PDCD1 Inhibitors
11 drugsAbout PDCD1
PDCD1 (programmed cell death protein 1), also known as PD-1 or CD279, is an immune checkpoint protein that regulates T cell activity and prevents excessive immune responses. Cancer cells often exploit this mechanism to evade immune detection. As a result, PDCD1 has become a significant focus in oncology drug development.
Human genetics provide moderate support for PDCD1 as a therapeutic target, with a max genetic score of 0.58 linking it to atopic eczema, allergic rhinitis and asthma. Loss-of-function variants are associated with increased risk of atopic eczema, suggesting activation of PDCD1 may be beneficial.
PDCD1 is targeted by 11 FDA-approved drugs, including KEYTRUDA, OPDIVO, and LIBTAYO, primarily in oncology. These drugs are biologics (other, 6 drugs) or antibodies (5 drugs). Eight companies have approved PDCD1-targeting drugs, including Merck and Bristol-Myers Squibb.
PDCD1 Genetic Evidence Moderate
Genetic evidence provides moderate support for PDCD1, with a max score of 0.58 for atopic eczema.
Further research into PDCD1 activation may reveal novel therapies for allergic and immune-related diseases.
💡 Why activation?
- • Loss-of-function variants increase disease risk (OR > 1) — restoring function may help
- • 100% directional consistency across 1 traits
- • Strong signal in integumentary system disease, immune system disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
5 totalGWAS and other genetic studies link PDCD1 to 5 diseases.
Loss-of-function causes disease; activation may help
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for PDCD1 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 PDCD1 Drugs
Eight companies have approved PDCD1-targeting drugs, with Merck and Bristol-Myers Squibb leading the market.
The presence of established players suggests high barriers to entry, requiring strong differentiation.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| TEVIMBRA | BEIGENE | 2024 | 4 |
| OPDIVO QVANTIG | Bristol-Myers Squibb | 2024 | 3 |
| JEMPERLI | GSK | 2021 | 2 |
| ZYNYZ | INCYTE CORP | 2023 | 2 |
| OPDUALAG | Bristol-Myers Squibb | 2022 | 2 |
| KEYTRUDA QLEX | Merck | 2025 | 2 |
| LOQTORZI | COHERUS BIOSCIENCES INC | 2023 | 1 |
| PENPULIMAB-KCQX | AKESO BIOPHARMA | 2025 | 1 |
PDCD1 Drug Modality Landscape
Modalities
Routes of Administration
PDCD1 requires biologic approaches (biologic (other)), likely due to its structure or location.
Consider novel modalities to differentiate from existing antibody and biologic-based therapies.
📈 Modality Evolution
Antibodies pioneered PDCD1 targeting (2014), with other biologics entering more recently (2018).
PDCD1 Clinical Trials 4,308 trials
Phase 1-4 trials started in 2008 or later that test an FDA-approved drug acting on PDCD1. Source: ClinicalTrials.gov. How we count
Completion by Phase
| Phase | Total | Completed | Stopped | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 1625 | 575 | 380 | 654 | 60% |
| Phase 2 | 2156 | 605 | 377 | 1161 | 62% |
| Phase 3 | 498 | 152 | 50 | 295 | 75% |
| Phase 4 | 29 | 10 | 2 | 17 | 83% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
8 Phase 3 trials testing approved PDCD1 drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting PDCD1. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
PDCD1 Drug Approval Timeline (2014 - 2025)
The first PDCD1-targeting drug was approved in 2014 (KEYTRUDA), with the most recent in 2025 (PENPULIMAB-KCQX).
The continued approval of new drugs indicates ongoing interest and potential for further innovation in this space.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 8 companies competing
- • Market share by company
Full Drug Portfolio
- • All 11 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 11-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Sponsors: 1,184 (490 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 PDCD1. A trial counts if an approved drug acting on PDCD1 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.