Protein / target

Dual specificity mitogen-activated protein kinase kinase 1

MAP2K1Q02750Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
8
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Mitogen-activated protein kinase kinase kinase binding

Primary system

Nervous system

Strongest disease association

RASopathy

Genetic evidence · score 0.94

Therapeutic maturity

Clinically validated target

8 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

8 approved · 11 in clinical development

30 linked trials

Derived from structured UniProt, Open Targets and literature data on this page.

Protein profile

UniProt 2026_02

Canonical identity and biological annotation from UniProt.

Function

Dual specificity protein kinase which acts as an essential component of the MAP kinase signal transduction pathway. Binding of extracellular ligands such as growth factors, cytokines and hormones to their cell-surface receptors activates RAS and this initiates RAF1 activation. RAF1 then further activates the dual-specificity protein kinases MAP2K1/MEK1 and MAP2K2/MEK2. Both MAP2K1/MEK1 and MAP2K2/MEK2 function specifically in the MAPK/ERK cascade, and catalyze the concomitant phosphorylation of a threonine and a tyrosine residue in a Thr-Glu-Tyr sequence located in the extracellular signal-regulated kinases MAPK3/ERK1 and MAPK1/ERK2, leading to their activation and further transduction of the signal within the MAPK/ERK cascade. Activates BRAF in a KSR1 or KSR2-dependent manner; by binding to KSR1 or KSR2 releases the inhibitory intramolecular interaction between KSR1 or KSR2 protein kinase and N-terminal domains which promotes KSR1 or KSR2-BRAF dimerization and BRAF activation (PubMed:29433126). Depending on the cellular context, this pathway mediates diverse biological functions such as cell growth, adhesion, survival and differentiation, predominantly through the regulation of transcription, metabolism and cytoskeletal rearrangements. One target of the MAPK/ERK cascade is peroxisome proliferator-activated receptor gamma (PPARG), a nuclear receptor that promotes differentiation and apoptosis. MAP2K1/MEK1 has been shown to export PPARG from the nucleus. The MAPK/ERK cascade is also involved in the regulation of endosomal dynamics, including lysosome processing and endosome cycling through the perinuclear recycling compartment (PNRC), as well as in the fragmentation of the Golgi apparatus during mitosis

Subcellular location

Cytoplasm, cytoskeleton, microtubule organizing center, centrosomeCytoplasm, cytoskeleton, microtubule organizing center, spindle pole bodyCytoplasmNucleusMembrane
Domains and Gene Ontology detail (71)

Domains & features

Protein kinase

Gene Ontology

  • Caxon
  • Ccell cortex
  • Ccentrosome
  • Cciliary basal body
  • Ccytosol
  • Cdendrite cytoplasm
  • Cearly endosome
  • Cendoplasmic reticulum
  • Cfocal adhesion
  • Cglutamatergic synapse
  • CGolgi apparatus
  • Clate endosome

393 aa · 43 kDa · 2 isoforms

Biological roles

Reactome v97

What this protein does, drawn together from its UniProt function, Gene Ontology terms and Reactome pathways.

Kinase signallingUniProt · GO · ReactomeTranscriptional regulationUniProt · GOSynaptic signallingGOMuscle contractionGOExcitatory neurotransmissionGONuclear receptor signallingUniProt
View supporting evidence

Kinase signalling

  • ·Dual specificity protein kinase which acts as an essential component of the MAP kinase s…
  • ·MAP kinase kinase activity
  • ·mitogen-activated protein kinase kinase kinase binding
  • ·protein kinase activator activity

Transcriptional regulation

  • ·Dual specificity protein kinase which acts as an essential component of the MAP kinase s…
  • ·negative regulation of gene expression
  • ·positive regulation of DNA-templated transcription
  • ·positive regulation of gene expression

Synaptic signalling

  • ·glutamatergic synapse
  • ·postsynaptic density
  • ·regulation of neurotransmitter receptor localization to postsynaptic specialization memb…

Muscle contraction

  • ·positive regulation of muscle contraction
  • ·regulation of vascular associated smooth muscle contraction

Excitatory neurotransmission

  • ·glutamatergic synapse

Nuclear receptor signalling

  • ·Dual specificity protein kinase which acts as an essential component of the MAP kinase s…
View underlying pathways (15)

Concepts derived from UniProt GO Reactome — each badge above shows which sources supported that role.

Interaction neighbourhood

STRING v12.0

Proteins with the strongest functional or physical association. Node size and line weight reflect STRING confidence; hover or select a partner to inspect one association.

MAPK3BRAFMAPK1RAF1LAMTOR3KSR2KSR1MAP2K2KRASARAFMAP2K1

10 strongest partners — larger node and heavier line mean higher confidence

Functional and physical associations from STRING v12.0. Only associations with this protein are drawn — partner-to-partner links are not part of this evidence.

Drugs targeting this protein

2

Whether each drug engages this protein directly or through a complex, and how many other targets are recorded for it. Direct binders with few recorded targets are listed first.

binimetinib
Narrow target profileApprovedInhibitor

Dual specificity mitogen-activated protein kinase kinase 1 inhibitor

Appears in clinical studies involving melanoma, neoplasm, metastatic melanoma, metastatic colorectal cancer

Direct interaction with this protein · 1 of 2 recorded protein targets — narrow recorded profile

trametinib
ApprovedInhibitor

Dual specificity mitogen-activated protein kinase kinase; MEK1/2 inhibitor

Appears in clinical studies involving metastatic melanoma, melanoma, glioma, neoplasm

Acts on a complex — shared with MAP2K2 · 1 of 2 recorded protein targets — narrow recorded profile

ChEMBL mechanism, action type and target identity. Open Targets clinical status and indications.

Translational evidence

Open Targets 26

Why this target matters therapeutically — disease associations, drugs in development, tractability and safety, from Open Targets.

Strongest genetic associations

Human genetic evidence — the most direct causal link between this target and a disease.

RASopathy0.94

Genetic · overall 0.60

cardiofaciocutaneous syndrome 30.91

Genetic · overall 0.81

cardiofaciocutaneous syndrome0.88

Genetic literature · overall 0.83

melorheostosis0.85

Genetic · overall 0.72

Noonan syndrome0.85

Genetic · overall 0.67

Highest-confidence therapeutic associations

Diseases where a drug acting on this target has already reached clinical development.

melanoma0.97

Clinical · overall 0.73

neurofibromatosis type 10.93

Clinical · overall 0.57

neoplasm0.90

Clinical · overall 0.60

cancer0.42

Clinical · overall 0.69

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

Noonan syndrome with multiple lentigines0.55

Genetic literature

Show all associations
cardiofaciocutaneous syndrome0.83
cardiofaciocutaneous syndrome 30.81
melanoma0.73
melorheostosis0.72
cancer0.69
Noonan syndrome0.67
neoplasm0.60
RASopathy0.60
neurofibromatosis type 10.57
Noonan syndrome with multiple lentigines0.55

Open Targets ranks 1,320 associations for this target and we store the top 10 by aggregated score. The long tail is largely literature co-mention and RNA-expression evidence, not evidence that this target drives those diseases.

Known drugs · 19 total

MIRDAMETINIBApproval

neurofibromatosis type 1 · plexiform neurofibroma · neoplasm

REFAMETINIBPhase 3

hepatocellular carcinoma · colorectal cancer · malignant pancreatic neoplasm

TRAMETINIB DIMETHYL SULFOXIDEApproval

lung cancer · melanoma · low grade glioma

PIMASERTIBPhase 2

colorectal cancer · acute myeloid leukemia · hematopoietic and lymphoid cell neoplasm

CI-1040Phase 2

non-small cell lung carcinoma · malignant colon neoplasm · breast cancer

E-6201Phase 2

psoriasis vulgaris · acute myeloid leukemia · chronic myelomonocytic leukemia

TAK-733Phase 2

Familial adenomatous polyposis · melanoma

BINIMETINIBApproval

melanoma · neoplasm · metastatic melanoma

COBIMETINIBApproval

melanoma · neoplasm · colorectal adenocarcinoma

AZD-8330Phase 1

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · UniProt loc high confAB · GO CC high confAB · Human Protein Atlas locPR · LiteraturePR · Database UbiquitinationPR · Half-life DataPR · Small Molecule Binder

Clinical trials

30

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein.

Show remaining trials (24)

ClinicalTrials.gov via the drug-target graph.

Related literature

1

Papers indexed under “MAP Kinase Kinase 1” — a subject heading that covers this protein without being specific to it. Shown as context; not counted as this protein's own research activity.

Improved survival with MEK inhibition in BRAF-mutated melanoma.

Flaherty KT · The New England journal of medicine · 2012

Europe PMC literature, reached through a MeSH descriptor linked to this protein.

Forefront confidence

Synthesis

Our own weighting of the evidence behind each disease association. Human genetics and clinical evidence count for most; literature co-mention counts for little, because two entities sharing an abstract is not evidence that one drives the other.

RASopathyWell supported
0.95
agreement 0.811.00
Genetic90%Literature11%

Open Targets aggregate 0.60 · 2 independent evidence families

melorheostosisWell supported
0.93
agreement 0.821.00
Genetic60%Somatic mutation34%Literature6%Genetic literaturedup

Open Targets aggregate 0.72 · 3 independent evidence families · 1 not counted as duplicate

Noonan syndromeWell supported
0.92
agreement 0.831.00
Genetic60%Pathway25%Animal model8%Clinical7%Literature1%Genetic literaturedup

Open Targets aggregate 0.67 · 5 independent evidence families · 1 not counted as duplicate

cardiofaciocutaneous syndrome 3Well supported
0.91
agreement 0.771.00
Genetic99%Literature1%Genetic literaturedup

Open Targets aggregate 0.81 · 2 independent evidence families · 1 not counted as duplicate

cardiofaciocutaneous syndromeWell supported
0.90
agreement 0.781.00
Genetic70%Pathway25%Literature5%Genetic literaturedup

Open Targets aggregate 0.83 · 3 independent evidence families · 1 not counted as duplicate

This ranking differs from Open Targets' own: re-weighting moves genetically-evidenced diseases above more heavily co-mentioned ones. The evidence agreement range shows how closely the independent evidence families agree — it is not a statistical confidence interval, and nothing here is fitted to outcome data. Derived from Open Targets evidence types under Forefront weighting; the underlying per-type scores are shown above so the calculation can be checked.

What's happening now

13

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 2 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. Trial status changed2026-07-22

    Phase I Study of the Pan-ERBB Inhibitor Neratinib Given in Combination With Everolimus, Palbociclib, or Trametinib in Advanced Cancer Subjects With EGFR Mutation/Amplification, HER2 Mutation/Amplification, or HER3/4 Mutation or KRAS Mutation

    Status changed to Terminated · ClinicalTrials.gov · via trametinib

  2. New publication2024-09-23
    Molecular profiling of BRAF-V600E-mutant metastatic colorectal cancer in the phase 3 BEACON CRC trial.

    Nature medicine · 2024 · 52 citations · Europe PMC · via binimetinib

  3. New publication2024-09-09
    Efficacy and safety of the combination of encorafenib/cetuximab with or without binimetinib in patients with BRAF V600E-mutated metastatic colorectal cancer: an AGEO real-world multicenter study.

    ESMO open · 2024 · 12 citations · Europe PMC · via binimetinib

  4. Regulatory approval2024-01-05

    Approval: Spexotras (EMA)

    ema · regulatory · ema · via trametinib

  5. New publication2022-09-27
    Combination Dabrafenib and Trametinib Versus Combination Nivolumab and Ipilimumab for Patients With Advanced <i>BRAF</i>-Mutant Melanoma: The DREAMseq Trial-ECOG-ACRIN EA6134.

    Journal of clinical oncology : official journal of the American Society of Clinical Oncology · 2023 · 302 citations · Europe PMC · via trametinib

  6. New publication2019-09-30
    Encorafenib, Binimetinib, and Cetuximab in <i>BRAF</i> V600E-Mutated Colorectal Cancer.

    The New England journal of medicine · 2019 · 1,029 citations · Europe PMC · via binimetinib

  7. Regulatory approval2018-09-20

    Approval: Mektovi (EMA)

    ema · regulatory · ema · via binimetinib

  8. New publication2018-03-21
    Encorafenib plus binimetinib versus vemurafenib or encorafenib in patients with BRAF-mutant melanoma (COLUMBUS): a multicentre, open-label, randomised phase 3 trial.

    The Lancet. Oncology · 2018 · 756 citations · Europe PMC · via binimetinib

  9. Safety communication2016-03-10

    Drug Safety Update: Trametinib (Mekinist▼): risk of gastrointestinal perforation and colitis

    mhra · safety · mhra · via trametinib

  10. New publication2014-11-16
    Improved overall survival in melanoma with combined dabrafenib and trametinib.

    The New England journal of medicine · 2015 · 1,914 citations · Europe PMC · via trametinib

  11. New publication2014-09-29
    Combined BRAF and MEK inhibition versus BRAF inhibition alone in melanoma.

    The New England journal of medicine · 2014 · 1,440 citations · Europe PMC · via trametinib

  12. Regulatory approval2014-06-30

    Approval: Mekinist (EMA)

    ema · regulatory · ema · via trametinib

Objective event titles are shown unmodified; the event kind and significance line are derived from structured fields. Forefront AttentionEvent stream aggregating Europe PMC Regulatory filings ClinicalTrials.gov.