VEGFR Inhibitors
2 drugsAbout VEGFR
Vascular Endothelial Growth Factor Receptor (VEGFR) is crucial for angiogenesis, the formation of new blood vessels, playing a significant role in development and disease.
Human genetic studies provide strong validation for VEGFR (KDR) as a therapeutic target (max score 0.92), with variants linked to endometriosis and female infertility. Loss-of-function variants are protective, suggesting inhibition is likely beneficial.
VEGFR is targeted by 2 approved small molecule drugs: CAPRELSA (Sanofi) and ICLUSIG (Takeda), primarily in 'other' therapeutic areas.
VEGFR Genetic Evidence Strong
Strong genetic evidence supports VEGFR as a drug target with a max score of 0.92.
Strong genetic support increases clinical success probability, especially for endometriosis indications.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 100% directional consistency across 1 traits
- • Strong signal in reproductive system or breast disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
16 totalGWAS and other genetic studies link KDR to 16 diseases.
Inhibiting this target may be therapeutic
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for KDR 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 VEGFR Drugs
Sanofi and Takeda are the only companies with approved VEGFR-targeting drugs.
The market is not highly concentrated, suggesting opportunities for new entrants with differentiated VEGFR inhibitors.
VEGFR Drug Modality Landscape
Modalities
Routes of Administration
VEGFR is amenable to small molecule drugs, with oral options available for convenient dosing.
Explore non-small molecule modalities like antibodies or fusion proteins to differentiate from existing therapies.
VEGFR Clinical Trials 95 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on VEGFR. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 31 | 16 | 13 | 2 | 55% |
| Phase 2 | 56 | 26 | 16 | 14 | 62% |
| Phase 3 | 7 | 2 | 2 | 3 | 50% |
| Phase 4 | 1 | 1 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
VEGFR Drug Approval Timeline (2011 - 2012)
The first VEGFR-targeting drug was approved in 2011, with the most recent in 2012.
The limited recent activity suggests potential for novel VEGFR-targeting therapies to address unmet needs.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 2 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 49 (8 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 VEGFR. A trial counts if an approved drug acting on VEGFR 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.