CLINICAL TRIALS PROFILE FOR PEMBROLIZUMAB

Biosimilar Clinical Trials for pembrolizumab

This table shows clinical trials for biosimilars. See the next table for all clinical trials

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT03988036 ↗	A Study With Pembrolizumab in Combination With Dual Anti-HER2 Blockade With Trastuzumab and Pertuzumab in Early Breast Cancer Patients With Molecular HER2-enriched Intrinsic Subtype (Keyriched-1)	Recruiting	Amgen	Phase 2	2020-08-18	Keyriched-1 is a multicenter, interventional, prospective, single arm, open label, neoadjuvant phase II trial evaluating the pathological complete response (pCR) rate induced by pembrolizumab in combination with the dual anti-HER2 blockade consisting of trastuzumab biosimilar ABP 980 and pertuzumab in early breast cancer patients with molecular HER2-enriched intrinsic subtype tested by PAM50.
NCT03988036 ↗	A Study With Pembrolizumab in Combination With Dual Anti-HER2 Blockade With Trastuzumab and Pertuzumab in Early Breast Cancer Patients With Molecular HER2-enriched Intrinsic Subtype (Keyriched-1)	Recruiting	Merck Sharp & Dohme Corp.	Phase 2	2020-08-18	Keyriched-1 is a multicenter, interventional, prospective, single arm, open label, neoadjuvant phase II trial evaluating the pathological complete response (pCR) rate induced by pembrolizumab in combination with the dual anti-HER2 blockade consisting of trastuzumab biosimilar ABP 980 and pertuzumab in early breast cancer patients with molecular HER2-enriched intrinsic subtype tested by PAM50.
NCT03988036 ↗	A Study With Pembrolizumab in Combination With Dual Anti-HER2 Blockade With Trastuzumab and Pertuzumab in Early Breast Cancer Patients With Molecular HER2-enriched Intrinsic Subtype (Keyriched-1)	Recruiting	NanoString Technologies, Inc.	Phase 2	2020-08-18	Keyriched-1 is a multicenter, interventional, prospective, single arm, open label, neoadjuvant phase II trial evaluating the pathological complete response (pCR) rate induced by pembrolizumab in combination with the dual anti-HER2 blockade consisting of trastuzumab biosimilar ABP 980 and pertuzumab in early breast cancer patients with molecular HER2-enriched intrinsic subtype tested by PAM50.

All Clinical Trials for pembrolizumab

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01042379 ↗	I-SPY TRIAL: Neoadjuvant and Personalized Adaptive Novel Agents to Treat Breast Cancer	Recruiting	QuantumLeap Healthcare Collaborative	Phase 2	2010-03-01	The purpose of this study is to further advance the ability to practice personalized medicine by learning which new drug agents are most effective with which types of breast cancer tumors and by learning more about which early indicators of response (tumor analysis prior to surgery via magnetic resonance imaging (MRI) images along with tissue and blood samples) are predictors of treatment success.
NCT01174121 ↗	Immunotherapy Using Tumor Infiltrating Lymphocytes for Patients With Metastatic Cancer	Suspended	National Cancer Institute (NCI)	Phase 2	2010-08-26	Background: The NCI Surgery Branch has developed an experimental therapy that involves taking white blood cells from patients' tumors, growing them in the laboratory in large numbers, and then giving the cells back to the patient. These cells are called Tumor Infiltrating Lymphocytes, or TIL and we have given this type of treatment to over 200 patients with melanoma. Researchers want to know if TIL shrink s tumors in people with digestive tract, urothelial, breast, or ovarian/endometrial cancers. In this study, we are selecting a specific subset of white blood cells from the tumor that we think are the most effective in fighting tumors and will use only these cells in making the tumor fighting cells. Objective: The purpose of this study is to see if these specifically selected tumor fighting cells can cause digestive tract, urothelial, breast, or ovarian/endometrial tumors to shrink and to see if this treatment is safe. Eligibility: - Adults age 18-70 with upper or lower gastrointestinal, hepatobiliary, genitourinary, breast, ovarian/endometrial cancer, or glioblastoma refractory to standard chemotherapy. Design: Work up stage: Patients will be seen as an outpatient at the NIH clinical Center and undergo a history and physical examination, scans, x-rays, lab tests, and other tests as needed. Surgery: If the patients meet all of the requirements for the study they will undergo surgery to remove a tumor that can be used to grow the TIL product. Leukapheresis: Patients may undergo leukapheresis to obtain additional white blood cells. {Leukapheresis is a common procedure, which removes only the white blood cells from the patient.} Treatment: Once their cells have grown, the patients will be admitted to the hospital for the conditioning chemotherapy, the TIL cells and aldesleukin. They will stay in the hospital for about 4 weeks for the treatment. Follow up: Patients will return to the clinic for a physical exam, review of side effects, lab tests, and scans about every 1-3 months for the first year, and then every 6 months to 1 year as long as their tumors are shrinking. Follow up visits will take up to 2 days.
NCT01295827 ↗	Study of Pembrolizumab (MK-3475) in Participants With Progressive Locally Advanced or Metastatic Carcinoma, Melanoma, or Non-small Cell Lung Carcinoma (P07990/MK-3475-001/KEYNOTE-001)	Active, not recruiting	Merck Sharp & Dohme Corp.	Phase 1	2011-03-01	This study will be done in 6 parts. In Part A the dose of intravenous (IV) pembrolizumab (MK-3475) will be escalated to find the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) for participants with a histologically or cytologically confirmed diagnosis of any type of carcinoma or melanoma (MEL). Part B of the study will explore the safety, tolerability, and efficacy of the drug in participants with advanced or metastatic MEL and compare every 2 week to every 3 week dosing. Part C of the study will explore the safety, tolerability, and efficacy of the drug in participants with non-small cell lung carcinoma (NSCLC) that is locally advanced or metastatic. Part D of the study will explore the low and high doses of study drug identified in Parts A and B in participants with advanced or metastatic MEL. Part E (closed with Amendment 7) will explore low, medium, and high doses of study drug in combination with standard chemotherapy in participants with locally advanced or metastatic NSCLC. Part F will explore low and high doses of study drug in treatment-naive and previously-treated participants with NSCLC with programmed cell death 1 ligand (PD-L1) gene expression. In Parts D and F and some of Part B participants will be randomized to one dose level. The primary hypotheses are the following: that pembrolizumab has acceptable safety and tolerability; and that pembrolizumab shows a clinically meaningful response rate (RR) or disease-control rate (DCR) in participants with melanoma (ipilimumab-refractory or not), and a clinically meaningful RR in participants with NSCLC, especially a clinically meaningful RR in those participants with either cancer, whose tumors express PD-L1.

Clinical Trial Conditions for pembrolizumab

Condition Name

Condition Name for pembrolizumab
Intervention	Trials
Non-small Cell Lung Cancer	133
Melanoma	116
Breast Cancer	91

Condition MeSH

Condition MeSH for pembrolizumab
Intervention	Trials
Carcinoma	401
Carcinoma, Non-Small-Cell Lung	374
Lung Neoplasms	291

Clinical Trial Locations for pembrolizumab

Trials by Country

Trials by Country for pembrolizumab
Location	Trials
Japan	949
France	823
United States	7,373
Spain	681
Australia	553

Trials by US State

Trials by US State for pembrolizumab
Location	Trials
California	495
Texas	469
New York	411
Pennsylvania	337
Florida	331

Clinical Trial Progress for pembrolizumab

Clinical Trial Phase

Clinical Trial Phase for pembrolizumab
Clinical Trial Phase	Trials
PHASE4	7
PHASE3	73
PHASE2	182

Clinical Trial Status

Clinical Trial Status for pembrolizumab
Clinical Trial Phase	Trials
Recruiting	1042
Not yet recruiting	423
Active, not recruiting	310

Clinical Trial Sponsors for pembrolizumab

Sponsor Name

Sponsor Name for pembrolizumab
Sponsor	Trials
Merck Sharp & Dohme Corp.	711
National Cancer Institute (NCI)	195
Merck Sharp & Dohme LLC	158

Sponsor Type

Sponsor Type for pembrolizumab
Sponsor	Trials
Industry	1976
Other	1905
NIH	202

Clinical Trials Update, Market Analysis, and Projection for Pembrolizumab

Last updated: November 21, 2025

Introduction

Pembrolizumab, commercially known as Keytruda, is a PD-1 (programmed death-1) immune checkpoint inhibitor developed by Merck & Co. Since its FDA approval in 2014, pembrolizumab has transformed cancer immunotherapy, becoming one of the most commercially successful oncology drugs globally. This report provides an updated analysis of its ongoing clinical trials, assesses its current market landscape, and projects future growth trajectories amid evolving cancer treatment paradigms.

Clinical Trials Landscape

Recent Clinical Trials and Indications

Pembrolizumab has been evaluated across a broad spectrum of malignancies, with an extensive pipeline of ongoing clinical trials. As of 2023, over 400 active clinical trials investigate its efficacy in indications including melanoma, non-small cell lung cancer (NSCLC), gastric cancer, Hodgkin lymphoma, and emerging applications such as triple-negative breast cancer and neuro-oncology. Notably:

• Keytruda in NSCLC: Continuing Phase III trials (e.g., KEYNOTE-598) assess combinations with chemotherapy and targeted therapies. These aim to expand indications, notably in treatment-naïve and resistant settings.
• Combination Trials: Trials combining pembrolizumab with other immune checkpoint inhibitors or novel agents—like LAG-3 inhibitors—seek to enhance efficacy and overcome resistance.
• Adjuvant and Neoadjuvant Settings: Several Phase III trials evaluate pembrolizumab as an adjuvant therapy in melanoma (e.g., KEYNOTE-716) and other tumor types, reflecting a strategic expansion into earlier treatment lines.
• Biomarker-driven Studies: Emphasis on PD-L1 expression levels influences trial designs and patient selection, optimizing therapeutic outcomes.

Regulatory Status and Approvals

Beyond its initial approvals for melanoma, NSCLC, and head and neck cancers, pembrolizumab has gained broad regulatory acceptance. As of 2023, approvals include:

• Gastric and gastroesophageal junction cancers (MSI-H/dMMR tumors).
• Cervical and anal carcinomas.
• Hodgkin lymphoma in relapsed/refractory settings.
• Tamoxifen-resistant triple-negative breast cancer.

The expanding indication portfolio underscores its flexible mechanism and robust clinical evidence supporting efficacy.

Innovative Clinical Strategies

Emerging clinical strategies include:

• Combination regimens with targeted therapies, chemotherapy, and radiation.
• Biomarker-based selection to optimize patient stratification and efficacy.
• Neoadjuvant/adjuvant therapy to improve long-term outcomes.
• Real-world evidence (RWE) initiatives to validate trial findings and inform broader use.

Market Analysis

Current Market Position

Pembrolizumab ranks among the top-selling oncology drugs, generating approximately $13 billion in annual revenues in 2022, according to IQVIA data. Its success stems from:

• Broad efficacy across multiple cancers.
• Favorable safety profile.
• FDA approvals expanding the patient population.
• Strategic combination therapies enhancing its clinical utility.

Competitive Landscape

Key competitors include other checkpoint inhibitors:

• Nivolumab (Opdivo): Developed by Bristol-Myers Squibb, with overlapping indications.
• Atezolizumab (Tecentriq): Roche's PD-L1 inhibitor, with significant market share.
• Durvalumab (Imfinzi): AstraZeneca’s PD-L1 agent, especially in lung and bladder cancers.

Despite intense competition, pembrolizumab maintains a leading position owing to its broader approvals and extensive pipeline.

Market Penetration and Challenges

While penetration remains strong in developed markets (North America, Europe, Japan), challenges persist:

• Price pressures and reimbursement hurdles.
• Emergence of biosimilars in certain regions.
• Resistance mechanisms limiting durability of responses.
• Patient selection variability, impacting real-world effectiveness.

Market Growth Projections

Industry analysts project the global pembrolizumab market will grow at a compound annual growth rate (CAGR) of approximately 12-15% over the next five years. Key factors driving this growth include:

• Expansion into earlier-line therapies.
• Approval for additional indications, including rare and pediatric cancers.
• Combination strategies increasing overall market size.
• Increasing prevalence of target cancers and better screening.

Forecasts indicate that by 2028, the global market could surpass $30 billion, solidifying pembrolizumab’s position as a cornerstone agent in oncology.

Future Outlook

Pipeline and Innovation

Merck continually advances its pipeline, with anticipated approvals potentially adding to the current indications:

• Novel combination therapies could redefine standard care.
• Predictive biomarkers may enable personalized treatments.
• Next-generation checkpoint inhibitors and related immunotherapies could coexist, fostering competition but also highlighting distal innovation.

Regulatory and Scientific Trends

Ongoing emphasis on:

• Early diagnosis and intervention.
• Combination regimens for resistant cancers.
• Real-world data integration to validate trial outcomes.

Regulatory agencies increasingly endorse immunotherapy in earlier treatment settings, promising further expansion.

Market Risks

Potential risks include:

• Regulatory setbacks due to safety concerns.
• Market saturation in established indications.
• Cost-effectiveness debates, especially in lower-income regions.
• Development of resistance mechanisms threatening long-term efficacy.

Key Takeaways

• Clinical Trials Indicate Broad Expansion: Pembrolizumab’s extensive ongoing trials aim to solidify its role across diverse cancers, emphasizing combinations and earlier treatment phases.
• Market Leadership Maintains: Despite competitive pressures, pembrolizumab remains a dominant force, driven by its broad approval base and pipeline-driven innovations.
• Future Growth Driven by Indication Expansion: Expected approvals for additional indications and combination therapies are poised to sustain double-digit growth rates.
• Strategic Challenges Persist: Cost considerations, resistance mechanisms, and competitive dynamics necessitate strategic innovation and patient selection improvements.
• Projections Favor Continued Growth: Industry forecasts indicate a robust trajectory, with the global market potentially surpassing $30 billion by 2028, affirming pembrolizumab’s strategic importance in oncology.

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FAQs

1. What are the key indications for pembrolizumab currently approved by regulatory agencies?
Pembrolizumab is approved for multiple indications, including melanoma, NSCLC, Hodgkin lymphoma, microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) tumors, gastric cancer, cervical cancer, and Merkel cell carcinoma.

2. How does pembrolizumab outperform its competitors in the market?
Its broad spectrum of approved indications, extensive clinical evidence, favorable safety profile, and strategic combination research have established pembrolizumab as a market leader compared to similar checkpoint inhibitors.

3. What are the main challenges facing pembrolizumab’s future market growth?
Challenges include rising treatment costs, emerging resistance, increased competition from biosimilars, and regulatory hurdles in expanding into new markets or indications.

4. Which emerging clinical trial developments could reshape pembrolizumab’s application?
Trials focusing on early-stage cancers, combination regimens with novel agents, and personalized biomarker-driven approaches are poised to deepen pembrolizumab’s clinical utility.

5. What is the projected market growth for pembrolizumab over the next five years?
Analysts project a CAGR of approximately 12-15%, with the global market surpassing $30 billion by 2028, reflecting continued demand driven by indication expansion and innovations.

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References

[1] IQVIA. (2022). Oncology market report.
[2] FDA. (2023). Pembrolizumab (Keytruda) approvals and indications.
[3] ClinicalTrials.gov. (2023). Pembrolizumab ongoing trials.
[4] Merck & Co. Annual Report 2022.
[5] GlobalData. (2023). Oncology immunotherapy market analysis.