Protein / target

Androgen receptor

ARP10275Homo sapiensSwiss-Prot
Clinically validated
Therapeutic maturity
19
Approved medicines
30
Clinical trials
Small-molecule tractable
Druggability
Approved Drug

Protein at a glance

Biological role

RNA polymerase II core promoter sequence-specific DNA binding

Primary system

Endocrine & metabolic

Strongest disease association

androgen insensitivity syndrome

Genetic evidence · score 0.98

Therapeutic maturity

Clinically validated target

19 approved medicines against this target

Druggability

Small molecule

Open Targets tractability · Approved Drug

Clinical development

19 approved · 20 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

Steroid hormone receptors are ligand-activated transcription factors that regulate eukaryotic gene expression and affect cellular proliferation and differentiation in target tissues (PubMed:19022849). Transcription factor activity is modulated by bound coactivator and corepressor proteins like ZBTB7A that recruits NCOR1 and NCOR2 to the androgen response elements/ARE on target genes, negatively regulating androgen receptor signaling and androgen-induced cell proliferation (PubMed:20812024). Transcription activation is also down-regulated by NR0B2. Activated, but not phosphorylated, by HIPK3 and ZIPK/DAPK3

Subcellular location

NucleusCytoplasm
Domains and Gene Ontology detail (57)

Domains & features

NR LBD

Gene Ontology

  • Cchromatin
  • Ccytoplasm
  • Ccytosol
  • Cnuclear speck
  • Cnucleoplasm
  • Cnucleus
  • Cplasma membrane
  • Cprotein-containing complex
  • Fandrogen binding
  • FATPase binding
  • Fbeta-catenin binding
  • Fchromatin binding

920 aa · 99 kDa · 4 isoforms

Biological roles

Reactome v97

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

Nuclear receptor signallingUniProt · GO · ReactomeTranscriptional regulationUniProt · GO · ReactomeImmune signallingUniProtProteolysisReactome
View supporting evidence

Nuclear receptor signalling

  • ·Steroid hormone receptors are ligand-activated transcription factors that regulate eukar…
  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor activity, RNA polymerase II-specific
  • ·nuclear receptor activity

Transcriptional regulation

  • ·Steroid hormone receptors are ligand-activated transcription factors that regulate eukar…
  • ·DNA-binding transcription activator activity, RNA polymerase II-specific
  • ·DNA-binding transcription factor activity
  • ·DNA-binding transcription factor activity, RNA polymerase II-specific

Immune signalling

  • ·Steroid hormone receptors are ligand-activated transcription factors that regulate eukar…

Proteolysis

  • ·Ub-specific processing proteases
View underlying pathways (6)

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.

NCOA2NCOA4HSP90A…HSP90A…MDM2NCOA3KDM1AFOXA1CTNNB1KLK3AR

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

7

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.

enzalutamide
Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Appears in clinical studies involving prostate cancer, prostate carcinoma, prostate neoplasm, neoplasm

Direct interaction with this protein · Only this protein recorded as a target

Cyproterone Acetate
Narrow target profilePhase 3Antagonist

Androgen Receptor antagonist

Appears in clinical studies involving prostate cancer, Hot flashes, prostate adenocarcinoma, endometriosis

Direct interaction with this protein · 1 of 3 recorded protein targets — narrow recorded profile

apalutamide
Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Appears in clinical studies involving prostate cancer, acute myeloid leukemia by FAB classification, prostate neoplasm, neoplasm

Direct interaction with this protein · Only this protein recorded as a target

Clascoterone
Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Appears in clinical studies involving acne, acne, androgenetic alopecia

Direct interaction with this protein · Only this protein recorded as a target

Testosterone
Narrow target profileApprovedAgonist

Androgen Receptor agonist

Appears in clinical studies involving hypogonadism, hypogonadotropic hypogonadism, orchitis, Klinefelter syndrome

Direct interaction with this protein · Only this protein recorded as a target

darolutamide
Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Appears in clinical studies involving prostate cancer, neoplasm, breast cancer, prostate cancer

Direct interaction with this protein · Only this protein recorded as a target

abiraterone
ApprovedAntagonist

Androgen Receptor antagonist

Appears in clinical studies involving prostate cancer, prostate neoplasm, neoplasm, cancer

Direct interaction with this protein · 1 of 5 recorded protein targets

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.

androgen insensitivity syndrome0.98

Genetic · overall 0.84

partial androgen insensitivity syndrome0.94

Genetic · overall 0.82

Familial prostate cancer0.89

Genetic · overall 0.62

Kennedy disease0.86

Genetic · overall 0.74

prostate cancer0.84

Genetic · overall 0.80

Highest-confidence therapeutic associations

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

hypogonadism0.99

Clinical · overall 0.68

hypogonadotropic hypogonadism0.98

Clinical · overall 0.62

neoplasm0.96

Clinical · overall 0.68

prostate carcinoma0.89

Clinical · overall 0.74

prostate adenocarcinoma0.68

Clinical · overall 0.67

Show all associations
androgen insensitivity syndrome0.84
partial androgen insensitivity syndrome0.82
prostate cancer0.80
Kennedy disease0.74
prostate carcinoma0.74
neoplasm0.68
hypogonadism0.68
prostate adenocarcinoma0.67
Familial prostate cancer0.62
hypogonadotropic hypogonadism0.62

Open Targets ranks 2,938 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 · 40 total

OXANDROLONEApproval

alcoholic hepatitis · Turner syndrome · Weight loss

CR 1447Phase 2

breast cancer

GSK2849466Phase 1

heart failure

NANDROLONE DECANOATEPhase 3

aplastic anemia · hip fracture · sarcopenia

TESTOSTERONE UNDECANOATEApproval

hypogonadism · orchitis · neoplasm

GLPG0492Phase 1

Cachexia

HE3235Phase 2

breast cancer · prostate cancer

VK5211Phase 2

hip fracture · musculoskeletal system disorder

GALETERONEPhase 3

prostate cancer · pancreatic adenocarcinoma

LGD-2941Phase 1

osteoporosis

Tractability

SM · Approved DrugSM · Structure with LigandSM · High-Quality LigandSM · High-Quality PocketSM · Druggable FamilyAB · GO CC med confPR · Advanced ClinicalPR · LiteraturePR · UniProt UbiquitinationPR · Database UbiquitinationPR · Small Molecule Binder

Safety liabilities

sleep apnoeaN/A, Impairment of reproductive capacityincreased body weightincreased muscle massDecrease, Population growth ratedepressiondeathdecreased anxietyimpotencehot flashesbone painregulation of transcription factor activity

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)

RECRUITING · via Testosterone · NCT04460872

UNKNOWN · via Cyproterone Acetate · NCT00363285

COMPLETED · via Testosterone · NCT00260143

ClinicalTrials.gov via the drug-target graph.

Related literature

20

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

Framework for the Pathology Workup of Metastatic Castration-Resistant Prostate Cancer Biopsies.

Haffner MC · Clinical cancer research : an official journal of the American Association for Cancer Research · 2025

Neurology of Androgens and Androgenic Supplements.

Dengri C · Current neurology and neuroscience reports · 2025

RISING STARS: Androgens and immune cell function.

Ainslie RJ · The Journal of endocrinology · 2024

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.

androgen insensitivity syndromeWell supported
0.99
agreement 0.871.00
Genetic78%Animal model13%Literature9%Genetic literaturedup

Open Targets aggregate 0.84 · 3 independent evidence families · 1 not counted as duplicate

prostate cancerWell supported
0.97
agreement 0.881.00
Genetic42%Clinical37%Somatic mutation14%Literature7%

Open Targets aggregate 0.80 · 4 independent evidence families

partial androgen insensitivity syndromeWell supported
0.96
agreement 0.841.00
Genetic75%Animal model19%Literature6%Genetic literaturedup

Open Targets aggregate 0.82 · 3 independent evidence families · 1 not counted as duplicate

prostate carcinomaWell supported
0.95
agreement 0.861.00
Clinical34%Genetic33%Somatic mutation26%Literature8%

Open Targets aggregate 0.74 · 4 independent evidence families

Familial prostate cancerWell supported
0.93
agreement 0.821.00
Genetic68%Somatic mutation21%Literature11%

Open Targets aggregate 0.62 · 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

60

Recent activity around this target, drawn from one canonical event stream. Every item is reached through 6 drugs that target this protein, so each event is news about that drug rather than about the protein directly.

  1. Indication expanded2026-07-24

    Indication expansion: ENZALUTAMIDE (NDA213674)

    fda · regulatory · fda · via enzalutamide

  2. Indication expanded2026-07-24

    Indication expansion: ENZALUTAMIDE (NDA203415)

    fda · regulatory · fda · via enzalutamide

  3. Industry development2026-07-21
    Opinion: Pete Hegseth, ‘The Odyssey,’ and the testosterone myth

    STAT News · news · via Testosterone

  4. Industry development2026-07-18
    Opinion: Beware the unintended consequences of testosterone screening for military servicemembers

    STAT News · news · via Testosterone

  5. Industry development2026-07-17
    Who can access testosterone hormone medication in the U.S.?

    STAT News · news · via Testosterone

  6. Industry development2026-07-16
    Pete Hegseth’s announcement of annual testosterone screenings for service members divides medical experts

    STAT News · news · via Testosterone

  7. Label change2026-06-29

    Label change: APALUTAMIDE (NDA210951)

    fda · regulatory · fda · via apalutamide

  8. Industry development2026-06-19
    Briefing Chat: Testosterone and sperm might get a boost from obesity drugs

    Nature News · news · via Testosterone

  9. Industry development2026-06-11
    ‘Not simply saving cost’: Inside Astellas CEO’s 5-year strategy to counter Xtandi’s patent cliff

    Fierce Biotech · news · via enzalutamide

  10. Industry development2026-06-11
    ‘Not simply saving cost’: Inside Astellas CEO’s 5-year strategy to counter Xtandi’s patent cliff

    Fierce Pharma · news · via enzalutamide

  11. Label change2026-03-05

    Label change: APALUTAMIDE (NDA210951)

    fda · regulatory · fda · via apalutamide

  12. Label change2025-12-11

    Label change: ENZALUTAMIDE (NDA213674)

    fda · regulatory · fda · via enzalutamide

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.