FLT-3 Inhibitors
7 drugsAbout FLT-3
FLT3 (Fms-like tyrosine kinase 3) is a receptor tyrosine kinase crucial for the proliferation, differentiation, and survival of hematopoietic stem cells. As a key regulator of hematopoiesis, FLT3 signaling impacts immune cell development and function.
Human genetic studies provide strong validation for FLT3 as a therapeutic target (max score 0.85), with loss-of-function variants linked to skeletal system abnormalities (0.85), hypothyroidism (0.77), and rheumatoid arthritis (0.66). Inhibition of FLT3 is likely beneficial based on genetic evidence.
FLT3 is targeted by 7 FDA-approved small molecule drugs including CABOMETYX, SORAFENIB TOSYLATE and ALUNBRIG. These drugs span oncology (2 drugs), respiratory (1 drug) and other therapeutic areas (4 drugs).
Strategic Insights
ℹ️ How we calculate- White space opportunity in Sarcomatoid Renal Cell Carcinoma with only 5 trials.
- phase2 represents biological uncertainty with 59% completion.
Human Genetic Evidence Strong
FLT3 has strong genetic support with a maximum score of 0.85 across 16 diseases.
Strong genetic support suggests a higher probability of clinical success for FLT3-targeting drugs.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 100% directional consistency across 1 traits
- • Strong signal in immune system disease, musculoskeletal or connective tissue disease, phenotype pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
16 totalGWAS and other genetic studies link FLT3 to 16 diseases.
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for FLT3 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
Seven companies have approved FLT3-targeting drugs, including Viatris, Takeda and EXELIXIS.
The presence of multiple players indicates a competitive market with moderate entry barriers.
Drug Modality Landscape
Modalities
Routes of Administration
FLT-3 is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities represents a whitespace opportunity for novel therapeutic approaches.
Clinical Trials 744 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 267 | 174 | 47 | 45 | 79% |
| Phase 2 | 348 | 166 | 83 | 98 | 67% |
| Phase 3 | 106 | 55 | 18 | 33 | 75% |
| Phase 4 | 23 | 14 | 3 | 6 | 82% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
3 Phase 3 trials testing approved FLT-3 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting FLT-3. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
Drug Approval Timeline (2005 - 2020)
The first FLT3-targeting drug was approved in 2005, with the most recent in 2020.
The approval timeline suggests a mature target with potential for incremental innovation.
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
- • Competition: High (15 sponsors)
- • White space: 3 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 393 clinical trials targeting FLT-3.
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