Androgen Receptor Modulators
7 drugsAbout Androgen Receptor
The Androgen Receptor (AR) is a steroid hormone receptor crucial for male development and maintenance. As a ligand-activated transcription factor, it binds androgens like testosterone, regulating gene expression. Its pivotal role makes AR a significant drug target, especially in oncology.
Human genetic studies provide strong validation for AR as a therapeutic target (score 0.98), with variants linked to androgen insensitivity syndrome (score 0.98) and familial prostate cancer (score 0.89). Loss-of-function variants increase disease risk, supporting activation-based therapies.
AR is targeted by 7 FDA-approved small molecule drugs, including XTANDI, ERLEADA and NUBEQA, across oncology and other therapeutic areas. Approvals span 31 years, from CASODEX (1995) to APALUTAMIDE (2025).
Androgen Receptor Genetic Evidence Strong
Strong genetic evidence supports AR's role in multiple diseases, with a maximum score of 0.98.
High genetic support suggests that clinical trials targeting AR have an increased likelihood of success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 3 traits
- • Strong signal in genetic, familial or congenital disease, endocrine system disease, reproductive system or breast disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link AR to 26 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 3 strong
max H4: 0.97eQTL/pQTL signals for AR colocalize with these GWAS traits, providing causal evidence that gene expression changes drive disease risk.
Understanding these scores
Association Score (0-1): Combines all evidence types (genetic, literature, drugs, animal models). Higher = more evidence linking target to disease. This is a ranking heuristic, not a confidence score.
L2G Score: Open Targets uses a machine learning model (Locus-to-Gene) to predict which gene is causal at each GWAS locus. L2G=0.5 means ~50% probability of being the causal gene. Only associations with L2G > 0.05 are included.
Odds Ratio (OR): From gene burden studies (UK Biobank, AstraZeneca PheWAS). Measures how loss-of-function variants affect disease risk. OR<1 = protective (inhibiting target may help), OR>1 = risk (losing function causes disease).
Beta (β): Effect size for continuous traits. β<0 = protective, β>0 = risk.
Clinical Translation (~1.8x): Based on Nelson et al. 2015: drug targets with genetic evidence have ~2x higher success rates in clinical trials. We estimate: Strong support (score ≥0.7) → ~1.8x, Moderate (0.3-0.7) → ~1.3x, Weak → baseline.
Colocalization (H4): Tests whether a GWAS signal and an eQTL/pQTL signal share the same causal variant. H4 is the posterior probability that both traits are associated AND share a causal variant. H4 > 0.8 = strong evidence that gene expression/protein levels drive disease risk. This links genetic variation → gene expression → disease, supporting the target-disease connection.
Top Androgen Receptor Drugs
Seven companies have approved AR-targeting drugs, including ASTELLAS, Bayer and ADVANZ PHARMA.
The presence of multiple players indicates a competitive market, requiring strong differentiation for new entrants.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| APALUTAMIDE | ZYDUS LIFESCIENCES | 2025 | 2 |
| NILANDRON | ADVANZ PHARMA | 1996 | 1 |
| WINLEVI | Sun Pharma | 2020 | 1 |
| NUBEQA | Bayer | 2019 | 1 |
Androgen Receptor Drug Modality Landscape
Modalities
Routes of Administration
Androgen Receptor is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like antibodies or PROTACs could provide a competitive advantage.
Androgen Receptor Clinical Trials 531 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Androgen Receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 146 | 53 | 35 | 58 | 60% |
| Phase 2 | 262 | 111 | 41 | 110 | 73% |
| Phase 3 | 100 | 22 | 15 | 63 | 59% |
| Phase 4 | 23 | 13 | 2 | 8 | 87% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
8 Phase 3 trials testing approved Androgen Receptor drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting Androgen Receptor. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
Androgen Receptor Drug Approval Timeline (1995 - 2025)
AR drug approvals span 31 years, with the most recent approval in 2025.
Continued approvals suggest ongoing interest and potential for further innovation in AR-targeted therapies.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 7 companies competing
- • Market share by company
Full Drug Portfolio
- • All 7 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 7-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 238 (72 companies)
- • Completion rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Count Trials
- Trials: phase 1-4 interventional trials on ClinicalTrials.gov that started in 2008 or later and test an FDA-approved drug acting on Androgen Receptor. A trial counts if an approved drug acting on Androgen Receptor is in any arm, including as a comparator, and a trial that tests two such drugs counts once. Trials that test only unapproved drugs are not counted. A trial listed as phase 1/2 counts as phase 1, and phase 2/3 as phase 2.
- Active: recruiting, not yet recruiting, enrolling by invitation, or active and no longer recruiting.
- Completion rate: of the trials that have ended, the share marked Completed rather than Terminated or Withdrawn. Completed means the trial ended as planned; it does not mean the drug worked. Suspended trials are not counted as ended.
- Stopped: terminated or withdrawn. Trials stop for many reasons, including slow enrolment and business decisions, not only safety or efficacy.
- Sponsors: the lead sponsor of each trial. Companies are the sponsors ClinicalTrials.gov classes as industry; the rest are universities, hospitals, cooperative groups, government bodies and individuals.