.4.3 Rest-of-the-World Corneal Ulcer Market, by Country ($Million), 2023-2035
• 3.4.3.1 Latin America
• 3.4.3.2 Middle East and Africa

4. Competitive Landscape and Company Profiles

• 4.1 Competitive Landscape
• 4.1.1 Mergers and Acquisitions
• 4.1.2 Partnership, Alliances and Business Expansion
• 4.1.3 New Offerings
• 4.1.4 Regulatory Activities
• 4.1.5 Funding Activities
• 4.2 Company Profiles
• 4.2.1 Overview
• 4.2.2 Top Products / Product Portfolio
• 4.2.3 Top Competitors
• 4.2.4 Target Customers/End-Users
• 4.2.5 Key Personnel
• 4.2.6 Analyst View

5. Research Methodology

For more information about this report visit https://www.researchandmarkets.com/r/mlqff0

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

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For E.S.T Office Hours Call 1-917-300-0470

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Dyslipidemia Market Research Report 2025-2035 | Expansion Fueled by Rising Cardiovascular Disease Burden, Lifestyle Risks, Early Diagnostics, and Preventive Health Initiatives – ResearchAndMarkets.com




Dyslipidemia Market Research Report 2025-2035 | Expansion Fueled by Rising Cardiovascular Disease Burden, Lifestyle Risks, Early Diagnostics, and Preventive Health Initiatives – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Dyslipidemia Market – A Global and Regional Analysis: Focus on Drug Class, Route of Administration, and Region – Analysis and Forecast, 2025-2035” report has been added to ResearchAndMarkets.com’s offering.

Dyslipidemia, characterized by abnormal levels of lipids such as cholesterol and triglycerides in the blood, significantly elevates the risk of cardiovascular diseases. Advances in lipid profiling, genetic testing, and risk assessment tools are enabling earlier and more precise diagnosis, thereby boosting demand for personalized therapeutic interventions. Statins remain the cornerstone of treatment, with newer drug classes such as PCSK9 inhibitors and RNA-based therapies gaining traction due to their efficacy in managing resistant cases.

However, challenges including patient non-compliance, side effects associated with long-term statin use, and high costs of novel therapies limit broader market penetration, especially in low and middle-income countries. Additionally, variations in treatment guidelines and healthcare infrastructure disparities complicate standardized care delivery. Nonetheless, growing government health initiatives, increased investment in cardiovascular research, and expanding awareness campaigns are driving improved screening and treatment rates.

Impact

Technological advancements in lipid diagnostics, genetic screening, and telemedicine platforms are significantly enhancing the early detection and personalized management of dyslipidemia. Innovations such as advanced lipid profiling, AI-driven risk prediction models, and wearable cardiovascular monitoring devices are improving the precision of disease assessment and treatment customization. Additionally, the integration of digital therapeutics and remote patient management is expanding access to care, particularly in underserved or rural areas, enabling continuous monitoring and better adherence to lipid-lowering therapies.

Statins hold the highest market share in the dyslipidemia market due to their proven efficacy, safety profile, and widespread clinical adoption as the first-line therapy for lowering LDL cholesterol and reducing cardiovascular risk. Extensive clinical trial evidence supports statins’ ability to significantly decrease morbidity and mortality associated with dyslipidemia-related conditions, which has solidified their position as the gold standard treatment globally.

Oral administration holds the highest market share in the dyslipidemia market, primarily because the majority of lipid-lowering drugs, including statins, fibrates, niacins, bile acid resins, and omega-3 fatty acids, are formulated for oral use. Oral medications offer greater patient convenience, ease of administration, and higher compliance compared to parenteral routes.

North America holds the highest market share in the dyslipidemia market, driven by its advanced healthcare infrastructure, high prevalence of cardiovascular diseases, and widespread adoption of lipid-lowering therapies. The region benefits from strong healthcare spending, well-established reimbursement frameworks, and early access to innovative treatments such as PCSK9 inhibitors.

How can this report add value to an organization?

Product/Innovation: This report provides comprehensive insights into the current trends in dyslipidemia, helping companies identify opportunities for drug and technology development. Organizations can leverage these insights to design therapies, medications, and platforms tailored to the needs of patients suffering from dyslipidemia, improving outcomes and enhancing market penetration.

Competitive: A detailed competitive landscape analysis helps organizations benchmark their market standing against key players. By understanding the strengths and weaknesses of competitors, companies can position themselves more effectively in the global dyslipidemia market.

Demand Drivers and Limitations

Drivers for the Global Dyslipidemia Market:

• Increasing cases of heart disease and stroke worldwide drive demand for effective lipid-lowering treatments.
• Lifestyle changes leading to obesity and diabetes contribute to higher dyslipidemia rates, boosting market growth
• Improved lipid profiling and genetic testing facilitate early diagnosis, increasing treatment uptake
• Public health initiatives promote regular cholesterol screening, leading to earlier intervention and therapy adoption

Limitations for the Global Dyslipidemia Market:

• Long-term medication adherence is often poor due to side effects and lack of symptom awareness, limiting treatment effectiveness
• Expensive drugs such as PCSK9 inhibitors restrict accessibility, especially in low- and middle-income countries.
• Adverse effects such as muscle pain and liver issues lead to discontinuation or reluctance in therapy initiation.

Key Market Players

• Amgen Inc.
• Sanofi S.A.
• Regeneron Pharmaceuticals, Inc.
• Pfizer Inc.
• Novartis AG
• Viatris Inc.
• AbbVie Inc.
• AstraZeneca plc
• Horizon Therapeutics plc
• Esperion Therapeutics, Inc.

Key Topics Covered:

1. Global Dyslipidemia Market: Industry Analysis

• 1.1 Market Overview and Ecosystem
• 1.2 Epidemiological Analysis
• 1.3 Key Market Trends
• 1.3.1 Impact Analysis
• 1.4 Regulatory Landscape
• 1.5 Pipeline Analysis
• 1.6 Market Dynamics
• 1.6.1 Overview
• 1.6.2 Market Drivers
• 1.6.3 Market Restraints
• 1.6.4 Market Opportunities

2. Global Dyslipidemia Market (by Drug Class), Value ($million), 2023-2035

• 2.1 Statins
• 2.2 Bile Acid Resins
• 2.3 Fibrates
• 2.4 Niacins
• 2.5 Omega-3 Fatty Acids
• 2.6 Others

3. Global Dyslipidemia Market (by Route of Administration), Value ($million), 2023-2035

• 3.1 Oral
• 3.2 Parenteral

4. Global Dyslipidemia Market (by Region), Value ($Million), 2023-2035

• 4.1 North America
• 4.1.1 Market Dynamics
• 4.1.2 Market Sizing and Forecast
• 4.1.3 North America Dyslipidemia Market, by Country ($Million), 2023-2035
• 4.1.3.1 U.S.
• 4.1.3.2 Canada
• 4.2 Europe
• 4.2.1 Market Dynamics
• 4.2.2 Market Sizing and Forecast
• 4.2.3 Europe Dyslipidemia Market, by Country ($Million), 2023-2035
• 4.2.3.1 U.K.
• 4.2.3.2 France
• 4.2.3.3 Germany
• 4.2.3.4 Italy
• 4.2.3.5 Spain
• 4.2.3.6 Rest-of-Europe
• 4.3 Asia-Pacific
• 4.3.1 Market Dynamics
• 4.3.2 Market Sizing and Forecast
• 4.3.3 Asia-Pacific Dyslipidemia Market, by Country ($Million), 2023-2035
• 4.3.3.1 Japan
• 4.3.3.2 China
• 4.3.3.3 India
• 4.3.3.4 Australia
• 4.3.3.5 South Korea
• 4.3.3.6 Rest-of-Asia-Pacific
• 4.4 Rest-of-the-World
• 4.4.1 Market Dynamics
• 4.4.2 Market Sizing and Forecast Rest-of-the-World Dyslipidemia Market, by Type ($Million), 2023-2035
• 4.4.3 Rest-of-the-World Dyslipidemia Disorder Market, by Country ($Million), 2023-2035
• 4.4.3.1 Latin America
• 4.4.3.2 Middle East and Africa

5. Competitive Landscape and Company Profiles

• 5.1 Competitive Landscape
• 5.1.1 Mergers and Acquisitions
• 5.1.2 Partnership, Alliances and Business Expansion
• 5.1.3 New Offerings
• 5.1.4 Regulatory Activities
• 5.1.5 Funding Activities
• 5.2 Company Profiles
• 5.2.1 Overview
• 5.2.2 Top Products / Product Portfolio
• 5.2.3 Top Competitors
• 5.2.4 Target Customers/End-Users
• 5.2.5 Key Personnel
• 5.2.6 Analyst View

6. Research Methodology

For more information about this report visit https://www.researchandmarkets.com/r/htp9qf

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

Chronic Inducible Urticaria (CIndU) Market Analysis Report 2025-2035 | Growth Driven by Rising Awareness, Immune Mechanism Insights, and Advancements in Biologic Therapies – ResearchAndMarkets.com




Chronic Inducible Urticaria (CIndU) Market Analysis Report 2025-2035 | Growth Driven by Rising Awareness, Immune Mechanism Insights, and Advancements in Biologic Therapies – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Chronic Inducible Urticaria Market – A Global and Regional Analysis: Focus on Drug Class and Region – Analysis and Forecast, 2025-2035” report has been added to ResearchAndMarkets.com’s offering.

The chronic inducible urticaria (CIndU) market is positioned for significant growth, driven by advancements in biologic therapies, targeted immunomodulatory treatments, and improved diagnostic tools that enhance the identification and management of this chronic skin condition. As awareness of CIndU increases, particularly regarding its diverse triggers such as cold, heat, and pressure, there is a growing demand for personalized treatment options tailored to individual triggers and immune profiles.

The rising prevalence of autoimmune and allergic conditions, combined with more widespread understanding of CIndU, is fuelling greater demand for effective therapies. Expanding healthcare access, especially in emerging markets with improving medical infrastructures, is expected to drive market growth. Furthermore, evolving reimbursement policies and the integration of precision medicine, which includes biomarker-based risk stratification and tailored treatment regimens.

Market growth is primarily driven by a deeper understanding of the disease’s underlying immune mechanisms, particularly its links to mast cell activation and immune system dysregulation. As awareness of CIndU increases among healthcare providers, especially in high-risk groups or patients with recurrent hives or unexplained allergic reactions, the demand for targeted treatments is set to rise. This is further accelerated by advancements in biologic therapies, particularly monoclonal antibodies such as omalizumab, which have shown promise in managing chronic spontaneous urticaria and are now being studied for CIndU.

Emerging diagnostic tools, including skin prick tests, cold stimulation tests, and electrodermal activity monitoring, are playing a key role in improving the speed and accuracy of diagnosis, reducing the time to treatment. Additionally, molecular diagnostics and biomarkers that identify specific triggers and immune profiles in patients are expected to further personalize and enhance treatment strategies. The evolution of these diagnostic modalities is allowing healthcare professionals to better identify and treat CIndU, leading to more effective management and improved patient outcomes.

Therapeutic strategies for CIndU are expanding, with antihistamines remaining the first-line treatment, complemented by biologic therapies and immunosuppressants for patients with moderate to severe disease. The increasing use of biologics, including omalizumab and dupilumab, which target key pathways involved in urticaria, is expected to significantly improve the quality of life for patients suffering from chronic urticaria. These biologics have shown clinical promise by reducing the frequency and severity of hives, and they represent a new era of treatment that is increasingly gaining traction.

Healthcare policy improvements, including better reimbursement frameworks for biologics and newer treatments, are driving market expansion. This is particularly evident in regions such as North America and Europe, where access to advanced therapies is improving. Additionally, updated clinical practice guidelines from organizations such as the American Academy of Dermatology (AAD) and the European Academy of Allergy and Clinical Immunology (EAACI) are promoting earlier and more proactive treatment strategies for CIndU, increasing patient access to specialized care and expanding eligibility for biologic therapies.

Technological advancements in patient monitoring and digital health are playing a growing role in the management of CIndU. Digital tools such as mobile apps that track symptoms and triggers, as well as telemedicine platforms that provide remote consultations, are enabling patients to better manage their disease on a day-to-day basis. These innovations also support long-term management by helping both patients and healthcare providers keep track of flare-ups, identify environmental triggers, and adhere to prescribed treatments.

However, the chronic inducible urticaria market faces several challenges. One of the most significant barriers is the lack of awareness about the condition, which often leads to delays in diagnosis and treatment. Given that CIndU is frequently misunderstood or misdiagnosed as other types of urticaria or allergic reactions, patients may suffer for years before receiving the appropriate treatment. Additionally, while antihistamines and corticosteroids provide relief, they often fall short for more severe cases, leaving a gap in effective treatment for a subset of patients. Furthermore, cost-related challenges, particularly for biologic therapies, limit access for certain patient groups, especially in developing regions or low-income populations.

Another challenge is the heterogeneity of the disease, with different forms of CIndU triggered by diverse physical stimuli. This variability complicates treatment protocols, as a single approach may not be effective for all patients. The lack of a one-size-fits-all treatment means that a personalized treatment approach will become increasingly important, but also more complex to implement.

The competitive landscape in the chronic inducible urticaria market is becoming more dynamic, with increased interest from pharmaceutical companies, biotechnology firms, and research institutions. Collaborations and strategic partnerships are accelerating the development of novel therapies and diagnostics. Companies are focusing on biologic treatments, monoclonal antibodies, and targeted immunotherapies, while also exploring new ways to deliver these treatments more efficiently.

Looking forward, the chronic inducible urticaria market is expected to continue its growth trajectory, fuelled by technological advancements, growing awareness, and the development of new therapeutic strategies. The integration of precision medicine, including molecular diagnostics, will play a central role in improving patient outcomes and refining treatment approaches. With a more personalized and proactive approach to care, CIndU patients are likely to experience better disease management and improved quality of life. The future of CIndU treatment holds promise with the ongoing clinical development of new biologics, digital health tools, and advanced therapies that aim to address the unmet needs of this patient population.

Companies Featured

• AstraZeneca
• Celldex Therapeutics, Inc.
• Johnson & Johnson Private Limited
• Merck & Co, Inc.
• Novartis AG
• Pfizer Inc.
• Regeneron Pharmaceuticals
• Sanofi
• Sun Pharmaceutical Industries Ltd.

Key Topics Covered:

1. Global Chronic Inducible Urticaria Market: Industry Analysis

1.1 Market Overview and Ecosystem

1.2 Epidemiological Analysis

1.3 Key Market Trends

1.3.1 Impact Analysis

1.4 Patent Analysis

1.4.1 Patent Filing Trend (by Country)

1.4.2 Patent Filing Trend (by Year)

1.5 Regulatory Landscape

1.6 Pipeline Analysis

1.7 Market Dynamics

1.7.1 Overview

1.7.2 Market Drivers

1.7.3 Market Restraints

1.7.4 Market Opportunities

2. Global Chronic Inducible Urticaria Market (by Drug Class), Value ($million), 2023-2035

2.1 Antihistamines

2.2 Corticosteroids

2.3 Monoclonal Antibodies

2.4 PDE4 Inhibitors

3. Global Chronic Inducible Urticaria Market (by Region), Value ($Million), 2023-2035

3.1 North America

3.1.1 Market Dynamics

3.1.2 Market Sizing and Forecast

3.1.3 North America Chronic Inducible Urticaria Market, by Country ($Million), 2023-2035

3.1.3.1 U.S.

3.1.3.2 Canada

3.2 Europe

3.2.1 Market Dynamics

3.2.2 Market Sizing and Forecast

3.2.3 Europe Chronic Inducible Urticaria Market, by Country ($Million), 2023-2035

3.2.3.1 U.K.

3.2.3.2 France

3.2.3.3 Germany

3.2.3.4 Italy

3.2.3.5 Spain

3.2.3.6 Rest-of-Europe

3.3 Asia-Pacific

3.3.1 Market Dynamics

3.3.2 Market Sizing and Forecast

3.3.3 Asia-Pacific Chronic Inducible Urticaria Market, by Country ($Million), 2023-2035

3.3.3.1 Japan

3.3.3.2 China

3.3.3.3 India

3.3.3.4 Australia

3.3.3.5 South Korea

3.3.3.6 Rest-of-Asia-Pacific

3.4 Rest-of-the-World

3.4.1 Market Dynamics

3.4.2 Market Sizing and Forecast Rest-of-the-World Chronic Inducible Urticaria Market, by Type ($Million), 2023-2035

3.4.3 Rest-of-the-World Chronic Inducible Urticaria Market, by Country ($Million), 2023-2035

3.4.3.1 Latin America

3.4.3.2 Middle East and Africa

4. Competitive Landscape and Company Profiles

4.1 Competitive Landscape

4.1.1 Mergers and Acquisitions

4.1.2 Partnership, Alliances and Business Expansion

4.1.3 New Offerings

4.1.4 Regulatory Activities

4.1.5 Funding Activities

4.2 Company Profiles

4.2.1 Overview

4.2.2 Top Products / Product Portfolio

4.2.3 Top Competitors

4.2.4 Target Customers/End-Users

4.2.5 Key Personnel

4.2.6 Analyst View

5. Research Methodology

For more information about this report visit https://www.researchandmarkets.com/r/d1o41u

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

For E.S.T Office Hours Call 1-917-300-0470

For U.S./ CAN Toll Free Call 1-800-526-8630

For GMT Office Hours Call +353-1-416-8900

Tardive Dyskinesia Market Research 2025 | Drug Class, Route of Administration, and Regional Analysis and Forecasts, 2023-2035 – ResearchAndMarkets.com




Tardive Dyskinesia Market Research 2025 | Drug Class, Route of Administration, and Regional Analysis and Forecasts, 2023-2035 – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Tardive Dyskinesia Market – A Global and Regional Analysis: Focus on Drug Class, Route of Administration, and Region – Analysis and Forecast, 2025-2035” report has been added to ResearchAndMarkets.com’s offering.

The global tardive dyskinesia (TD) treatment market is undergoing significant transformation, fuelled by the rising prevalence of psychiatric disorders, increased antipsychotic medication usage, growing clinical awareness of TD, and notable advancements in both pharmacological and digital therapeutic interventions. Tardive dyskinesia a persistent, often irreversible, movement disorder caused primarily by long-term use of dopamine receptor-blocking agents represents a major neuropsychiatric concern. The market has seen renewed interest as healthcare systems worldwide confront the complexities of treating movement disorders induced by medications intended to manage chronic psychiatric conditions.

TD’s increasing diagnosis rates are closely tied to heightened recognition among clinicians and public health authorities, particularly in high-burden countries with elevated prescriptions of antipsychotic and gastrointestinal medications. The disorder is most commonly seen in patients with schizophrenia, bipolar disorder, and major depressive disorder who are on long-term treatment regimens. Market growth is accelerated by the growing demand for therapies that can offer symptom mitigation, improved functional outcomes, and long-term safety, particularly in vulnerable populations such as the elderly, those with cognitive impairment, and individuals with multiple comorbidities.

A major catalyst for expansion is the widening availability and uptake of VMAT2 (vesicular monoamine transporter 2) inhibitors, especially valbenazine and deutetrabenazine, which have become first-line treatments due to their proven efficacy and tolerability profiles. These drugs have not only reshaped the therapeutic landscape, also opened doors for novel delivery mechanisms and formulations, including once-daily and sprinkle variants, designed for greater adherence and patient-centric care. In parallel, the growing exploration of non-dopaminergic targets, gene therapies, and neuromodulation techniques such as deep brain stimulation (DBS) and transcranial magnetic stimulation (TMS) are contributing to an expanding and increasingly diversified treatment pipeline.

Improved healthcare infrastructure, especially in emerging markets such as India, Brazil, and Southeast Asia, is allowing for broader diagnosis and treatment. These developments are complemented by rising public and professional awareness, alongside enhanced access to movement disorder specialists and psychiatric neurologists. Furthermore, the integration of digital health tools, such as AI-powered video diagnostics, symptom tracking apps, and remote teleconsultations, is modernizing how TD is monitored and managed across diverse healthcare settings.

Despite these growth drivers, the TD market faces several pressing challenges. These include the high cost of branded VMAT2 inhibitors, particularly in markets with limited or no insurance coverage, as well as uneven access to specialists in rural or low-income regions. Diagnostic variability remains a critical barrier, with many cases going undiagnosed or misclassified, especially when symptoms are subtle or overlap with other movement disorders. Additionally, concerns around long-term efficacy and side effect profiles, as well as the lack of validated biomarkers for early detection or treatment monitoring, hinder broader adoption of advanced therapeutics.

Looking forward, the global TD market is poised to maintain a strong growth trajectory, driven by rising clinical demand, innovative treatment modalities, and increased emphasis on patient-centered care. The integration of real-world evidence (RWE), precision medicine frameworks, and wearable technologies is expected to personalize therapy and improve clinical outcomes. In combination with expanded diagnostic infrastructure, enhanced clinician training, and policy-level prioritization of neurological disorders, the TD market holds the potential to significantly improve the lives of patients affected by this complex, medication-induced movement disorder.

Companies Featured

• AbbVie Inc.
• H. Lundbeck A/S
• Ipsen Pharma
• Luye Pharma Group
• Mitsubishi Tanabe Pharma Corporation
• Neurocrine Biosciences, Inc.
• SOM BIOTECH
• SteriMax Inc.
• Sun Pharmaceutical Industries Ltd.
• Teva Pharmaceutical Industries Ltd.

Key Topics Covered:

1. Global Tardive Dyskinesia Market: Industry Analysis

1.1 Market Overview and Ecosystem

1.2 Epidemiological Analysis

1.3 Key Market Trends

1.3.1 Impact Analysis

1.4 Regulatory Landscape

1.5 Pipeline Analysis

1.6 Market Dynamics

1.6.1 Overview

1.6.2 Market Drivers

1.6.3 Market Restraints

1.6.4 Market Opportunities

2. Global Tardive Dyskinesia Market (by Drug Class), Value ($million), 2023-2035

2.1 Atypical Antipsychotics

2.2 Tetrabenazine

2.3 Vesicular Monoamine Transporter (VMAT) Inhibitors

2.4 Others

3. Global Tardive Dyskinesia Market (by Route of Administration), Value ($million), 2023-2035

3.1 Injectable

3.2 Intravenous

3.3 Oral

4. Global Tardive Dyskinesia Market (by Region), Value ($Million), 2023-2035

4.1 North America

4.1.1 Market Dynamics

4.1.2 Market Sizing and Forecast

4.1.3 North America Tardive Dyskinesia Market, by Country ($Million), 2023-2035

4.1.3.1 U.S.

4.1.3.2 Canada

4.2 Europe

4.2.1 Market Dynamics

4.2.2 Market Sizing and Forecast

4.2.3 Europe Tardive Dyskinesia Market, by Country ($Million), 2023-2035

4.2.3.1 U.K.

4.2.3.2 France

4.2.3.3 Germany

4.2.3.4 Italy

4.2.3.5 Spain

4.2.3.6 Rest-of-Europe

4.3 Asia-Pacific

4.3.1 Market Dynamics

4.3.2 Market Sizing and Forecast

4.3.3 Asia-Pacific Tardive Dyskinesia Market, by Country ($Million), 2023-2035

4.3.3.1 Japan

4.3.3.2 China

4.3.3.3 India

4.3.3.4 Australia

4.3.3.5 South Korea

4.3.3.6 Rest-of-Asia-Pacific

4.4 Rest-of-the-World

4.4.1 Market Dynamics

4.4.2 Market Sizing and Forecast Rest-of-the-World Tardive Dyskinesia Market, by Type ($Million), 2023-2035

4.4.3 Rest-of-the-World Tardive Dyskinesia Market, by Country ($Million), 2023-2035

4.4.3.1 Latin America

4.4.3.2 Middle East and Africa

5. Competitive Landscape and Company Profiles

5.1 Competitive Landscape

5.1.1 Mergers and Acquisitions

5.1.2 Partnership, Alliances and Business Expansion

5.1.3 New Offerings

5.1.4 Regulatory Activities

5.1.5 Funding Activities

5.2 Company Profiles

5.2.1 Overview

5.2.2 Top Products / Product Portfolio

5.2.3 Top Competitors

5.2.4 Target Customers/End-Users

5.2.5 Key Personnel

5.2.6 Analyst View

For more information about this report visit https://www.researchandmarkets.com/r/p88rwx

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Liquid Biopsy Market Industry Report 2025-2035, with Profiles of ArcherDX, Biocartis, CellMax Life, DiaCarta, Exosome Diagnostics, GeneCast Biotechnology, NeoGenomics, OncoDNA, ScreenCell, and More – ResearchAndMarkets.com




Liquid Biopsy Market Industry Report 2025-2035, with Profiles of ArcherDX, Biocartis, CellMax Life, DiaCarta, Exosome Diagnostics, GeneCast Biotechnology, NeoGenomics, OncoDNA, ScreenCell, and More – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “Liquid Biopsy Market Industry Trends and Global Forecasts to 2035, by Application, Target Disease Indication, Type of Circulating Biomarker, Type of Sample, End-user, Type of Technique, and Key Geographical Regions” report has been added to ResearchAndMarkets.com’s offering.

The global liquid biopsy market is estimated to grow from USD 6.09 billion in the current year to USD 29.8 billion by 2035, at a CAGR of 15.53% during the forecast period to 2035.

In the recent past, liquid biopsy has emerged as a viable technique for cancer detection. The past decade has witnessed substantial innovation in liquid biopsy platforms that have resulted in increased regulatory approvals for minimally invasive blood based liquid biopsy tests. These tests use blood samples or other bodily fluids, such as plasma or urine to identify various circulating biomarkers, including cell free DNA, circulating tumor DNA and extracellular vesicles. Driven by lucrative investments, ongoing research and development efforts, and increased involvement of prominent players, the liquid biopsy market is anticipated to witness significant growth in the foreseen future.

Liquid Biopsy Market: Key Insights

The report delves into the current state of the liquid biopsy market and identifies potential growth opportunities within the industry. Some key findings from the report include:

• Over 180 liquid biopsy products are commercially available / being developed by various stakeholders; of these, ~45% use next generation sequencing techniques for the detection of various cancer indications.
• Close to 160 liquid biopsy products comprise of assay kits and laboratory developed tests; of these, ~55% have the capability to detect circulating tumor DNA biomarkers.
• Over the past five years, ~110 partnerships have been established by liquid biopsy product manufacturers; of these, ~20% were established for the development of liquid biopsy product / technology.
• Having realized the opportunity in this domain, several investors have invested around USD 7.3 billion (across over 125 funding instances) in the past nine years.
• Driven by the growing burden of cancer and increasing investments, the market for liquid biopsy and other non-invasive cancer diagnostics is poised to witness significant growth in the foreseeable future.
• The liquid biopsy market is projected to grow at a CAGR of ~15%, till 2035; the forecasted opportunity is likely to be distributed across various disease indications and biomarkers.
• The growing demand for different non-invasive techniques is anticipated to drive the growth of the liquid biopsy market; the market growth is likely to be the fastest in North America and Asia-Pacific.

Liquid Biopsy Market: Key Segments

Early Cancer Diagnosis Segment is Likely to Dominate the Liquid Biopsy Market During the Forecast Period

Based on the application, the market is segmented into early cancer diagnosis, patient monitoring and recurrence monitoring. At present, early cancer diagnosis holds the maximum share of the liquid biopsy market. This trend is likely to remain the same in the coming decade.

Breast Cancer Occupies the Largest Share of the Liquid Biopsy Market

Based on the target disease indication, the market is segmented into breast cancer, gastric cancer, ovarian cancer, leukemia, head and neck cancer, cervical cancer, lung cancer, brain cancer, colorectal cancer, bladder cancer, liver cancer, melanoma, nasopharyngeal cancer, oesophagus cancer, pancreatic cancer, prostate cancer, sarcoma and thyroid cancer. Currently, breast cancer captures the highest proportion of the liquid biopsy market. It is worth highlighting that the liquid biopsy market for leukemia is likely to grow at a relatively higher CAGR.

Circulating tumor DNA is the Preferred Biomarkers of the Liquid Biopsy Market

Based on the type of circulating biomarker, the market is segmented into cell free DNA, cell free RNA, circulating tumor DNA, exosomes and others circulating biomarkers. At present, circulating tumor DNA holds the maximum share of the liquid biopsy market. It is worth highlighting that the liquid biopsy market for exosomes is likely to grow at a relatively higher CAGR.

Blood Segment is Likely to Dominate the Liquid Biopsy Market During the Forecast Period

Based on the type of sample, the market is segmented into blood and other samples. At present, blood samples hold the maximum share of the liquid biopsy market. This trend is likely to remain the same in the coming decade.

Currently, Hospitals / Laboratories Occupies the Largest Share of the Liquid Biopsy Market

Based on the end-users, the market is segmented into hospitals / laboratories, research institutes and other end-users. It is worth highlighting that, currently, hospitals / laboratories hold a larger share of the liquid biopsy market. This trend is unlikely to change in the near future.

Next-Generation Sequencing Segment is the Fastest Growing Segment of the Liquid Biopsy Market During the Forecast Period

Based on the type of technique, the market is segmented into polymerase chain reaction and next generation sequencing. At present, polymerase chain reaction holds the maximum share of the liquid biopsy market. It is worth highlighting that the liquid biopsy market for next generation sequencing is likely to grow at a relatively higher CAGR.

North America Accounts for the Largest Share of the Market

Based on key geographical regions, the market is segmented into North America, Europe and Asia-Pacific. The majority share is expected to be captured by players based in North America. It is worth highlighting that the majority share is expected to be captured by players based in North America.

Key Players in the Liquid Biopsy Market, Profiled in the Report Include:

• Amoy Diagnostics
• ArcherDX
• Biocartis
• Cell Search
• CellMax Life
• Datar Cancer Genetics
• DiaCarta
• EONE-DIAGNOMICS
• Exosome Diagnostics
• GeneCast Biotechnology
• Integrated DNA Technologies
• Lucence
• MDNA Life Sciences
• Miltenyi Biotec
• NeoGenomics
• ONCODE Scientific
• OncoDNA
• QIAGEN
• PANAGENE
• Personal Genome Diagnostics
• Predicin
• ScreenCell
• Tecan
• Thermo Fisher Scientific

Liquid Biopsy Market: Research Coverage

• Market Sizing and Opportunity Analysis: The report features an in-depth analysis of the liquid biopsy market, focusing on key market segments, including application, target disease indication, type of circulating biomarker, type of sample, end-user, stage of development, type of product, type of technique, application area and key geographical regions.
• Market Landscape: A comprehensive evaluation of liquid biopsy products, considering various parameters, such as stage of development, type of product, type of sample, type of technique, type of circulating biomarker, target disease indication, application and application area. In addition, it provides a list of players engaged in manufacturing liquid biopsy products, along with the information on their year of establishment, company size, location of headquarters, most active players.
• Non-Invasive Cancer Screening and Diagnosis: An overview on the need for non invasive cancer diagnostics and their importance; it also features different imaging techniques, screening assays and advanced approaches used for diagnosis of cancer along with their advantages and disadvantages.
• Company Profiles: In-depth profiles of key industry players manufacturing liquid biopsy products, focusing on company overviews, financial information, product portfolio, recent developments and an informed future outlook.
• Partnerships and Collaborations: An analysis of partnerships established in this sector, since 2020, based on several parameters, such as year of partnership, type of partnership, type of partner, type of circulating biomarker, target disease indication, most active players. This section also highlights the regional distribution of partnership activity in this market.
• Funding and Investment Analysis: A detailed evaluation of the investments made in the liquid biopsy domain, encompassing seed financing, venture capital, capital raised from IPOs, secondary offerings, grants / awards, other equity and debt financing, based on several parameters, such as year of investment, amount invested, type of funding, type of circulating biomarker, target disease indication, application area, geography, most active players and leading investors.
• Product Competitiveness Analysis: A comprehensive competitive analysis of liquid biopsy products, examining factors, such as supplier strength, product competitiveness and company size.
• Big Pharma Analysis: A comprehensive examination of various initiatives focused on liquid biopsy products undertaken by major pharmaceutical companies. This analysis covers various relevant parameters, such as number of initiatives, type of initiative, stage of development, type of product, type of circulating biomarker, target disease indication, application and application area.
• Key Acquisition Targets: A detailed analysis of the key acquisition targets, taking into consideration the historical trend of the acquisition activity of the players that have acquired other firms. It offers a means for other industry stakeholders to identify potential acquisition targets.
• Other Non-Invasive Cancer Diagnostics: A detailed overview of the various non invasive diagnostic tests other than liquid biopsies, being manufactured by various companies for cancer screening and early detection.
• Market Impact Analysis: The report analyzes various factors such as drivers, restraints, opportunities, and challenges affecting the market growth.

Key Questions Answered in this Report

• How many companies are currently engaged in this market?
• Which are the leading companies in this market?
• What kind of partnership models are commonly adopted by industry stakeholders?
• What factors are likely to influence the evolution of this market?
• What is the current and future market size?
• What is the CAGR of this market?
• How is the current and future market opportunity likely to be distributed across key market segments?

Reasons to Buy this Report

• The report provides a comprehensive market analysis, offering detailed revenue projections of the overall market and its specific sub-segments. This information is valuable to both established market leaders and emerging entrants.
• Stakeholders can leverage the report to gain a deeper understanding of the competitive dynamics within the market. By analyzing the competitive landscape, businesses can make informed decisions to optimize their market positioning and develop effective go-to-market strategies.
• The report offers stakeholders a comprehensive overview of the market, including key drivers, barriers, opportunities, and challenges. This information empowers stakeholders to stay abreast of market trends and make data-driven decisions to capitalize on growth prospects.

Additional Benefits

• Complimentary PPT Insights Packs
• Complimentary Excel Data Packs for all Analytical Modules in the Report
• 10% Free Content Customization
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CapVest to Acquire Majority Stake in STADA from Bain Capital and Cinven




CapVest to Acquire Majority Stake in STADA from Bain Capital and Cinven

• CapVest has signed a definitive agreement to acquire the majority stake in STADA from Bain Capital and Cinven
• Bain Capital and Cinven will each retain a minority stake in STADA
• STADA CEO Peter Goldschmidt: “CapVest’s focus on working closely with management in transforming the size and scale of its portfolio companies through significant capital investment, a combination of organic and acquisition led growth, operational excellence, and long-term value creation reflects the same principles that have underpinned STADA’s success to date.”

BAD VILBEL, Germany & LONDON–(BUSINESS WIRE)–CapVest Partners LLP (“CapVest”), a leading global investment firm, has today signed a definitive agreement with Bain Capital and Cinven to acquire the majority stake in STADA Arzneimittel AG “(STADA” or “the Company”), a leading healthcare and pharmaceuticals company specializing in Consumer Healthcare, Generics and Specialty pharmaceuticals.

Headquartered in Bad Vilbel, Germany, STADA is a leading European pharmaceutical company focused on three strategic pillars: Consumer Healthcare, Generics and Specialty Pharmaceuticals. The company was acquired by Bain Capital and Cinven in 2017. Working with management, the two international investment firms helped transform the company from a traditional German generics business into a leading, diversified global healthcare platform with a strategic focus on Consumer Healthcare, Generics and Specialty Pharmaceuticals. During this period, STADA grew into a multifaceted, resilient company, generating revenues in excess of €4 billion, delivering a compound annual net sales growth rate of 9%, and more than doubling EBITDA since 2017. The firm currently employs approximately 11,600 people worldwide.

A leading international private equity investor that partners with ambitious companies supplying essential goods and services, CapVest brings deep sector expertise and a strong track record of over 20 years of investing in the healthcare industry, making the firm ideally positioned to support STADA in its next phase of growth. Following completion of the transaction, Bain Capital and Cinven will each retain a minority stake in STADA, underscoring their confidence in the company’s future growth trajectory and commitment to supporting the management team.

The terms of the transaction have not been disclosed and are subject to regulatory approvals and other customary closing conditions. Closing of the transaction is expected in early in 2026.

Matthew Fargie of CapVest said: “We have admired STADA for several years, including its deep heritage, leading product portfolio and a culture underpinned by caring for people’s health. Peter and the entire STADA team have a best-in-class track record of performance. STADA is a unique strategic platform through which we will leverage our significant healthcare and consumer expertise to accelerate the development of the company in Germany and internationally. We intend to deploy significant new capital towards this objective. We look forward to working with Peter Goldschmidt and his team as custodians of this great company in its next phase of development.”

Peter Goldschmidt, Chief Executive Officer of STADA, commented: “CapVest’s focus on working closely with management in transforming the size and scale of its portfolio companies through significant capital investment, a combination of organic and acquisition led growth, operational excellence, and long-term value creation reflects the same principles that have underpinned STADA’s success to date. With their deep knowledge in the healthcare and pharmaceuticals markets, CapVest is an ideal next partner to work with us to capitalise on the many opportunities emerging in our sector and realise our ambitious plans for the company. Cinven and Bain Capital have been excellent partners on our journey to become a global leader in Consumer Healthcare, Generics and Specialty Pharmaceuticals. Their support and conviction in our vision enabled us to accelerate growth, innovate, and expand internationally.”

Lead financial advisors to CapVest were Canson Capital Partners and Centerview Partners.

About STADA Arzneimittel AG

STADA Arzneimittel AG is headquartered in Bad Vilbel, Germany. The company focuses on a three-pillar strategy consisting of consumer healthcare products, generics and specialty pharma. Worldwide, STADA Arzneimittel AG sells its products in over 100 countries. In financial year 2024, STADA achieved group sales of € 4,059 million and adjusted constant-currency earnings before interest, taxes, depreciation and amortization (adj. cc EBITDA) of € 886 million. As of 31 December 2024, STADA employed 11,649 people worldwide.

About CapVest

CapVest is a leading international private equity investor that partners with ambitious companies supplying essential goods and services to transform their businesses. As an active and patient investor, CapVest has established a strong record of success in delivering attractive returns by working closely with management in transforming the size and scale of its portfolio companies through a combination of organic and acquisition led growth.

CapVest seeks to invest in highly resilient industries where the demand driver for the product or service is non-discretionary. Its core sectors include healthcare, consumer staples and essential services.

Contacts

CapVest media contact
Ben Valdimarsson – Reputation Inc

Phone: +44 (0) 7889 805 930

E-mail: bvaldimarsson@reputation-inc.com

STADA information for journalists
STADA Arzneimittel AG – Media Relations

Stadastrasse 2-18, 61118 Bad Vilbel – Germany

Phone: +49 (0) 6101 603-165

E-Mail: press@stada.de
Or visit us via our website at https://www.stada.com/media/newsroom
Follow @stadaGroup on LinkedIn

STADA information for capital market participants
STADA Arzneimittel AG – Investor & Creditor Relations

Stadastrasse 2-18, 61118 Bad Vilbel – Germany

Phone: +49 (0) 6101 603-4689

Fax: +49 (0) 6101 603-215

E-mail: ir@stada.de
Or visit us via our website at https://www.stada.com/investor-relations/

New Data From Landmark HELIOS-B Phase 3 Study Presented at ESC Congress 2025 Demonstrate Vutrisiran’s Long-Term Cardiovascular Benefit in ATTR-CM




New Data From Landmark HELIOS-B Phase 3 Study Presented at ESC Congress 2025 Demonstrate Vutrisiran’s Long-Term Cardiovascular Benefit in ATTR-CM

− Sustained Benefit of Vutrisiran Across Mortality, Cardiovascular Events, Quality of Life and Cardiac Biomarkers Observed Through Up to 48 Months, Including 12 Months from the Ongoing Open-Label Extension, Reinforce Potential as First-Line Treatment for ATTR-CM –

− Vutrisiran, which Delivers Rapid Knockdown of Transthyretin, Reduced the Risk of Composite of All-Cause Mortality or First Cardiovascular Event by 37% in the Overall Population and 42% in the Monotherapy Group During the Same Period –

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced results of new analyses from the HELIOS-B Phase 3 study of AMVUTTRA^® (vutrisiran), an RNAi therapeutic approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults. Data from the 12-month follow-up of the ongoing open-label extension (OLE) period of HELIOS-B were presented during an oral session at the European Society of Cardiology (ESC) Congress 2025 held in Madrid, Spain. These data reflect outcomes of treatment through up to 48 months, including the initial double-blind period of 33-36 months, and highlight the ongoing clinical benefit of vutrisiran, which causes rapid knockdown of the disease-causing transthyretin (TTR) protein, including a 37% risk reduction in the composite endpoint of all-cause mortality (ACM) or first cardiovascular (CV) event in the overall population (p<0.001) and a 42% risk reduction in the monotherapy group (p<0.001), reinforcing vutrisiran’s potential as a first-line treatment for patients with ATTR-CM. Vutrisiran also reduced the risk of ACM alone by 37% in the overall population (p<0.01) and 39% in the monotherapy group (p<0.01) during this same period.

The findings from the ongoing HELIOS-B study show that the clinical benefits seen during the double-blind period of 33-36 months across key clinical measures of disease progression, including quality of life (QoL) as measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) and cardiac biomarkers, were maintained with continued vutrisiran treatment during the 12-month OLE period. The long-term safety and tolerability profile during the OLE period was consistent with the established profile of vutrisiran. Additionally, a post hoc analysis of the HELIOS-B trial presented during a poster session highlighted reductions in days lost to death and/or hospitalization (DLDH) and improvements in functional and QoL outcomes compared to placebo. Vutrisiran was associated with a reduction in mean DLDH of more than one month versus placebo over a three-year period, with a greater effect observed when accounting for impaired function and QoL.

“The HELIOS-B study continues to deliver robust evidence supporting the clinically differentiated profile of vutrisiran,” said John Vest, M.D., Senior Vice President, TTR Global Clinical Lead, Alnylam. “These longer-term data demonstrate that, through up to 48 months, vutrisiran treatment conferred continued clinical benefit across key endpoints, including survival, cardiovascular events, and quality of life. These results reinforce the potential for vutrisiran, which provides rapid and sustained knockdown of TTR, to modify the course of disease over the long-term and to be a first-line therapy for ATTR-CM.”

The landmark HELIOS-B Phase 3 clinical trial evaluated vutrisiran for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM) in a population representative of today’s patients. Of the 654 patients who received treatment with vutrisiran in HELIOS-B, 466 entered the OLE period. At the data cutoff (April 9, 2025), vital status was ascertained for >99% of patients. Key findings from 12-month follow-up include:

Safety data for vutrisiran treatment through one year of the OLE period were consistent with that reported for the double-blind period. The rate of adverse events (AEs), including cardiac events, did not increase with longer treatment. Event rates for serious adverse events (SAEs) and severe AEs in the placebo/vutrisiran group were consistent with more advanced disease following placebo treatment during the double-blind period. No new safety concerns were identified.

“The open-label extension of HELIOS-B adds important long-term evidence to inform the management of ATTR-CM, a progressive and life-threatening condition that remains a significant clinical challenge,” said Dr. Pablo Garcia-Pavia, Head of the Inherited Cardiac Diseases and Heart Failure Unit at the Department of Cardiology of Hospital Universitario Puerta de Hierro and group leader at the Spanish Cardiovascular Research Network (CIBERCV), in Madrid, Spain. “The irreversible nature of disease progression, taken together with the sustained clinical benefit seen in patients treated with vutrisiran through 48 months, collectively underscore the importance of early treatment initiation and further support the role of vutrisiran as a first-line treatment for ATTR-CM. The results were observed in a group of patients reflective of the contemporary ATTR-CM population, including those receiving substantial concurrent use of available standard-of-care therapies such as tafamidis and SGLT2 inhibitors.”

A post hoc analysis from HELIOS-B further characterized the practical impact of vutrisiran treatment on all-cause mortality and recurrent CV events by assessing days lost to death and/or hospitalization (DLDH). This metric captures both the occurrence and burden of events over time and evaluated vutrisiran versus placebo on DLDH and days lost to death and/or cardiovascular hospitalization (DLDCVH) in both the overall and monotherapy populations. In the overall population, vutrisiran was associated with 32.2 fewer DLDH over 3 years compared to placebo. A similar benefit was seen for DLDCVH. Vutrisiran treatment was associated with a higher likelihood of patients experiencing no DLDH or DLDCVH and showed an even greater impact when factoring in days of impaired well-being. Results in the monotherapy group were consistent with the overall population. For more details, please visit Capella.

Data from the HELIOS-B study supported the recent approvals of AMVUTTRA for the treatment of the cardiomyopathy of wild-type or hereditary ATTR-CM in adults in the United States (US), Brazil, European Union (EU), Japan, United Arab Emirates (UAE) and United Kingdom (UK). Collectively, AMVUTTRA has more than 8,000 patient-years of experience worldwide and is the first RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR) in adults.

For additional information on Alnylam’s presentations at the ESC 2025 Congress, please visit Capella.

AMVUTTRA^® (vutrisiran) INDICATIONS AND IMPORTANT SAFETY INFORMATION

Indications

In the EU, AMVUTTRA^® (vutrisiran) is indicated for the treatment of:

• hereditary transthyretin amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy (hATTR-PN).
• wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Availability across the EU is subject to local reimbursement timelines.

Important Safety Information

Reduced Serum Vitamin A Levels and Recommended Supplementation

Vutrisiran treatment leads to a decrease in serum vitamin A levels. Supplementation of approximately, but not exceeding, 2500 IU to 3000 IU vitamin A per day is advised for patients taking vutrisiran. Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g., night blindness).

Adverse Reactions

Commonly reported adverse reactions with vutrisiran were injection site reactions and increase in blood alkaline phosphatase and alanine transaminase.

For additional information about vutrisiran, please see the full Summary of Product Characteristics.

About AMVUTTRA^® (vutrisiran)

AMVUTTRA^® (vutrisiran) is an RNAi therapeutic that delivers rapid knockdown of transthyretin (TTR), addressing the underlying cause of transthyretin (ATTR) amyloidosis. It is marketed in more than 15 countries for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults and it is also approved for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults in the US, Europe, Brazil, Japan, UAE and UK. Administered quarterly via subcutaneous injection, AMVUTTRA is the first and only RNAi therapeutic approved for the treatment of both the cardiomyopathy manifestations of ATTR amyloidosis and the polyneuropathy manifestations of hereditary transthyretin-mediated amyloidosis (hATTR).

About ATTR

Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly progressive, debilitating and fatal disease caused by misfolded transthyretin (TTR) proteins, which accumulate as amyloid deposits in various parts of the body, including the nerves, heart and gastrointestinal tract. Patients may present with polyneuropathy, cardiomyopathy, or both manifestations of disease. There are two different forms of ATTR – hereditary ATTR (hATTR), which is caused by a TTR gene variant and affects approximately 50,000 people worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR gene variant and impacts an estimated 200,000 – 300,000 people worldwide.^1-4

About RNAi

RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today.⁵ Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine.⁶ By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made.⁵ This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

About Alnylam Pharmaceuticals

Alnylam (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA.

Alnylam Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam’s expectations, beliefs, goals, plans or prospects including, without limitation, Alnylam’s expectations regarding the safety and efficacy of vutrisiran for the treatment of ATTR-CM; vutrisiran’s sustained and long-term benefits in ATTR-CM; vutrisiran’s potential as a first-line treatment for patients with ATTR-CM; the potential for vutrisiran to have continued clinical benefits and to modify the course of disease in ATTR-CM over the long term; and Alnylam’s ability to execute on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to Alnylam’s ability to successfully execute on its “Alnylam P5x25” goals; Alnylam’s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam’s product candidates; actions or advice of regulatory agencies and Alnylam’s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; successfully launching, marketing and selling Alnylam’s approved products globally; delays, interruptions or failures in the manufacture and supply of Alnylam’s product candidates or its marketed products; obtaining, maintaining and protecting intellectual property; Alnylam’s ability to manage its growth and operating expenses through disciplined investment in operations and its ability to achieve a self-sustainable financial profile in the future; Alnylam’s ability to maintain strategic business collaborations; Alnylam’s dependence on third parties for the development and commercialization of certain products; the outcome of litigation; the potential risk of future government investigations; and unexpected expenditures; as well as those risks more fully discussed in the “Risk Factors” filed with Alnylam’s 2024 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam’s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam’s views only as of today and should not be relied upon as representing Alnylam’s views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

¹ Hawkins PN, Ando Y, Dispenzeri A, et al. Ann Med. 2015;47(8):625-638.

² Gertz MA. Am J Manag Care. 2017;23(7):S107-S112.

³ Conceicao I, Gonzalez-Duarte A, Obici L, et al. J Peripher Nerv Syst. 2016;21:5-9.

⁴ Ando Y, Coelho T, Berk JL, et al. Orphanet J Rare Dis. 2013;8:31.

⁵ Elbashir SM, Harborth J, Lendeckel W, et al. Nature. 2001;411(6836):494-498.

⁶ Zamore P. Cell. 2006;127(5):1083-1086.

Contacts

Alnylam Pharmaceuticals, Inc.

Christine Akinc

(Investors and Media)

+1-617-682-4340

Josh Brodsky

(Investors)

+1-617-551-8276

Daiichi Sankyo Announces the Initiation of the Development of Oral Triple Combination Lipid-Lowering Tablets to Support the Management of LDL-C




Daiichi Sankyo Announces the Initiation of the Development of Oral Triple Combination Lipid-Lowering Tablets to Support the Management of LDL-C

• Initiation of the development of new oral triple combination tablets, aimed at improving adherence and outcomes in low-density lipoprotein cholesterol (LDL-C) management.
• Recent findings from MILOS and SANTORINI registries presented at ESC Congress 2025, highlight ongoing challenges in accurately assessing cardiovascular (CV) risk and helping patients reach LDL-C targets.^1-5 Furthermore, data from a four-country cohort of the MILOS study support the real-world effectiveness and safety of bempedoic acid in both sexes.⁶
• Simulation using SANTORINI data reveals the potential of improved LDL-C goal attainment with tailored oral combination therapy of statins, ezetimibe and bempedoic acid, in line with the latest 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias.^7,8
• Daiichi Sankyo has a long-standing commitment to easing treatment of cardiovascular disease (CVD) and exploring new treatment options that improve CVD outcomes and treatment adherence.

MUNICH, Germany–(BUSINESS WIRE)–Daiichi Sankyo Europe is pleased to announce the initiation of the development of new oral triple combination tablets in Europe of bempedoic acid, ezetimibe, and different doses of a statin (atorvastatin or rosuvastatin), with the potential to lower low-density lipoprotein cholesterol (LDL-C) levels.⁹ It is well known that combination therapies reduce the pill burden for patients, potentially enhancing treatment adherence and facilitating treatment with a goal of improving cardiovascular outcomes.^9,10

“As bempedoic acid and ezetimibe are already approved as a single-dose therapy, the development of an oral triple combination tablet with different doses of a statin, can make it easier for physicians to tailor treatment to the individual needs of each patient,” says Dr. Stefan Seyfried, Vice President and Head Medical Affairs Specialty Medicines, Daiichi Sankyo Europe. “This approach exemplifies our dedication to our motto: ‘we care for every heartbeat’.”

“The management of dyslipidaemias is undergoing the same evolution we previously witnessed in hypertension management. First, we learned to combine medication to achieve higher efficacy with fewer side effects, then we ascertained that replacing multiple separate pills with fixed-dose combination options increased adherence and improved outcomes,” says Prof. Yvonne Winhofer-Stöckl, Medical University of Vienna, Austria.

The announcement of the development of the oral triple combination coincides with the presentation at European Society of Cardiology (ESC) Congress 2025 of new clinical data from the MILOS and SANTORINI registries, which continue to emphasise ongoing challenges in LDL-C management.^1,2

• Data from four-country cohort of the MILOS study reveal that patients’ real cardiovascular (CV) risk is often underestimated in clinical practice, with up to 50% of high-risk patients and up to 70% of very high-risk patients assigned a lower CV risk and highlight treatment disparities between genders, with women less likely to receive intensive lipid-lowering therapy (LLT) than men.^3,6 Furthermore, the addition of bempedoic acid over eight weeks, with or without other LLTs, was associated with a reduction in LDL-C levels in both sexes with no new safety signals, supporting the real-world effectiveness and safety of bempedoic acid in both sexes.⁶
• SANTORINI data also expose gaps in LLT intensification, with only 20% of high-risk and very-high-risk patients with elevated LDL-C receiving treatment intensification, and older patients less likely to be treated intensively than younger patients.^4,5

In addition, real-world prescribing data show that nearly ¾ of high or very high CV risk patients in Germany are not prescribed any LLT.¹¹

The SANTORINI cohort of high-risk and very high-risk patients was also used in a simulation suggesting that 6 in 10 patients could achieve LDL-C goals and meaningfully reduce their long-term CV risk with an optimised triple oral LLT pathway (bempedoic acid, ezetimibe and a statin), in line with results presented at European Atherosclerosis Society (EAS) Congress 2025 showing that intensification with combination therapies resulted in better control of LDL-C levels than statin up titration.^7,12 The lipid-lowering potential of the oral triple combination is recognised in the latest 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias.⁸

CVD continues to pose a significant global challenge, and is the leading cause of death in Europe.¹³ Dyslipidaemia, or elevated LDL-C levels, is one of the most prevalent modifiable risk factors contributing to CV events, and its management remains a critical need in CV risk reduction.^14-16 Lipid-lowering therapies have been shown to reduce the risk of major adverse cardiovascular events (MACE).^14,15 However, despite the protective CV effects of these treatments, their use is often suboptimal due to inconsistent clinical practices, insufficient adherence (partly due to polypharmacy), treatment inertia and underutilisation of combination therapies, resulting in a substantial proportion of patients not reaching their recommended levels of LDL-C and remaining at high CV risk.^10,15,16

The Specialty Medicines Unit of Daiichi Sankyo intends to protect people from CVD and support those affected, helping them enjoy every precious moment of life. Daiichi Sankyo Europe is dedicated to identifying and addressing challenges in cardiovascular disease. We use a patient-centric approach to develop new solutions that have the potential to improve long-term health outcomes.

About SANTORINI

The SANTORINI (NCT04271280) study is a multinational, prospective, observational study that enrolled 9,602 patients with high and very high cardiovascular (CV) risk from over 623 sites in 14 countries across Europe. Patients were recruited between March 2020 and February 2021.^2,16 The primary objective was to document, in the real-world setting, the effectiveness of current low-density lipoprotein cholesterol (LDL-C) management approaches in high- and very high-cardiovascular risk patients requiring lipid-lowering therapies (LLT) over a 1-year period.¹⁷ The study included both previously diagnosed and treated patients and those newly diagnosed and requiring treatment.¹⁶ Complete baseline data was included for 9,044 patients (mean age: 65.3 ± 10.9 years; 72.6% male).¹⁶ Physicians used 2019 ESC/EAS guidelines as a basis for CV risk classification in 52% of patients; 29.2% of patients were classified as high risk and 70.8% as very high risk.¹⁶ Central reassessment with the same guidelines classified 6.5% as high risk and 91% as very high-risk.¹⁶ Overall, 21.8% of patients had no documented LLTs, 54.2% were receiving monotherapy and 24% combination LLT.¹⁶ Median LDL-C was 2.1 mmol/L with 20.1% of patients achieving risk based LDL-C goals as per the 2019 ESC/EAS guidelines.¹⁶

About MILOS

MILOS (NCT04579367) is an ongoing, multinational, European observational study in adult patients diagnosed with primary hypercholesterolaemia or mixed dyslipidaemia.¹ The aim is to evaluate the real-world use of bempedoic acid and bempedoic plus ezetimibe fixed-dose combination. Beyond Germany, MILOS has sites in Austria, Belgium, Italy, the Netherlands, Spain, Switzerland and the UK.¹

About Bempedoic Acid and its Fixed-Dose Combination with Ezetimibe

Bempedoic acid (NILEMDO®) is a first-in-class, oral, once-daily treatment that lowers low-density lipoprotein cholesterol (LDL-C) by inhibiting ATP citrate lyase (ACL), upstream of the statin target in the cholesterol synthesis pathway.^18,19 It is not activated in skeletal muscle and can be used alone or in combination with statins or other lipid-lowering therapies, particularly in patients who are statin-intolerant or not achieving LDL-C goals. The fixed-dose combination of bempedoic acid and ezetimibe (NUSTENDI®) combines two different ways of reducing cholesterol in a once-daily tablet.²⁰

For full prescribing information, including indications and usage, refer to the respective Summary of Product Characteristics (SmPC).^18,20 Daiichi Sankyo Europe holds exclusive commercialisation rights for NILEMDO® and NUSTENDI® in the EEA, UK, Turkey, and Switzerland under license from Esperion.

About Atorvastatin and Rosuvastatin

These medicines belong to a group of medication that lower low-density lipoprotein cholesterol to block HMG-CoA reductase enzyme in the liver and thus the production of cholesterol. They act in the same pathway as bempedoic acid, but at a different point.^21,22

About Daiichi Sankyo

Daiichi Sankyo is an innovative global healthcare company contributing to the sustainable development of society that discovers, develops, and delivers new standards of care to enrich the quality of life around the world. With more than 120 years of experience, Daiichi Sankyo leverages its world-class science and technology to create new modalities and innovative medicines for people with cancer, cardiovascular, and other diseases facing significant therapeutic needs. For more information, please visit www.daiichisankyo.com.

References

1. Treatment with bempedoic acid and/​or its fixed-dose combination with ezetimibe in primary hypercholesterolemia or mixed dyslipidemia (MILOS). Available at: https://clinicaltrials.gov/study/NCT04579367. Last accessed July 2025.

2. Treatment of high and very high risk dyslipidemic patients for the prevention of cardiovascular events (SANTORINI). Available at: https://clinicaltrials.gov/study/NCT04271280. Last accessed July 2025.

3. Parhofer KG, et al. Underestimation of cardiovascular risk in four European countries: results from the prospective, noninterventional MILOS. Presented at European Society of Cardiology (ESC) Congress 2025.

4. Aguiar C, et al. Predictors of lipid-lowering therapy intensification over 1 year of prospective follow-up in Europe: insights from the SANTORINI study. Presented at European Society of Cardiology (ESC) Congress 2025.

5. Connolly DL, et al. Age-stratified analysis of real-world lipid-lowering therapy use and LDL-C goal attainment at 1-year follow-up: insights from the European SANTORINI study. Presented at European Society of Cardiology (ESC) Congress 2025.

6. Gouni-Berthold I, et al. Undertreatment of women with lipid-lowering therapies in four European countries: results from the prospective, non-interventional MILOS Study. Presented at European Society of Cardiology (ESC) Congress 2025.

7. Katzmann JL, et al. Triple oral lipid-lowering treatment pathway and associated outcomes in high- and very high-CV risk patients: a simulation using the SANTORINI study cohort. Presented at European Society of Cardiology (ESC) Congress 2025.

8. Mach F, et al. 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias: Developed by the task force for the management of dyslipidaemias of the European Society of Cardiology (ESC) and the European Atherosclerosis Society (EAS). Eur Heart J. 2025;ehaf190. doi:10.1093/eurheartj/ehaf190

9. Rubino J, et al. Combination of bempedoic acid, ezetimibe, and atorvastatin in patients with hypercholesterolemia: A randomized clinical trial. Atherosclerosis. 2021;320:122–8.

10. Weisser B, et al. Effect of a single pill concept on clinical and pharmacoeconomic outcomes in cardiovascular diseases. Eur Heart J Cardiovasc Pharmacother. 2025;10(8):686–93.

11. Weingaertner O, et al. Real-world prescribing patterns for lipid-lowering therapy in high cardiovascular risk patients in Germany: a cross-sectional analysis. Presented at European Society of Cardiology (ESC) Congress 2025.

12. Connolly DL, et al. Initiating combination therapy facilitates better LDL-C goal attainment than statin up-titration over 1 year: an analysis from the SANTORINI study. Presented at European Atherosclerosis Society (EAS) Congress 2025.

13. World Health Organisation. Cardiovascular diseases. Available at: https://www.who.int/europe/news-room/fact-sheets/item/cardiovascular-diseases. Last accessed July 2025.

14. Nissen SE, et al. Bempedoic acid and cardiovascular outcomes in statin-intolerant patients. N Engl J Med. 2023. 13;388(15):1353-1364.

15. Mach F, et al. 2019 ESC/EAS Guidelines for the Management of dyslipidaemias: Lipid Modification to Reduce Cardiovascular Risk. European Heart Journal. 2019 Aug 31;41(1).

16. Ray KK, et al. Treatment gaps in the implementation of LDL cholesterol control among high- and very high-risk patients in Europe between 2020 and 2021: the multinational observational SANTORINI study. The Lancet Reg Health Eur. 2023 Jun 1;29:100624.

17. Gouni-Berthold I, et al. Real-world effectiveness and safety of bempedoic acid in Europe: final 2-year results from the MILOS German cohort. Presented at DGK Hertztage 2024.

18. European Medicines Agency. Nilemdo – summary of product characteristics (SmPC). Available at: https://www.ema.europa.eu/en/documents/product-information/nilemdo-epar-product-information_en.pdf. Last accessed July 2025.

19. Pinkosky SL, et al. Liver-specific ATP-citrate lyase inhibition by bempedoic acid decreases LDL-C and attenuates atherosclerosis. Nat Commun. 2016. 7: 13457.

20. European Medicines Agency. Nustendi – summary of product characteristics (SmPC). Available at: https://www.ema.europa.eu/en/documents/product-information/nustendi-epar-product-information_en.pdf. Last accessed July 2025.

21. European Medicines Agency. Atorvastatin – summary of product characteristics (SmPC). Available at: https://www.ema.europa.eu/en/documents/referral/sortis-article-6-12-referral-annex-i-ii-iii_en.pdf. Last accessed July 2025.

22. European Medicines Agency. Rosuvastatin –summary of product characteristics (SmPC). Available at: https://www.ema.europa.eu/en/documents/referral/crestor-5-mg-article-29-referral-annex-i-ii-iii_en.pdf. Last accessed July 2025.

Contacts

Media Contact
Shreya Pandey

Daiichi Sankyo Europe GmbH

Senior Public Relations Manager

+49 151 5519 4223

Daiichi Sankyo Announces the Development of a Fixed-Triple Combination Lipid-Lowering Tablet to Address Persistent Gaps in the Management of Elevated LDL-C




Daiichi Sankyo Announces the Development of a Fixed-Triple Combination Lipid-Lowering Tablet to Address Persistent Gaps in the Management of Elevated LDL-C

MUNICH, Germany–(BUSINESS WIRE)–Daiichi Sankyo Europe is pleased to announce the development of a fixed-triple oral combination lipid-lowering tablet of bempedoic acid, ezetimibe, and different doses of a statin, with the potential to lower low-density lipoprotein cholesterol (LDL-C) levels.^{i ii}

“As bempedoic acid and ezetimibe are already approved as a single-dose therapy, the development of an oral triple combination with different doses of a statin, will make it easier for physicians to tailor treatment to the individual needs of each patient”, says Dr. Stefan Seyfried, Vice President, and Head Medical Affairs Specialty Medicines, Daiichi Sankyo Europe. “This approach exemplifies our dedication to our motto: ‘we care for every heartbeat’”.

Daiichi Sankyo is prioritising the development programme further to ongoing data readouts from their funded MILOS and SANTORINI registries which continue to emphasise the persistent gaps in LDL-C management and the health inequalities this exacerbates.

Professor of Public Health and Honorary Cardiologist at Imperial College London, Professor Kausik Ray added, “Heart disease remains the leading cause of death in Europe despite the significant strides forward we have taken in recent years; improvements in part enabled by more effective cholesterol management. However, data from the MILOS and SANTORINI registries exposes the ongoing inconsistencies in LDL-C management, in particular the disparities with worse LDL-C control and lower uses of evidence based therapies in women. I welcome further research that continues to explore ways that we can close these gaps and improve outcomes.”

Professor Raj Thakkar, President and CKD lead, Primary Care Cardiovascular Society, said, “The systematic and proactive population management of lipids is essential to reduce cardiovascular risk burden. To deliver this in an expedient, sustainable and efficient way is paramount. Ensuring all available therapies are used to minimise risk, including combination medicines, is key to delivering these goals.”

Notes to editors

The European Society of Cardiology (ESC) Congress takes place 29 August to 1 September 2025 in Madrid, Spain.

At ESC 2025 further clinical data from the SANTORINI and MILOS studies will be published, see about the trials below.

About SANTORINI

The SANTORINI study, funded by Daiichi Sankyo Europe, is a multinational, prospective, observational study that enrolled 9,602 patients with high and very high CV risk from over 623 sites in 14 countries across Europe. Patients were recruited between March 2020 and February 2021.^{iii iv} The primary objective was to document, in the real-world setting, the effectiveness of current LDL-C management approaches in high- and very high-cardiovascular-risk patients requiring lipid-lowering therapies over a 1-year period.^v

About MILOS

The MILOS study (NCT04579367), funded by Daiichi Sankyo Europe, is an ongoing, multinational, European observational study in adult patients diagnosed with primary hypercholesterolaemia or mixed dyslipidaemia. The aim is to evaluate the real-world use of bempedoic acid and bempedoic plus ezetimibe FDC.^vi

About Bempedoic Acid and its Fixed-Dose Combination with Ezetimibe

Concomitant use of bempedoic acid (NILEMDO^®)/bempedoic acid / ezetimibe FDC (NUSTENDI^®) with simvastatin > 40mg daily is contraindicated.

Bempedoic acid inhibits ATP citrate lyase (ACL), an enzyme which is involved in the production of cholesterol in the liver. Bempedoic acid is indicated in adults with primary hypercholesterolaemia (heterozygous familial and nonfamilial) or mixed dyslipidaemia, as an adjunct to diet:^vii

• in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin or,^viii
• alone or in combination with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.^ix

Bempedoic acid is indicated in adults with established or at high risk for atherosclerotic CV disease to reduce CV risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:

• in patients on a maximum tolerated dose of statin with or without ezetimibe or, ^x
• alone or in combination with ezetimibe in patients who are statin-intolerant, or for whom a statin in contraindicated.^xi

The bempedoic acid / ezetimibe FDC is indicated in adults with primary hypercholesterolaemia (heterozygous familial and nonfamilial) or mixed dyslipidaemia, as an adjunct to diet:^xii

• in combination with a statin in patients unable to reach LDL-C goals with the maximum tolerated dose of a statin in addition to ezetimibe,
• alone in patients who are either statin-intolerant or for whom a statin is contraindicated, and are unable to reach LDL-C goals with ezetimibe alone,
• in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets with or without statin.^xiii

Bempedoic acid / ezetimibe FDC is indicated in adults with established or at high risk for atherosclerotic CV disease to reduce CV risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:²¹

• in patients on a maximum tolerated dose of statin and not adequately controlled with additional ezetimibe treatment or,
• in patients who are either statin-intolerant, or for whom a statin in contraindicated, and not adequately controlled with ezetimibe treatment or,
• in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets.^xiv

Daiichi Sankyo Europe has licensed exclusive commercialisation rights to bempedoic acid and its FDC with ezetimibe in the European Economic Area, UK, Turkey and Switzerland from Esperion and is the full Marketing Authorisation Holder in these territories.

About Daiichi Sankyo

Daiichi Sankyo is an innovative global healthcare company contributing to the sustainable development of society that discovers, develops, and delivers new standards of care to enrich the quality of life around the world. With more than 120 years of experience, Daiichi Sankyo leverages its world-class science and technology to create new modalities and innovative medicines for people with cancer, cardiovascular, and other diseases with high unmet medical need.

For more information, please visit www.daiichi-sankyo.co.uk.

Contacts

James Hargrave

Director of Government Affairs

Daiichi Sankyo UK

James.Hargrave@daiichisankyo.com
+44 7591950136

Genentech and Alnylam Advance Zilebesiran Into Global Phase III Cardiovascular Outcomes Trial for People With Uncontrolled Hypertension




Genentech and Alnylam Advance Zilebesiran Into Global Phase III Cardiovascular Outcomes Trial for People With Uncontrolled Hypertension

–  Phase III trial informed by comprehensive KARDIA data set generated through three Phase II studies: KARDIA-1, KARDIA-2 and KARDIA-3 –

–  In the Phase II KARDIA-3 study, presented today as a late breaker at the European Society of Cardiology Congress 2025, zilebesiran demonstrated clinically meaningful reductions in office systolic blood pressure at month three with continuous control through month six –

– Zilebesiran, a potential best-in-disease RNAi antihypertensive with twice-yearly subcutaneous dosing, demonstrated encouraging safety when combined with two or more antihypertensives –

– Phase III cardiovascular outcomes trial expected to be initiated by the end of the year –

SOUTH SAN FRANCISCO, Calif.–(BUSINESS WIRE)–Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY) and Alnylam (Nasdaq: ALNY) today announced the decision to initiate a Phase III cardiovascular outcomes trial (CVOT) to evaluate the ability of zilebesiran, an RNAi therapeutic, to reduce the risk of major adverse cardiovascular events in patients with uncontrolled hypertension. This decision was informed by the comprehensive KARDIA Phase II program, including KARDIA-1, KARDIA-2 and the most recent KARDIA-3 study evaluating the efficacy and safety of zilebesiran in patients with uncontrolled hypertension and high cardiovascular (CV) risk, on two to four standard of care antihypertensives. In particular, KARDIA-3 aimed to define the patient population to be investigated in the Phase III CV outcomes trial.

Results of KARDIA-3 showed a single dose of zilebesiran (300 mg every six months, subcutaneous injection) resulted in clinically meaningful placebo-adjusted reductions of office systolic blood pressure (SBP) in all comers at the month three primary endpoint (-5.0 mmHg; p=0.0431) with sustained benefits out to month six (-3.9 mmHg; 95% CI: [-8.5, 0.7]). There were no additional benefits of the 600 mg dose at month three (-3.3 mmHg; p=0.1830) or month six (-3.6 mmHg; 95% CI: [-8.2, 1.0]). The overall KARDIA-3 study did not meet the pre-specified definition for statistical significance, because of a multiplicity statistical testing approach. However the study met the aim of identifying the patient population that could potentially benefit the most from zilebesiran and also showed encouraging safety and clinically meaningful placebo adjusted reductions in blood pressure.

As observed in the KARDIA-2 Phase II study, the KARDIA-3 results support a robust benefit of combining zilebesiran with a diuretic, a commonly used antihypertensive. In an analysis of patients that were on diuretics and had a baseline BP > 140 mmHg, the placebo-adjusted reduction was -9.2 mmHg; (-17.3, -1.2) at month three and -8.3 mmHg (-16.4, -0.2) at month six. A precedent for enhanced blood pressure reduction conferred by this type of combination is established in both literature and clinical practice.

“Zilebesiran has the potential to become a best-in-disease treatment for many patients with uncontrolled hypertension. Its blood pressure-lowering effects and twice-yearly dosing could reduce the risk of serious health complications and death,” said Levi Garraway, M.D., Ph.D., Genentech’s chief medical officer and head of Global Product Development. “Detailed analysis of our comprehensive Phase II clinical trials have informed our decision to move zilebesiran into Phase III. Despite current treatment options, up to 80% of people with hypertension do not achieve adequate blood pressure control putting them at higher risk of cardiovascular events. Therefore, additional treatment options are needed.”

Zilebesiran also demonstrated encouraging safety in patients with comorbidities on multiple background therapies – over 90% of whom were receiving treatment with an ACE inhibitor or an Angiotensin Receptor Blocker (ARB). These findings reinforce confidence in zilebesiran’s ability to be combined with standard of care antihypertensives.

As a result, the global ZENITH (ZilebEsiraN cardIovascular ouTcome study in Hypertension) Phase III trial has been submitted to global regulators and is expected to be initiated by the end of 2025. ZENITH will be a CVOT enrolling approximately 11,000 patients and evaluating zilebesiran (300 mg) every six months compared to placebo in patients with uncontrolled hypertension with either established CV disease or at high risk for CV disease on two or more antihypertensives, one being a diuretic.

Hypertension is the primary cause of and number one modifiable risk factor for cardiovascular disease. An estimated one in three adults, over 1.2 billion people worldwide, have hypertension and despite the wide availability of antihypertensives, up to 80% of them do not achieve adequate blood pressure control. Poor adherence to daily oral therapies is an important contributor to poor blood pressure control and CV outcomes. An effective long-acting therapy that provides continuous control of blood pressure may help to reduce the burden of uncontrolled hypertension.

With its growing cardiometabolic portfolio and strong diagnostic expertise, Genentech is advancing transformative standards of care to improve the lives of people living with cardiometabolic diseases as well as reducing the significant burden on healthcare systems and society.

About the KARDIA-3 Study

KARDIA-3 (NCT06272487), the third Phase II study in the KARDIA program, included two Cohorts (A and B). Cohort A assessed zilebesiran in patients with eGFR ≥ 45 mL/min/1.73m2, while Cohort B included patients with advanced kidney dysfunction (i.e., eGFR between 30 and <45 mL/min/1.73m2). Cohort A enrolled 270 patients who were randomized to treatment with zilebesiran (300 mg or 600 mg) or placebo. Randomization was stratified by background diuretic use, baseline blood pressure, and race. The primary endpoint was change in office SBP at month three. Key secondary endpoints were changes in office SBP at month six and change in 24-hour mean ambulatory SBP at months three and six.

At baseline, 144 (53.3%), 96 (35.6%) and 30 (11.1%) patients were on two, three, or over three antihypertensives, respectively, with ~91% of patients taking ACE inhibitors/ARBs, ~66% of patients taking a diuretic, and ~58% of patients taking calcium channel blockers. The mean baseline office and 24-hour mean ambulatory SBP were 143.6 mmHg and 142.4 mmHg, respectively (N= 270).

KARDIA-3 Cohort A Primary Results (Placebo-Adjusted Changes from Baseline):

In summary, in the Cohort A overall study population, zilebesiran 300 mg achieved clinically meaningful reductions in office SBP at month three, with sustained benefits out to month six, compared to placebo. No incremental SBP reductions were observed with zilebesiran 600 mg at months three or six. Post-hoc analyses suggest that a greater blood pressure-lowering effect with zilebesiran was observed in patients on diuretic therapy and uncontrolled hypertension at baseline (with office SBP ≥140). Reductions in blood pressure were sustained over six months, and the entire 24-hour period. Incremental reductions were also observed at nighttime, a period during which blood pressure elevations is a strong predictor of cardiovascular risk.

Consistent with prior studies, zilebesiran demonstrated an encouraging safety profile when added to two or more background antihypertensives (over 90% of whom were receiving treatment with an ACE inhibitor or an ARB). Most adverse events were mild or moderate, non-serious, and transient with few requiring intervention; rates of hyperkalaemia, kidney dysfunction, and hypotension were low. Across study arms, serious adverse events were observed in 3.8% and 4.5% in zilebesiran and placebo-treated patients, respectively. No deaths were reported during the six-month double-blind period.

Results from KARDIA-3 Cohort B are expected to be presented at an upcoming medical meeting.

About the ZENITH CVOT

The global Phase III ZENITH CVOT is an event-driven study that will enroll approximately 11,000 patients in over 30 countries to evaluate zilebesiran 300 mg in patients with uncontrolled hypertension, despite the use of at least two standard of care antihypertensives (one being a diuretic), and with either established cardiovascular disease (CVD) or at high risk for CVD. The primary objective will be to assess the impact of zilebesiran on reducing the risk of CV death, nonfatal myocardial infarction (MI), nonfatal stroke, or heart failure (HF) events (hospitalization for HF or urgent HF visit), compared to placebo.

About Zilebesiran

Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of hypertension to reduce cardiovascular risk in high unmet need populations. Zilebesiran targets angiotensinogen (AGT), the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a role in blood pressure (BP) regulation. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein, and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam’s Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent biannual subcutaneous dosing and increased selectivity. Zilebesiran has demonstrated the ability to provide continuous control of blood pressure with biannual dosing in patients with mild-to-moderate hypertension as a monotherapy and in combination with standard-of-care antihypertensives, as well as in patients with high cardiovascular risk and uncontrolled hypertension despite the use of multiple background therapies. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche.

Zilebesiran Phase II clinical development overview:

About Cardiovascular Disease and Hypertension

Cardiovascular disease (CVD) is a global health crisis and a leading cause of death worldwide, responsible for approximately 20 million deaths annually. Hypertension is the primary cause of and number one modifiable risk factor for CVD. An estimated one in three adults worldwide have hypertension and despite wide availability of antihypertensives, up to 80% of all patients and up to a third of treated patients do not reach and maintain blood pressure (BP) targets. Even when blood pressure appears well managed, continuous control of BP may remain suboptimal, leading to variability in BP during the 24-hour period and in the long-term, putting patients at greater risk of cardiovascular events and end organ damage. These patients require novel approaches that not only reduce BP but also lower overall cardiovascular risk.

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.

Contacts

Media Contacts: Nicolette Baker (650) 467-6800

Advocacy Contact: Jenee Williams (650) 303-2958

Investor Contacts: Loren Kalm (650) 225-3217

Bruno Eschli +41 61 687 5284