Erythropoietin Receptor Inhibitors
4 drugsAbout Erythropoietin Receptor
The Erythropoietin Receptor (EpoR) is activated by erythropoietin (EPO) to stimulate red blood cell production. Activation triggers intracellular signaling pathways, leading to the proliferation and differentiation of erythroid progenitor cells in the bone marrow.
Human genetics provide moderate support for EPOR as a therapeutic target (max score 0.61). Variants are linked to primary familial polycythemia due to EPO receptor mutation, suggesting activation may be beneficial.
Four FDA-approved drugs (ARANESP, EPOGEN/PROCRIT, MIRCERA, RETACRIT) target EpoR, all of which are biologics. These drugs are used across infectious disease and other therapeutic areas.
Erythropoietin Receptor Genetic Evidence Moderate
Genetic evidence provides moderate support for EPOR with a max score of 0.61.
Further investigation of activating EPOR variants may reveal novel therapeutic strategies.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 1 traits
- • Strong signal in genetic, familial or congenital disease, immune system disease, hematologic disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
4 totalGWAS and other genetic studies link EPOR to 4 diseases.
Loss-of-function causes disease; activation may help
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 Erythropoietin Receptor Drugs
Three companies (Amgen, HOFFMAN-LA ROCHE, and Pfizer) have approved drugs targeting EPOR.
The concentrated market suggests high barriers to entry for new competitors.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| MIRCERA | HOFFMAN-LA ROCHE | 2007 | 2 |
Erythropoietin Receptor Drug Modality Landscape
Modalities
Routes of Administration
Erythropoietin Receptor requires biologic approaches (enzyme), likely due to its structure or location.
Exploring non-biologic modalities like small molecules could offer a competitive advantage.
Erythropoietin Receptor Clinical Trials 196 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Erythropoietin Receptor. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 19 | 15 | 2 | 2 | 88% |
| Phase 2 | 45 | 29 | 11 | 5 | 73% |
| Phase 3 | 93 | 74 | 9 | 10 | 89% |
| Phase 4 | 39 | 33 | 5 | 1 | 87% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
4 Phase 3 trials testing approved Erythropoietin Receptor drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting Erythropoietin Receptor. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
Erythropoietin Receptor Drug Approval Timeline (1989 - 2018)
The first drug was approved in 1989, and the most recent in 2018, spanning 30 years.
The approval timeline suggests a mature market with potential for incremental innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 4 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 4-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 98 (41 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 Erythropoietin Receptor. A trial counts if an approved drug acting on Erythropoietin Receptor 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.