KIT Inhibitors
17 drugsAbout KIT
KIT (KIT Proto-Oncogene Receptor Tyrosine Kinase) is a receptor tyrosine kinase on the cell surface involved in cell signaling pathways that regulate cell growth, differentiation, and survival. Activated KIT triggers intracellular events that can lead to uncontrolled cell proliferation.
Human genetics provide strong validation for KIT as a therapeutic target (max score 0.92), with variants linked to cutaneous mastocytosis, piebaldism and gastrointestinal stromal tumor. Loss-of-function variants are associated with increased risk for cutaneous mastocytosis and piebaldism, while also protecting against gastrointestinal stromal tumor, suggesting context-dependent therapeutic strategies.
KIT is targeted by 16 FDA-approved small molecule drugs including AYVAKIT, LENVIMA and CABOMETYX. While most approved drugs are used in other therapeutic areas, 4 are used in oncology, highlighting KIT's relevance in cancer treatment.
Strategic Insights
ℹ️ How we calculate- Validated target with strong trial activity and 81% attractiveness score.
KIT Genetic Evidence Strong
KIT has strong genetic support with a maximum score of 0.92 across 18 diseases.
Strong genetic support suggests a higher likelihood of clinical success, potentially justifying investment in novel therapies.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 67% directional consistency across 3 traits
- • Strong signal in hematologic disease, immune system disease, integumentary system disease pathways
Cross-Disease Effects
Trade-off: ModerateDirection of Effect
67% alignedEvidence Across Diseases
18 totalGWAS and other genetic studies link KIT to 18 diseases.
Loss-of-function causes disease; activation may help
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for KIT 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 KIT Drugs
Fourteen companies have approved drugs targeting KIT, with Viatris, DECIPHERA PHARMS and NOVUGEN among the top players.
The competitive landscape suggests moderate market fragmentation, potentially lowering entry barriers for new companies.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| CABOMETYX | EXELIXIS | 2016 | 5 |
| SUTENT | CPPI CV | 2006 | 4 |
| RYDAPT | Novartis | 2017 | 4 |
| SUNITINIB MALATE | MSN | 2021 | 3 |
| SORAFENIB TOSYLATE | Dr. Reddy's | 2020 | 3 |
| STIVARGA | Bayer | 2012 | 3 |
| NEXAVAR | Bayer | 2005 | 3 |
| PAZOPANIB HYDROCHLORIDE | Apotex | 2023 | 2 |
| ICLUSIG | Takeda | 2012 | 2 |
| VOTRIENT | Novartis | 2009 | 2 |
| QINLOCK | DECIPHERA PHARMS | 2020 | 1 |
| COMETRIQ | EXELIXIS | 2012 | 1 |
| TURALIO | DAIICHI SANKYO INC | 2019 | 1 |
| RESNIBEN | AZURITY | - | 1 |
KIT Drug Modality Landscape
Modalities
Routes of Administration
KIT is amenable to small molecule drugs, with oral options available for convenient dosing.
The absence of other modalities like antibodies or fusion proteins represents a whitespace opportunity for innovation.
KIT Clinical Trials 1,585 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 476 | 288 | 86 | 99 | 77% |
| Phase 2 | 892 | 384 | 185 | 317 | 67% |
| Phase 3 | 183 | 89 | 24 | 70 | 79% |
| Phase 4 | 34 | 20 | 3 | 11 | 87% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
8 Phase 3 trials testing approved KIT drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting KIT. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
KIT Drug Approval Timeline (2005 - 2023)
The first KIT-targeting drug was approved in 2005, with the most recent approval in 2023, spanning 19 years.
The continued approval of new drugs indicates sustained interest and potential for further development in this target space.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 14 companies competing
- • Market share by company
Full Drug Portfolio
- • All 17 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 17-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • 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 1000 clinical trials targeting KIT.
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