Plasmin Inhibitors
3 drugsAbout Plasmin
Plasmin (PLG) is a serine protease essential for fibrinolysis, the breakdown of blood clots. It's encoded by the PLG gene and plays a key role in wound healing, inflammation, and angiogenesis.
Human genetic studies strongly support plasmin as a therapeutic target, with variants linked to hypoplasminogenemia (score 0.90), hereditary angioedema (0.87), and coronary artery disease (0.81). Loss-of-function variants are associated with increased risk of hypoplasminogenemia and hereditary angioedema, suggesting activation may be beneficial.
Plasmin is targeted by 3 FDA-approved small molecule drugs: DEFITELIO, TRANEXAMIC ACID, and LYSTEDA. These drugs are marketed by EUGIA PHARMA, JAZZ PHARMS INC, and AMRING PHARMS for rare diseases and other indications.
Plasmin Genetic Evidence Strong
Genetic evidence strongly supports plasmin as a drug target, with a maximum genetic score of 0.90 across 47 diseases.
Strong genetic support suggests that clinical trials targeting plasmin have a higher likelihood of success.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 67% directional consistency across 3 traits
- • Strong signal in genetic, familial or congenital disease, hematologic disease, integumentary system disease pathways
Cross-Disease Effects
Trade-off: ModerateDirection of Effect
67% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link PLG to 47 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 PLG 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 Plasmin Drugs
Three companies, EUGIA PHARMA, JAZZ PHARMS INC, and AMRING PHARMS, have approved drugs targeting plasmin.
The limited number of companies suggests relatively low competitive intensity and potential for new market entrants.
Plasmin Drug Modality Landscape
Modalities
Routes of Administration
Plasmin is amenable to small molecule drugs, with oral options available for convenient dosing.
The exclusive use of small molecules indicates a whitespace opportunity for alternative modalities like antibodies or peptides.
Plasmin Clinical Trials 380 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on Plasmin. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 23 | 16 | 1 | 6 | 94% |
| Phase 2 | 81 | 47 | 15 | 18 | 76% |
| Phase 3 | 99 | 51 | 17 | 30 | 75% |
| Phase 4 | 177 | 111 | 28 | 38 | 80% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Plasmin Drug Approval Timeline (2009 - 2016)
The first drug targeting plasmin was approved in 2009 (LYSTEDA), with the most recent approval in 2016 (DEFITELIO).
The relatively short approval span of 8 years suggests a potentially unsaturated market with room for further innovation.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 3 companies competing
- • Market share by company
Full Drug Portfolio
- • All 3 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 3-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 255 (14 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 Plasmin. A trial counts if an approved drug acting on Plasmin 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.