Protein / target

Androgen receptor

Encoded byARP10275Homo sapiensSwiss-Prot

UniProt: P10275 Ensembl: ENSG00000169083 HGNC: HGNC:644 NCBI Gene: 367

Clinically validated

Therapeutic maturity

Approved medicines

Open Targets target-level

View by indication →

Clinical trials

Small-molecule tractable

Druggability

Approved Drug

Protein at a glance

Biological role

RNA polymerase II core promoter sequence-specific DNA binding

Strongest disease association

Androgen insensitivity syndrome

Via encoding gene AR · Genetic evidence · score 0.98

Therapeutic position

Established drug target

Small molecules and protein degraders

Research activity

Emerging research

20 papers · latest 2025

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 overview

Steroid hormone receptors are ligand-activated transcription factors that regulate eukaryotic gene expression and affect cellular proliferation and differentiation in target tissues.

View complete UniProt function annotation

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 · ReactomeCell proliferation & survivalGOTranscriptional regulationUniProt · GO · Reactome

View supporting evidence

Nuclear receptor signalling

·Steroid hormone receptors are ligand-activated transcription factors that regulate eukar…
·nuclear receptor activity
·HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand
·Nuclear Receptor transcription pathway

Cell proliferation & survival

·negative regulation of cell population proliferation
·positive regulation of cell population proliferation

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

View underlying pathways (6)

HSP90 chaperone cycle for steroid hormone receptors (SHR) in the presence of ligand

Nuclear Receptor transcription pathway

SUMOylation of intracellular receptors

Activated PKN1 stimulates transcription of AR (androgen receptor) regulated genes KLK2 and KLK3

Ub-specific processing proteases

RUNX2 regulates osteoblast differentiation

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.

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.

Approved medicines with mapped indications

9 medicines · 8 areas

Approved therapies targeting this protein, grouped by what they are approved to treat — the disease-first view of the medicines below. Relationships come from the canonical approved-indication graph (ChEMBL phase-4), the same source as the drug cards.

Prostatic Neoplasms5 medicines

apalutamide darolutamide enzalutamide flutamide nilutamide

Carcinoma2 medicines

bicalutamide flutamide

Prostatic Neoplasms, Castration-Resistant2 medicines

apalutamide darolutamide

Acne Vulgaris1 medicine

Clascoterone

Hypogonadism1 medicine

Testosterone

Klinefelter Syndrome1 medicine

Testosterone

Sexual Dysfunctions, Psychological1 medicine

Testosterone

Neoplasms8 medicines

abiraterone apalutamide bicalutamide darolutamide enzalutamide flutamide nilutamide Testosterone

19 medicines meet Open Targets' target-level approved-medicine definition; the 9 shown here are those on this page with a canonical approved disease indication in the graph. Approved indications from ChEMBL (phase-4), via the canonical drug→disease graph.

Drugs targeting this protein

How approved and investigational drugs engage this protein — mechanism and action type, direct vs complex targeting, and how broadly each acts across other targets. A drug-first view (the section above groups the approved ones disease-first); direct binders with few recorded targets are listed first.

enzalutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Prostatic Neoplasms, Neoplasms

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

Cyproterone Acetate

Narrow target profilePhase 3Antagonist

Androgen Receptor antagonist

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

apalutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Prostatic Neoplasms, Prostatic Neoplasms, Castration-Resistant, Neoplasms

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

Clascoterone

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Acne Vulgaris

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

Testosterone

Narrow target profileApprovedAgonist

Androgen Receptor agonist

Indicated for Hypogonadism, Klinefelter Syndrome, Sexual Dysfunctions, Psychological, Neoplasms

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

darolutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Prostatic Neoplasms, Prostatic Neoplasms, Castration-Resistant, Neoplasms

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

View all 10 targeting drugs

bicalutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Carcinoma, Neoplasms

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

nilutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Prostatic Neoplasms, Neoplasms

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

flutamide

Narrow target profileApprovedAntagonist

Androgen Receptor antagonist

Indicated for Carcinoma, Prostatic Neoplasms, Neoplasms

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

abiraterone

ApprovedAntagonist

Androgen Receptor antagonist

Indicated for Neoplasms

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

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

Translational evidence

Open Targets 26

Why this target matters therapeutically, strongest evidence first. Disease associations are gene-level (via the gene that encodes this protein) and open into the full confidence synthesis; the development universe, tractability and safety annotations are target-level, from Open Targets.

Strongest disease associations · via encoding gene AR

Gene-level evidence surfaced through the gene ARthat encodes this protein — not a direct protein–disease relationship. Ranked by Forefront's causal-directness weighting, so genetically- and clinically-evidenced diseases lead over ones that merely share the literature.

Androgen insensitivity syndrome

0.99Well supported

Genetic evidence dominant · Open Targets 0.84

Prostatic Neoplasms

0.97Well supported

Genetic evidence dominant · Open Targets 0.80

Partial androgen insensitivity syndrome

0.96Well supported

Genetic evidence dominant · Open Targets 0.82

Prostate carcinoma

0.95Well supported

Clinical evidence dominant · Open Targets 0.74

Familial prostate cancer

0.93Well supported

Genetic evidence dominant · Open Targets 0.62

View evidence synthesis (5)

Androgen insensitivity syndromeWell supported

0.99

agreement 0.87–1.00

Genetic78%Animal model13%Literature9%Genetic literaturedup

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

Prostatic NeoplasmsWell supported

0.97

agreement 0.88–1.00

Genetic42%Clinical37%Somatic mutation14%Literature7%

Open Targets aggregate 0.80 · 4 independent evidence families

Partial androgen insensitivity syndromeWell supported

0.96

agreement 0.84–1.00

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 0.86–1.00

Clinical34%Genetic33%Somatic mutation26%Literature8%

Open Targets aggregate 0.74 · 4 independent evidence families

Familial prostate cancerWell supported

0.93

agreement 0.82–1.00

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 per-type scores above show the calculation.

Show all associations

Androgen insensitivity syndrome0.84

Partial androgen insensitivity syndrome0.82

Prostatic Neoplasms0.80

Kennedy disease0.74

Prostate carcinoma0.74

Neoplasms0.68

Hypogonadism0.68

Prostate adenocarcinoma0.67

Familial prostate cancer0.62

Drug development

40 compounds recorded · 19 approved · 20 in clinical development · 1 earlier-stage

Open Targets' development universe — every compound recorded against the target at any stage, not all approved medicines. Distinct from the 10 drugs that target this protein in Forefront's canonical graph (9 with a mapped approved indication, shown above): these count different sets and are not a subset relation.

View all recorded compounds (10)

OXANDROLONEApproval

CR 1447Phase 2

GSK2849466Phase 1

NANDROLONE DECANOATEPhase 3

TESTOSTERONE UNDECANOATEApproval

GLPG0492Phase 1

HE3235Phase 2

VK5211Phase 2

GALETERONEPhase 3

LGD-2941Phase 1

Open Targets known-drugs universe. Drug name and highest clinical stage only — the disease relationship is NOT read from this slice (it carries trial-context noise); approved indications come from the canonical graph above.

Tractability

Small molecules — Strong

Approved Drug and Structure with Ligand support this modality.

Antibodies — Emerging

Feasibility evidence (go cc med conf) — no clinical-stage drug of this modality recorded.

Protein degraders — Strong

Advanced Clinical and Literature support this modality.

View underlying tractability evidence (11)

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

Raw Open Targets tractability assessment buckets, by modality.

Safety-related annotations

sleep apnoeaBowes et al. (2012)N/A, Impairment of reproductive capacityAOP-Wikiincreased body weightLynch et al. (2017)increased muscle massBowes et al. (2012)Decrease, Population growth rateAOP-WikidepressionLynch et al. (2017)deathLynch et al. (2017)decreased anxietyLynch et al. (2017)impotenceBowes et al. (2012)hot flashesLynch et al. (2017)bone painLynch et al. (2017)regulation of transcription factor activityToxCast

Terms indexed against this target in Open Targets' safety data, with their datasource. These are annotations, not causal claims: the direction of effect (whether activation or inhibition is implicated), species and evidence strength are not captured here, so an entry does not mean that modulating this target is known to cause that condition.

Clinical trials

Trials of drugs that target this protein — reached indirectly through those drugs, so a trial listed here studies the drug, not the protein. Ranked by active status, then clinical phase and recency, and spread across the targeting drugs.

A Randomized Trial of Neoadjuvant Leuprorelin, Darolutamide or Both Prior to Radical Prostatectomy for Intermediate or High-risk Prostate CancerPHASE4

ACTIVE_NOT_RECRUITING · via darolutamide · NCT06627530

Long-Term Extension Study for Participants Previously Enrolled in an Exelixis-Sponsored StudyPHASE3

NOT_YET_RECRUITING · via abiraterone · NCT07620574

Randomized Controlled Trial of Leuprolide Plus Abiraterone Acetate (AA) Versus Relugolix Plus AA for Advanced Prostate Cancer: The REVELUTION-2 TrialPHASE3

RECRUITING · via bicalutamide · NCT06650579

A PHASE 3, RANDOMIZED, OPEN-LABEL STUDY OF PF-06821497 (MEVROMETOSTAT) IN COMBINATION WITH ENZALUTAMIDE COMPARED WITH ENZALUTAMIDE OR DOCETAXEL IN PARTICIPANTS WITH METASTATIC CASTRATION RESISTANT PROSTATE CANCER PREVIOUSLY TREATED WITH ABIRATERONE ACETATE (MEVPRO-1)PHASE3

ACTIVE_NOT_RECRUITING · via enzalutamide · NCT06551324

View all trials (26)

A Randomized Phase III Clinical Trial for the Addition of Docetaxel to Androgen Receptor Pathway Inhibitors in Patients With Metastatic Castration Sensitive Prostate Cancer and Suboptimal PSA ResponsePHASE3

RECRUITING · via abiraterone · NCT06592924

A Multicenter, Randomized, Double-Blind, Vehicle-Controlled Clinical Study to Evaluate the Efficacy and Safety of Clascoterone Cream 1% in Treatment of Subjects With Facial Acne VulgarisPHASE3

RECRUITING · via Clascoterone · NCT06403501

Treatment of High-Risk Prostate Cancer Guided by Novel Diagnostic Radio- and Molecular Tracers (THUNDER): A Two-part Phase 2/ 3 TrialPHASE2/PHASE3

RECRUITING · via darolutamide · NCT06282588

A Double-blind Randomised Phase III Trial Evaluating the Efficacy of ADT +/- Darolutamide in de Novo Metastatic Prostate Cancer Patients With Vulnerable Functional Ability and Not Elected for Docetaxel or Androgen Receptor Targeted AgentsPHASE3

RECRUITING · via darolutamide · NCT04916613

Parallel Phase III Randomized Trials of Genomic-Risk Stratified Unfavorable Intermediate Risk Prostate Cancer: De-Intensification and Intensification Clinical Trial Evaluation (GUIDANCE)PHASE3

ACTIVE_NOT_RECRUITING · via bicalutamide · NCT05050084

CASPAR - A Phase III Trial of Enzalutamide and Rucaparib as a Novel Therapy in First-Line Metastatic Castration-Resistant Prostate CancerPHASE3

ACTIVE_NOT_RECRUITING · via enzalutamide · NCT04455750

Phase III Study of Local or Systemic Therapy INtensification DIrected by PET in Prostate CAncer Patients With Post-ProstaTEctomy Biochemical Recurrence (INDICATE)PHASE3

RECRUITING · via apalutamide · NCT04423211

DASL-HiCaP: Darolutamide Augments Standard Therapy for Localised Very High-Risk Cancer of the Prostate (ANZUP1801): A Randomised Phase 3 Double-blind, Placebo-controlled Trial of Adding Darolutamide to Androgen Deprivation Therapy and Definitive or Salvage Radiation in Very High Risk, Clinically Localised Prostate CancerPHASE3

ACTIVE_NOT_RECRUITING · via darolutamide · NCT04136353

Phase III Randomized Trial of Standard Systemic Therapy (SST) Versus Standard Systemic Therapy Plus Definitive Treatment (Surgery or Radiation) of the Primary Tumor in Metastatic Prostate CancerPHASE3

RECRUITING · via abiraterone · NCT03678025

STAMPEDE: Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy: A Multi-Stage Multi-Arm Randomised Controlled TrialPHASE2/PHASE3

ACTIVE_NOT_RECRUITING · via abiraterone · NCT00268476

A Multiple-Dose, Single Period, Phase II Dose Ranging Study to Examine TDS-Testosterone 5% in Adult Male SubjectsPHASE2

NOT_YET_RECRUITING · via Testosterone · NCT01894308

Assessing the Efficacy of Neoadjuvant Androgen Deprivation Therapy (ADT) Utilizing 18F-Flotufolastat PSMA PET/CT in Patients With High-Risk Localized Prostate Cancer (LHRPC)PHASE2

RECRUITING · via bicalutamide · NCT07455903

A Phase II Prospective, Open-Label Clinical Study of Darolutamide ± ADT as Neoadjuvant Therapy in High-Risk/Very High-Risk Localized Prostate CancerPHASE2

NOT_YET_RECRUITING · via darolutamide · NCT07476677

An Open-label, Multicenter Phase Ib/II Clinical Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Preliminary Anti-tumor Activity of QLC5508 and/or QLH12016 in Combination With Other Anti-tumor Therapies in Subjects With Advanced Prostate CancerPHASE1/PHASE2

RECRUITING · via enzalutamide · NCT07198633

Copenhagen Menopause Study (COMPASS): A Randomized Clinical TrialPHASE2

RECRUITING · via Testosterone · NCT07254429

MK-2400-01A Substudy: A Phase 1/2, Open-label Umbrella Substudy of MK-2400-U01 Master Protocol to Evaluate the Safety and Efficacy of Ifinatamab Deruxtecan-based Treatment Combinations or Ifinatamab Deruxtecan Alone in Participants With Metastatic Castration-resistant Prostate Cancer (mCRPC) (IDeate-Prostate02)PHASE1/PHASE2

RECRUITING · via abiraterone · NCT06863272

Presurgical Phase II Study of Talazoparib in Combination With Enzalutamide in de Novo Metastatic to Lymph Nodes Prostate CancerPHASE2

RECRUITING · via enzalutamide · NCT05873192

ASpiRE: A Proof-of-mechanism and Proof-of-concept Clinical Trial Evaluating the Safety, Tolerability, Biological and Anti-tumour Activity of Apalutamide With Dual CXCR1 and CXCR2 Blockade by SX-682 for Men Suffering From Metastatic Castration-resistant Prostate Cancer (mCRPC)PHASE1/PHASE2

RECRUITING · via apalutamide · NCT07002320

Phase 1/2 Dose-Escalation and Cohort Study of STEAP1 CART With Enzalutamide in Participants With mCRPCPHASE1/PHASE2

RECRUITING · via enzalutamide · NCT06236139

MC230502 Dynamic Investigator Initiated Enterprise (DIVINE) in Prostate CancerPHASE2

RECRUITING · via abiraterone · NCT06378866

Neoadjuvant ADT +/- Darolutamide Followed by Radical Prostatectomy for High-risk Prostate Cancer: a Randomized, Open Label TrialPHASE2

RECRUITING · via darolutamide · NCT06575257

Phase II Study of Apalutamide With Carotuximab in Metastatic, Castration-Resistant Prostate CancerPHASE2

RECRUITING · via apalutamide · NCT05534646

A Single Arm Open-label, Phase II Study of Sipuleucel-T With Bipolar Androgen Therapy in Men With Metastatic Castration-resistant Prostate CancerPHASE2

RECRUITING · via Testosterone · NCT06100705

A Randomized Phase II Trial of Docetaxel or Cabazitaxel With or Without Darolutamide in Men With Metastatic Castration-resistant Prostate CancerPHASE2

RECRUITING · via darolutamide · NCT05762536

A Phase 2 Study of Darolutamide in Combination With Leuprolide Acetate in Hormone-Therapy Naive Recurrent and/or Metastatic Androgen Receptor (AR) Positive Salivary Gland CancerPHASE2

ACTIVE_NOT_RECRUITING · via darolutamide · NCT05669664

ANZadapt: Phase II Randomised Controlled Trial of Patient-specific Adaptive Versus Continuous Abiraterone or eNZalutamide in Metastatic Castration-resistant Prostate CancerPHASE2

RECRUITING · via enzalutamide · NCT05393791

ClinicalTrials.gov via the drug-target graph.

What's happening now

Recent therapeutic activity around this target — regulatory actions, clinical trials and safety signals for 5 drugs that target this protein, plus publications where such a drug is a genuine subject. Every item is reached indirectly through the drug, not the protein itself; incidental mentions (a paper that merely measures a drug) and press items are excluded.

Indication expanded2026-07-24
Indication expansion: ENZALUTAMIDE (NDA203415)
fda · regulatory · fda · via enzalutamide
Indication expanded2026-07-24
Indication expansion: ENZALUTAMIDE (NDA213674)
fda · regulatory · fda · via enzalutamide
Label change2026-06-29
Label change: APALUTAMIDE (NDA210951)
fda · regulatory · fda · via apalutamide
Label change2026-03-05
Label change: APALUTAMIDE (NDA210951)
fda · regulatory · fda · via apalutamide
Label change2025-12-11
Label change: ENZALUTAMIDE (NDA203415)
fda · regulatory · fda · via enzalutamide
Label change2025-12-11
Label change: ENZALUTAMIDE (NDA213674)
fda · regulatory · fda · via enzalutamide
Regulatory approval2025-10-17
Approval: Winlevi (EMA)
ema · regulatory · ema · via Clascoterone
New publication2025-06-03
Neurology of Androgens and Androgenic Supplements.
Current neurology and neuroscience reports · 2025 · 2 citations · Europe PMC · via Testosterone
New publication2025-05-30
Enzalutamide plus radium-223 in metastatic castration-resistant prostate cancer: results of the EORTC 1333/PEACE-3 trial.
Annals of oncology : official journal of the European Society for Medical Oncology · 2025 · 23 citations · Europe PMC · via enzalutamide
New publication2025-05-13
Society for Endocrinology Clinical Practice Guideline for the Evaluation of Androgen Excess in Women.
Clinical endocrinology · 2025 · 2 citations · Europe PMC · via Testosterone
New publication2025-02-15
Comparative efficacy of pharmacological interventions on metabolic and hormonal outcomes in polycystic ovary syndrome: a Network Meta-Analysis of Randomized controlled trials.
BMC women's health · 2025 · 4 citations · Europe PMC · via Testosterone
Safety communication2020-06-29
Drug Safety Update: Cyproterone acetate: new advice to minimise risk of meningioma
mhra · safety · mhra · via Cyproterone Acetate

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.