CSF1R Inhibitors
6 drugsAbout CSF1R
CSF1R, or Colony Stimulating Factor 1 Receptor, is a tyrosine kinase receptor crucial for the survival, proliferation, and differentiation of myeloid lineage cells like macrophages and osteoclasts. It regulates immune cell function and bone remodeling.
Human genetics provide strong validation for CSF1R as a target, with a max score of 0.93 linking loss-of-function variants to hereditary diffuse leukoencephalopathy. Activation of CSF1R is likely beneficial based on genetic evidence.
Six FDA-approved drugs target CSF1R, including AYVAKIT, STIVARGA, OFEV, ROMVIMZA and VONJO, all of which are small molecules. These drugs are used in respiratory diseases and other therapeutic areas.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Stage IIIC Colorectal Cancer AJCC v8 with only 4 trials.
CSF1R Genetic Evidence Strong
CSF1R has strong genetic support with a max score of 0.93 linking it to hereditary diffuse leukoencephalopathy.
The strong genetic support suggests that clinical trials targeting CSF1R have a higher 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, nervous system disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
7 totalGWAS and other genetic studies link CSF1R to 7 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 CSF1R 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 CSF1R Drugs
Six companies have approved drugs targeting CSF1R, including DECIPHERA PHARMS, SOBI, and Bayer.
The presence of multiple established players suggests a moderately competitive market with potential entry barriers.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| VONJO | SOBI | 2022 | 1 |
| ROMVIMZA | DECIPHERA PHARMS | 2025 | 1 |
| TURALIO | DAIICHI SANKYO INC | 2019 | 1 |
CSF1R Drug Modality Landscape
Modalities
Routes of Administration
CSF1R is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like antibodies or fusion proteins could provide a competitive advantage.
CSF1R Clinical Trials 384 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 136 | 88 | 18 | 29 | 83% |
| Phase 2 | 181 | 62 | 38 | 80 | 62% |
| Phase 3 | 51 | 26 | 10 | 15 | 72% |
| Phase 4 | 16 | 12 | 2 | 2 | 86% |
Top Sponsors
By Modality
Top Conditions
Phase 3 Readout Calendar Pro
4 Phase 3 trials testing approved CSF1R drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting CSF1R. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
CSF1R Drug Approval Timeline (2012 - 2025)
The first CSF1R-targeting drug was approved in 2012, with the most recent approval in 2025.
The approval timeline indicates continued interest and development in targeting CSF1R, but also suggests increasing market saturation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 6 companies competing
- • Market share by company
Full Drug Portfolio
- • All 6 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 6-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 6 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 387 clinical trials targeting CSF1R.
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