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

Last updated: July 28, 2026

Binimetinib Clinical Trials Update, Market Analysis, and Exclusivity/Patent Risk Outlook (2026)

Binimetinib (MEK inhibitor; brand: Mektovi) is used in combination with encorafenib for BRAF V600-mutant metastatic melanoma. Post-approval clinical activity concentrates on additional melanoma combinations, earlier-line disease settings, and translational biomarker work tied to MEK/RAF pathway resistance. Commercial trajectory is shaped by (1) ongoing penetration of encorafenib/binimetinib regimens after first-line BRAFV600 disease, (2) competitive substitution from PD-1–first strategies and next-generation RAF/MEK combinations, and (3) patent and exclusivity structure that delays generic erosion.

What is the latest clinical trials update for binimetinib?

High-level status. Binimetinib’s clinical footprint remains anchored to encorafenib combinations, with most incremental development focused on melanoma cohorts, resistance mechanisms, and combination rationale rather than standalone binimetinib monotherapy trials.

Which ongoing or recently reported binimetinib studies matter commercially?

The commercial signal for binimetinib is driven by trials that can expand:

• Eligible lines of therapy (earlier lines vs later line)
• Patient subsets (BRAFV600E/K, high-risk resected disease or adjuvant/neo-adjuvant strategies where biologic rationale aligns)
• Indications beyond melanoma (where signal-to-safety supports pathway-anchored combinations)
• Combination sequencing with immunotherapy

What clinical endpoints are being used for binimetinib progression?

Across MEK inhibitor development, studies typically track:

• ORR, DoR, PFS (primary or key secondary endpoints)
• OS in later-phase confirmatory settings
• Safety tolerability to support longer duration dosing in earlier lines

How does binimetinib perform in the clinic versus standard-of-care?

Positioning. Binimetinib is a core component of the encorafenib plus binimetinib regimen, which is used when BRAFV600 mutation and clinical eligibility align. In practice, competitive advantage depends on:

• Treatment duration and tolerability
• Response durability in typical real-world melanoma progression patterns
• Manageability of MEK-class AEs (rash, diarrhea, edema, ocular events), which influences adherence and dose intensity

What patient populations drive clinical and commercial uptake?

Uptake is strongest where:

• BRAFV600 mutation testing is routine
• Patients are candidates for targeted therapy-based algorithms
• Regimens are used in settings where PD-1-based options do not dominate initial treatment decisions

When does binimetinib lose exclusivity and what is the Paragraph IV risk?

Executive answer. Exclusivity and patent protection for binimetinib are tied to its combination with encorafenib (MEK inhibitor + RAF inhibitor), with the highest regulatory and litigation risk centered on combination product patent coverage and formulation/process claims.

What is the Orange Book status of binimetinib/encorafenib?

Binimetinib’s exclusivity posture should be assessed in the FDA Orange Book for the encorafenib/binimetinib fixed regimen products. The risk profile generally maps as:

• Drug substance patents: earliest expiration
• Drug product/formulation patents: mid-term extensions via solid state, polymorph, and stability-related claims
• Combination method-of-use patents: later expiration, especially if claims cover specific therapeutic methods, dosing regimens, or patient selection

How much patent coverage typically blocks generic encorafenib/binimetinib combinations?

In MEK/RAF combination products, blocking is usually multi-layer:

• Composition or salt/form claim coverage on at least one component
• Combination or method-of-use claims tying the regimen to specific patient types and outcomes
• Manufacturing process and intermediates that constrain “workaround” manufacturing

When could biosimilar-type challenges be relevant?

Not applicable. Binimetinib is a small molecule; biosimilar frameworks do not apply.

What generic entry risks exist for binimetinib?

Key risk drivers:

• Whether combination-covering claims remain in force
• Whether alternative dosing or formulation approaches can avoid claim scope
• Whether challengers can carve out non-infringing strengths/dosing

What formulations are protected for binimetinib and why does that matter for generic entry?

Commercial relevance. Formulation and solid-state IP can delay generic approval even when core drug-substance patents expire or are weak, because ANDA challengers must prove bioequivalence with a product that may still fall within claim scope.

What formulation IP categories are usually used for binimetinib products?

Common categories in binimetinib-like small molecule oncology products include:

• Polymorph/crystal form claims
• Particle size and morphology claims
• Solid dispersion or matrix-based stabilization claims
• Excipients and coating compositions that support stability and dissolution
• Manufacturing and drying processes that preserve critical quality attributes

How do formulation barriers affect launch timing?

Formulation claims affect:

• Ability to use alternative excipients or particle engineering
• Whether generic developers must design around claim elements
• Time and cost of patent clearing

Which patent claims most likely cover binimetinib’s combination use with encorafenib?

Key commercial point. In combination oncology, the litigation hotspot often shifts from drug substance to:

• Combination method-of-use
• Dosing schedules (timing, titration, continuous administration)
• Patient stratification logic

What method-of-use patents can block non-infringing designs?

Method claims can block:

• Attempts to reposition indications
• Attempts to use different dosing frequencies if claims include regimen parameters
• Use in specific patient subgroups if claims cover biomarker-defined populations

What patent litigation affects binimetinib and its combination with encorafenib?

Executive answer. Litigation risk tracks the appearance of ANDA filers challenging the Orange Book-listed patents for the combination regimen. In the absence of identified, active Paragraph IV matters tied directly to binimetinib’s Orange Book patents, the forward-looking risk is driven by:

• Patent expiration calendar
• Presence of late-expiring formulation and method-of-use claims
• Strength of enforcement history for the encorafenib/binimetinib patent family

How does binimetinib compare with other MEK inhibitors in market share potential?

Competitive frame. The MEK inhibitor market is crowded, but encorafenib/binimetinib competes with targeted and immunotherapy-based pathways in melanoma. The main differentiator is whether MEK inhibition is delivered within a regimen that best balances response durability and tolerability.

What are the closest competitive substitutes?

Substitutes typically include:

• Other RAF/MEK combinations (different RAF partner + MEK partner)
• Immunotherapy-first sequencing (PD-1 ± CTLA-4)
• Newer targeted strategies that adjust RAF pathway control or resistance targeting

How does binimetinib’s value proposition usually translate into buying decisions?

Oncology adoption typically favors:

• Clear biomarker selection rules
• Manageable safety monitoring
• Evidence-backed sequencing and durability
• Simple regimen logistics relative to comparator combinations

Market analysis for binimetinib: demand drivers, pricing pressure, and uptake scenarios

What drives binimetinib demand?

Demand is mainly driven by:

• Number of patients with BRAFV600-mutant metastatic melanoma
• Testing rates and guideline adherence
• Preference for targeted regimens where rapid response is prioritized
• Real-world treatment continuation impacted by MEK-class tolerability

What creates pricing and utilization pressure?

• Market access negotiations and payer preferencing
• Competition from other targeted combinations and immunotherapy sequencing
• Consolidation by large oncology networks that standardize formularies

How does clinical expansion affect revenue?

Revenue impact comes from any trial outcome that:

• Moves regimen into earlier lines
• Expands eligible biomarker-defined subgroups
• Improves response durability enough to influence sequencing

Revenue projection framework for binimetinib (scenario-based)

Executive answer. Without a verified current revenue base and without a published, consistent multi-year dataset for binimetinib component revenue (distinct from combination encorafenib/binimetinib), forward projections cannot be stated as hard figures. The credible approach is a scenario grid based on exclusivity timeline, label expansion, and competitive penetration.

Scenario drivers

• Exclusivity step-down events from patent and regulatory exclusivity
• ORR/PFS improvements leading to guideline changes
• Uptake changes in BRAFV600 metastatic lines as PD-1–first algorithms evolve
• Managed care restrictions due to comparative value

Commercial timing logic

• Short term (next 1-2 years): demand stability if no major label setbacks and if trial readouts support continued targeted usage.
• Mid term (2-5 years): growth depends on trial outcomes that expand eligible lines or improve sequencing penetration.
• Long term (post-exclusivity): generic risk rises if late-expiring formulation/method patents are cleared or narrow.

Key risks that can change binimetinib’s market path

Clinical risk

• Safety/tolerability that affects real-world persistence
• Failure of combination strategies to show OS or clinically meaningful PFS gains in new settings

Regulatory/IP risk

• Narrowing or invalidation of combination/method claims
• Loss of exclusivity earlier than expected through patent settlement dynamics or regulatory carve-outs

Competitive risk

• Rapid substitution by immunotherapy-based algorithms
• Superior efficacy of competing RAF/MEK combinations in similar populations

Key Takeaways

• Binimetinib’s development and commercial trajectory remain tied to encorafenib combination use in BRAFV600-mutant metastatic melanoma, with incremental trials focused on expansion and resistance biology rather than a standalone reinvention.
• Future market upside depends on label expansion that changes sequencing or earlier-line use, and on maintaining real-world adherence through MEK-class tolerability.
• Generic/biosimilar risks are mediated by Orange Book patent layers that commonly include combination method-of-use and formulation claims, which can delay entry even after earlier drug-substance expiration.
• The biggest determinant of revenue durability is whether late-expiring combination/formulation IP blocks ANDA pathways at the encorafenib/binimetinib regimen level.

FAQs

1. What MEK/RAF combination regimens compete directly with encorafenib/binimetinib in BRAFV600 metastatic melanoma?
2. How do formulation and polymorph patents typically delay generic ANDAs for oncology small molecules like binimetinib?
3. What trial endpoints best predict label expansion for binimetinib-containing regimens?
4. When do method-of-use patents for combination targeted therapy most often become the bottleneck for generic entry?
5. What real-world safety management issues most affect persistence with binimetinib?

References

1. (Not available in the provided prompt context.)