CLINICAL TRIALS PROFILE FOR BINIMETINIB

All Clinical Trials for binimetinib

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01562899 ↗	A Study of MEK162 and AMG 479 in Patients With Selected Solid Tumors	Terminated	Array BioPharma	Phase 1/Phase 2	2012-08-27	This is a multi-center, open-label, phase Ib/II study. First, the aim of the phase Ib part is to estimate the MTD(s) and/or to identify the recommended phase II dose(s) (RP2D) for the combination of MEK162 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. The dose escalation part of the study will be guided by a Bayesian Logistic Regression Model (BLRM). At least 18 patients are expected to be enrolled in the dose escalation part. Following MTD/ RP2D declaration, patients will be enrolled in three phase II arms to assess efficacy of the combination as well as to better understand the safety, tolerability, PK, antibody concentrations and PD of the combination at MTD/RP2D. Phase II arm 1 will consist of approximately 25 patients with KRAS-mutant colorectal adenocarcinoma. Phase II arm 2 will consist of approximately 20 patients with metastatic pancreatic adenocarcinoma. Phase II arm 3 will consist of approximately 28 patients with mutant BRAFV600 melanoma. Patients will be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurs first. All patients will be followed up - at minimum patients must complete the safety follow-up assessments 30 days after the last dose of the study treatment.
NCT01562899 ↗	A Study of MEK162 and AMG 479 in Patients With Selected Solid Tumors	Terminated	Pfizer	Phase 1/Phase 2	2012-08-27	This is a multi-center, open-label, phase Ib/II study. First, the aim of the phase Ib part is to estimate the MTD(s) and/or to identify the recommended phase II dose(s) (RP2D) for the combination of MEK162 and AMG 479 (ganitumab), followed by the phase II part to assess the clinical efficacy and to further assess the safety of the combination in selected patient populations. The dose escalation part of the study will be guided by a Bayesian Logistic Regression Model (BLRM). At least 18 patients are expected to be enrolled in the dose escalation part. Following MTD/ RP2D declaration, patients will be enrolled in three phase II arms to assess efficacy of the combination as well as to better understand the safety, tolerability, PK, antibody concentrations and PD of the combination at MTD/RP2D. Phase II arm 1 will consist of approximately 25 patients with KRAS-mutant colorectal adenocarcinoma. Phase II arm 2 will consist of approximately 20 patients with metastatic pancreatic adenocarcinoma. Phase II arm 3 will consist of approximately 28 patients with mutant BRAFV600 melanoma. Patients will be treated until progression of disease, unacceptable toxicity develops, or withdrawal of informed consent, whichever occurs first. All patients will be followed up - at minimum patients must complete the safety follow-up assessments 30 days after the last dose of the study treatment.
NCT01801358 ↗	A Phase Ib/II Study of AEB071 and MEK162 in Adult Patients With Metastatic Uveal Melanoma	Terminated	Array BioPharma	Phase 1/Phase 2	2013-08-01	A phase Ib dose-escalation study of the AEB071 and MEK162 combination in adult patients with confirmed metastatic uveal melanoma. Cohorts of 3-6 patients will be assessed for dose limiting toxicities (DLTs) during Cycle 1 until the maximum tolerated dose (MTD) of the combination therapy is determined. The MTD or Phase 2 Recommended Dose (P2RD) will be used in a Phase II part of the study, which will enrol 55 patients each into two randomized groups: the combination therapy or MEK162 alone. The Phase II part will continue until proof of concept is established. Patients will continue treatment as long as clinical benefit is seen and no limiting adverse toxicity is observed
NCT01801358 ↗	A Phase Ib/II Study of AEB071 and MEK162 in Adult Patients With Metastatic Uveal Melanoma	Terminated	Array Biopharma, now a wholly owned subsidiary of Pfizer	Phase 1/Phase 2	2013-08-01	A phase Ib dose-escalation study of the AEB071 and MEK162 combination in adult patients with confirmed metastatic uveal melanoma. Cohorts of 3-6 patients will be assessed for dose limiting toxicities (DLTs) during Cycle 1 until the maximum tolerated dose (MTD) of the combination therapy is determined. The MTD or Phase 2 Recommended Dose (P2RD) will be used in a Phase II part of the study, which will enrol 55 patients each into two randomized groups: the combination therapy or MEK162 alone. The Phase II part will continue until proof of concept is established. Patients will continue treatment as long as clinical benefit is seen and no limiting adverse toxicity is observed
NCT02041481 ↗	MEK Inhibitor MEK162 in Combination With Leucovorin Calcium, Fluorouracil, and Oxaliplatin in Treating Patients With Advanced Metastatic Colorectal Cancer	Completed	Array BioPharma	Phase 1	2014-06-01	This phase I trial studies the side effects and best dose of MEK inhibitor MEK162 when given together with leucovorin calcium, fluorouracil, and oxaliplatin in treating patients with advanced metastatic colorectal cancer. MEK inhibitor MEK162 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as leucovorin calcium, fluorouracil, and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving MEK inhibitor MEK162 with leucovorin calcium, fluorouracil, and oxaliplatin may kill more tumor cells.
NCT02041481 ↗	MEK Inhibitor MEK162 in Combination With Leucovorin Calcium, Fluorouracil, and Oxaliplatin in Treating Patients With Advanced Metastatic Colorectal Cancer	Completed	National Cancer Institute (NCI)	Phase 1	2014-06-01	This phase I trial studies the side effects and best dose of MEK inhibitor MEK162 when given together with leucovorin calcium, fluorouracil, and oxaliplatin in treating patients with advanced metastatic colorectal cancer. MEK inhibitor MEK162 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as leucovorin calcium, fluorouracil, and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving MEK inhibitor MEK162 with leucovorin calcium, fluorouracil, and oxaliplatin may kill more tumor cells.
NCT02041481 ↗	MEK Inhibitor MEK162 in Combination With Leucovorin Calcium, Fluorouracil, and Oxaliplatin in Treating Patients With Advanced Metastatic Colorectal Cancer	Completed	City of Hope Medical Center	Phase 1	2014-06-01	This phase I trial studies the side effects and best dose of MEK inhibitor MEK162 when given together with leucovorin calcium, fluorouracil, and oxaliplatin in treating patients with advanced metastatic colorectal cancer. MEK inhibitor MEK162 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs used in chemotherapy, such as leucovorin calcium, fluorouracil, and oxaliplatin, work in different ways to stop the growth of tumor cells, either by killing the cells or by stopping them from dividing. Giving MEK inhibitor MEK162 with leucovorin calcium, fluorouracil, and oxaliplatin may kill more tumor cells.

Clinical Trial Conditions for binimetinib

Condition Name

Condition Name for binimetinib
Intervention	Trials
Melanoma	14
Metastatic Melanoma	10
Colorectal Cancer	6
BRAF V600 Mutation	6

Condition MeSH

Condition MeSH for binimetinib
Intervention	Trials
Melanoma	39
Colorectal Neoplasms	18
Carcinoma, Non-Small-Cell Lung	12
Lung Neoplasms	12

Clinical Trial Locations for binimetinib

Trials by Country

Trials by Country for binimetinib
Location	Trials
United States	307
Spain	45
China	38
Italy	35
Germany	30

Trials by US State

Trials by US State for binimetinib
Location	Trials
California	27
Texas	23
Florida	16
New York	16
Massachusetts	16

Clinical Trial Progress for binimetinib

Clinical Trial Phase

Clinical Trial Phase for binimetinib
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	3
Phase 4	1

Clinical Trial Status

Clinical Trial Status for binimetinib
Clinical Trial Phase	Trials
Recruiting	53
Not yet recruiting	20
Active, not recruiting	10

Clinical Trial Sponsors for binimetinib

Sponsor Name

Sponsor Name for binimetinib
Sponsor	Trials
Pfizer	27
Array BioPharma	26
National Cancer Institute (NCI)	22

Sponsor Type

Sponsor Type for binimetinib
Sponsor	Trials
Industry	111
Other	102
NIH	22

Clinical Trials Update, Market Analysis, and Projection for Binimetinib

Last updated: October 28, 2025

Introduction

Binimetinib, marketed as Mektovi, is a potent MEK 1/2 inhibitor developed by Array BioPharma (recently acquired by Pfizer) targeting the mitogen-activated protein kinase (MAPK) pathway. Its primary indication involves the treatment of BRAF V600-mutant melanoma, with ongoing investigations exploring its efficacy across various cancers. This report provides a comprehensive update on binimetinib's clinical trial landscape, analyzes its current market dynamics, and projects future market growth.

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Clinical Trials Landscape for Binimetinib

Regulatory Approvals and Clinical Development

Since its FDA approval in 2018 for BRAF V600-mutant melanoma in combination with encorafenib, binimetinib has established a critical role in targeted oncology therapy. Its approval, granted under accelerated pathways, was based on data demonstrating improved progression-free survival (PFS) and overall response rates (ORR).

Ongoing Clinical Trials

As of early 2023, binimetinib remains active in multiple clinical trials focusing on:

• Combination Therapy in Melanoma: Trials assessing its synergistic potential with other agents, such as immunotherapies, to enhance durability of response. For instance, Phase II studies evaluate its combination with pembrolizumab in advanced melanoma (NCT04555393).

• Other Solid Tumors: Investigations into non-small cell lung cancer (NSCLC), colorectal cancers, and low-grade ovarian tumors. Notably, the NCI initiated a phase II trial combining binimetinib with standard chemotherapy in KRAS-mutant NSCLC (NCT03875820).

• Tumor-Specific Biomarker Trials: Efforts are underway to refine patient selection through molecular profiling, such as identifying NRAS-mutant melanoma where MEK inhibition might be beneficial.

Key Clinical Trial Results

• Mektovi + Encorafenib (COLUMBUS trial): Demonstrated significant improvements in median PFS (14.9 months vs. 7.3 months for monotherapy) and ORR (66% vs. 20%) in BRAF V600-mutant melanoma.[1]

• Combination with Other Agents: Early data from phase I/II trials suggest feasible safety profiles when combined with PD-1 inhibitors, though durability remains underevaluation.

Challenges in Clinical Development

• Resistance Development: Despite initial efficacy, resistance mechanisms such as reactivation of MAPK pathway components diminish long-term responses.

• Toxicity Management: Adverse events like dermatitis, diarrhea, and cardiomyopathy necessitate vigilant management, which could affect trial designs and medication adherence.

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Market Analysis of Binimetinib

Market Overview

The global targeted oncology therapeutics market surpassed USD 150 billion in 2022, with a growing focus on precision medicine and targeted inhibitors such as binimetinib. The BRAF V600-mutant melanoma segment remains the primary revenue driver, with increased penetration driven by clinical success and regulatory approvals.

Current Market Position

Since its launch in 2018, binimetinib holds a pivotal position among MEK inhibitors, competing with drugs like trametinib (Novartis) and cobimetinib (Genentech). Its market share has been bolstered by favorable efficacy profiles when combined with BRAF inhibitors.

Key Market Drivers

• Efficacy Data and Regulatory Approvals: Strong clinical data have facilitated broad adoption in melanoma.

• Increasing Incidence of Melanoma: The American Cancer Society estimates over 97,000 new melanoma cases in the U.S. in 2022, underpinning demand.[2]

• Expanding Therapeutic Indications: Trials exploring binimetinib's efficacy in other cancers could open new markets.

Market Challenges

• Pricing and Reimbursement: As with many targeted therapies, high costs may limit access, especially in emerging markets.

• Competition: Superior or comparable efficacy data for emerging drugs could erode binimetinib's market share.

• Resistance and Relapse: Long-term survival benefits hinge on overcoming resistance mechanisms.

Market Projections (2023–2030)

Based on current dynamics, the binimetinib market is expected to grow at a CAGR of approximately 8–10%, reaching around USD 750–800 million by 2030 (from an estimated USD 350 million in 2022), driven by:

• Expanded indications through ongoing clinical trials.
• Increasing adoption in combination regimes with immunotherapies.
• Geographical expansion into Asia and Europe.

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Future Outlook and Strategic Opportunities

Potential Expansion into Other Cancers

Binimetinib's targeted mechanism suggests promising application in NRAS-mutant melanoma, colorectal cancer, and certain low-grade gliomas, pending positive trial outcomes.

Combination Therapies

Augmenting binimetinib with immune checkpoint inhibitors could overcome resistance and prolong response duration. These combination strategies are currently under clinical evaluation.

Biomarker-Driven Patient Selection

Advances in molecular diagnostics enable more precise deployment of binimetinib, optimizing therapeutic outcomes and reducing unnecessary exposure.

Partnerships and Licensing

Partnering with biotech firms for novel combination trials or expanding into emerging markets via licensing agreements could bolster sales.

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Key Takeaways

• Clinical Development: Binimetinib remains an active agent in ongoing trials, especially in melanoma and KRAS-mutant NSCLC, with promising strategies around combination therapy and biomarker selection.

• Market Position: It maintains a significant share within the MEK inhibitor space, driven by proven efficacy and FDA approval for melanoma, with potential to expand its indications.

• Growth Forecast: The drug's market is projected to grow at a healthy rate (~8–10% CAGR) through 2030, driven by clinical successes, new trial data, and expanding applications.

• Challenges: Overcoming resistance, managing toxicity, and competing with other targeted or immunotherapies remain critical for sustained growth.

• Strategic Opportunities: Investment in combination therapies, biomarker-driven patient selection, and geographic expansion represent avenues for continued success.

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FAQs

Q1: What are the primary approved indications for binimetinib?
A1: The FDA-approved indication for binimetinib is in combination with encorafenib for the treatment of patients with BRAF V600-mutant melanoma who have progressed on prior therapies.

Q2: Are there ongoing trials targeting binimetinib in non-melanoma cancers?
A2: Yes, numerous trials are exploring its efficacy in KRAS-mutant NSCLC, colorectal cancers, and other solid tumors, often in combination with chemotherapies or immunotherapies.

Q3: What are the main adverse effects associated with binimetinib?
A3: Common adverse events include rash, diarrhea, fatigue, and edema. Serious effects may involve cardiomyopathy, hepatotoxicity, and ocular toxicity, requiring vigilant monitoring.

Q4: How does binimetinib compare with other MEK inhibitors?
A4: While efficacy profiles are comparable, binimetinib’s differentiators include its safety profile and specific approval in combination regimens with BRAF inhibitors, which may influence prescription patterns.

Q5: What are the key factors influencing binimetinib's market growth?
A5: Prospects for growth hinge on expanding indications through ongoing trials, successful combination therapies, improved biomarker strategies, and market penetration in emerging economies.

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References

1. European Medicines Agency. Mektovi (binimetinib): Summary of Product Characteristics.
2. American Cancer Society. Cancer Facts & Figures 2022.