VDR Inhibitors
3 drugsAbout VDR
The Vitamin D Receptor (VDR) is a nuclear receptor that regulates gene expression in response to vitamin D. As a ligand-activated transcription factor, VDR interacts with RXR and binds DNA, influencing genes involved in diverse biological processes.
VDR is a validated drug target with strong genetic support (max score 0.92) linking it to diseases like hypocalcemic vitamin D-resistant rickets (score 0.92), hypertension (0.75), and cardiovascular disease (0.57). Loss-of-function variants increase risk of rickets, suggesting activation may be beneficial.
Three FDA-approved small molecule drugs, including DOXERCALCIFEROL (CAPLIN) and PARICALCITOL (CHARTWELL RX, ZEMPLAR), target VDR for various therapeutic areas. The first drug was approved in 1998, with the most recent in 2011.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Endometrial Cancer with only 1 trials.
Human Genetic Evidence Strong
VDR has strong genetic support with a maximum score of 0.92 linking it to multiple diseases.
Strong genetic validation suggests VDR agonists have a higher probability of clinical success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 1 traits
- • Strong signal in endocrine system disease, genetic, familial or congenital disease, nutritional or metabolic disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
18 totalGWAS and other genetic studies link VDR to 18 diseases.
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 8 strong
max H4: 1.00eQTL/pQTL signals for VDR 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 Drugs
Three companies, including CHARTWELL RX, CAPLIN, and AbbVie, have approved VDR-targeting drugs.
The limited number of players suggests a market with moderate barriers to entry.
Clinical Trials 76 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 18 | 13 | 3 | 2 | 81% |
| Phase 2 | 19 | 17 | 1 | 1 | 94% |
| Phase 3 | 17 | 11 | 5 | 1 | 69% |
| Phase 4 | 22 | 15 | 7 | 0 | 68% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
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Deep insights for drug target analysis
Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 3 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 3-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 10 underexplored indications
- • Success rates by condition
Full summary • All drugs • Genetic evidence • Trials • Timeline
How We Calculate These Metrics
Target Attractiveness Score
A 0-100 score based on trial activity, sponsor diversity, and completion rates. Calculated from 23 clinical trials targeting VDR.
Completion rate: Percentage of trials that reached their planned endpoint. Trials terminated early, withdrawn, or suspended are not counted—these often indicate safety issues, lack of efficacy, or strategic pivots.
- Highly Attractive (80+): High trial activity, many sponsors, strong completion rates
- Attractive (60-79): Good trial activity and validation
- Moderate (40-59): Moderate interest from sponsors
- Low (under 40): Limited trial activity or validation concerns
Strategic Insights
Auto-generated insights based on trial analytics including competition intensity, white space opportunities, modality shifts, and failure patterns. We analyze trial sponsors, phases, indications, and outcomes.
Risk Signals
- High Competition: Many sponsors competing for this target (may reduce market opportunity)
- High Failure Risk: Low trial completion rates suggest development challenges
- Low Validation: Limited trial activity or poor outcomes indicate uncertain viability
- White Space Available: Underexplored indications present opportunities