Protein / target

BDNF/NT-3 growth factors receptor

NTRK2Q16620Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
6
Approved medicines
22
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

Brain-derived neurotrophic factor receptor activity

Primary system

Nervous system

Strongest disease association

obesity, hyperphagia, and developmental delay

Genetic evidence · score 0.83

Therapeutic maturity

Clinically validated target

6 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

6 approved · 7 in clinical development

22 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

Receptor tyrosine kinase involved in the development and the maturation of the central and the peripheral nervous systems through regulation of neuron survival, proliferation, migration, differentiation, and synapse formation and plasticity (By similarity). Receptor for BDNF/brain-derived neurotrophic factor and NTF4/neurotrophin-4. Alternatively can also bind NTF3/neurotrophin-3 which is less efficient in activating the receptor but regulates neuron survival through NTRK2 (PubMed:15494731, PubMed:7574684). Upon ligand-binding, undergoes homodimerization, autophosphorylation and activation (PubMed:15494731). Recruits, phosphorylates and/or activates several downstream effectors including SHC1, FRS2, SH2B1, SH2B2 and PLCG1 that regulate distinct overlapping signaling cascades. Through SHC1, FRS2, SH2B1, SH2B2 activates the GRB2-Ras-MAPK cascade that regulates for instance neuronal differentiation including neurite outgrowth. Through the same effectors controls the Ras-PI3 kinase-AKT1 signaling cascade that mainly regulates growth and survival. Through PLCG1 and the downstream protein kinase C-regulated pathways controls synaptic plasticity. Thereby, plays a role in learning and memory by regulating both short term synaptic function and long-term potentiation. PLCG1 also leads to NF-Kappa-B activation and the transcription of genes involved in cell survival. Hence, it is able to suppress anoikis, the apoptosis resulting from loss of cell-matrix interactions. May also play a role in neutrophin-dependent calcium signaling in glial cells and mediate communication between neurons and glia

Subcellular location

Cell membraneEndosome membraneEarly endosome membraneCell projection, axonCell projection, dendriteCytoplasm, perinuclear regionPostsynaptic density
Domains and Gene Ontology detail (47)

Domains & features

LRRNTLRRCTIg-like C2-type 1Ig-like C2-type 2Protein kinase

Gene Ontology

  • Caxon
  • Caxon terminus
  • Ccytosol
  • Cdendrite
  • Cdendritic spine
  • Cearly endosome
  • Cearly endosome membrane
  • Cperinuclear region of cytoplasm
  • Cplasma membrane
  • Cpostsynaptic density
  • Creceptor complex
  • Cterminal bouton

822 aa · 92 kDa · 7 isoforms

Biological roles

Reactome v97

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

Synaptic signallingUniProt · GOKinase signallingUniProt · GOTranscriptional regulationUniProt · GOProteolysisGOApoptosis & cell deathGO
View supporting evidence

Synaptic signalling

  • ·Receptor tyrosine kinase involved in the development and the maturation of the central a…
  • ·Postsynaptic density
  • ·postsynaptic density
  • ·trans-synaptic signaling by BDNF, modulating synaptic transmission

Kinase signalling

  • ·Receptor tyrosine kinase involved in the development and the maturation of the central a…
  • ·positive regulation of phosphatidylinositol 3-kinase/protein kinase B signal transduction
  • ·protein autophosphorylation

Transcriptional regulation

  • ·Receptor tyrosine kinase involved in the development and the maturation of the central a…
  • ·positive regulation of gene expression

Proteolysis

  • ·protease binding

Apoptosis & cell death

  • ·negative regulation of neuron apoptotic process
View underlying pathways (14)

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.

NTF3NTF4SHC1NGFBDNFGDNFSHC3PLCG1FRS2NGFRNTRK2

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.

repotrectinib
ApprovedInhibitor

Neurotrophic tyrosine kinase receptor type 2 inhibitor

Appears in clinical studies involving non-small cell lung carcinoma, non-small cell lung carcinoma, non-small cell lung carcinoma, cancer

Direct interaction with this protein · 1 of 6 recorded protein targets

larotrectinib
ApprovedInhibitor

Neurotrophic tyrosine kinase receptor inhibitor

Appears in clinical studies involving neoplasm, cancer, plasma cell myeloma, lymphoma

Acts on a complex — shared with NTRK1, NTRK3 · 1 of 3 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.

obesity, hyperphagia, and developmental delay0.83

Genetic · overall 0.71

genetic developmental and epileptic encephalopathy0.80

Genetic · overall 0.61

undetermined early-onset epileptic encephalopathy0.61

Genetic · overall 0.38

infantile spasms0.61

Genetic · overall 0.38

neoplasm0.19

Genetic · overall 0.63

Highest-confidence therapeutic associations

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

non-small cell lung carcinoma0.95

Clinical · overall 0.65

cancer0.87

Clinical · overall 0.63

keratitis0.76

Clinical · overall 0.46

pancreatic ductal adenocarcinoma0.06

Clinical · overall 0.38

Highest overall evidence

Remaining associations by Open Targets' aggregated evidence score.

neurodegenerative disease0.50

Pathway

Show all associations
obesity, hyperphagia, and developmental delay0.71
non-small cell lung carcinoma0.65
neoplasm0.63
cancer0.63
genetic developmental and epileptic encephalopathy0.61
neurodegenerative disease0.50
keratitis0.46
undetermined early-onset epileptic encephalopathy0.38
infantile spasms0.38
pancreatic ductal adenocarcinoma0.38

Open Targets ranks 2,159 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 · 13 total

LESTAURTINIBApproval

acute myeloid leukemia by FAB classification · myeloid leukemia · acute lymphoblastic leukemia

LAROTRECTINIB SULFATEApproval

metastasis · cancer · neoplasm

CEP-2563Phase 1
LAROTRECTINIBApproval

neoplasm · cancer · plasma cell myeloma

CENEGERMINApproval

keratitis · eye disorder · Sensory neuropathy

ENTRECTINIBApproval

non-small cell lung carcinoma · non-small cell lung carcinoma · neoplasm

REPOTRECTINIBApproval

non-small cell lung carcinoma · non-small cell lung carcinoma · non-small cell lung carcinoma

ALTIRATINIBPhase 1
AZD-6918Phase 1
TALETRECTINIBPhase 3

non-small cell lung carcinoma · non-small cell lung carcinoma · neoplasm

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · Druggable FamilyAB · UniProt loc high confAB · GO CC high confAB · UniProt loc med confAB · UniProt SigP or TMHMMAB · Human Protein Atlas locPR · LiteraturePR · UniProt UbiquitinationPR · Half-life Data

Safety liabilities

suicidal thoughts

Clinical trials

22

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 (16)

ClinicalTrials.gov via the drug-target graph.

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.

obesity, hyperphagia, and developmental delayWell supported
0.85
agreement 0.730.97
Genetic88%Animal model11%Literature1%Genetic literaturedup

Open Targets aggregate 0.71 · 3 independent evidence families · 1 not counted as duplicate

neoplasmWell supported
0.84
agreement 0.750.93
Clinical51%Somatic mutation23%Genetic15%Literature11%

Open Targets aggregate 0.63 · 4 independent evidence families

genetic developmental and epileptic encephalopathyWell supported
0.83
agreement 0.710.95
Genetic81%Animal model19%Literature0%Genetic literaturedup

Open Targets aggregate 0.61 · 3 independent evidence families · 1 not counted as duplicate

non-small cell lung carcinomaWell supported
0.82
agreement 0.710.93
Clinical64%Somatic mutation27%Literature9%RNA expression1%

Open Targets aggregate 0.65 · 4 independent evidence families

cancerWell supported
0.76
agreement 0.640.89
Clinical65%Pathway24%Literature11%

Open Targets aggregate 0.63 · 3 independent evidence families

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

3

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. Regulatory approval2025-01-13

    Approval: Augtyro (EMA)

    ema · regulatory · ema · via repotrectinib

  2. New publication2024-01-01
    Repotrectinib in <i>ROS1</i> Fusion-Positive Non-Small-Cell Lung Cancer.

    The New England journal of medicine · 2024 · 168 citations · Europe PMC · via repotrectinib

  3. New publication2018-02-01
    Efficacy of Larotrectinib in TRK Fusion-Positive Cancers in Adults and Children.

    The New England journal of medicine · 2018 · 1,987 citations · Europe PMC · via larotrectinib

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.