PIK3CA Inhibitors
2 drugsAbout PIK3CA
PIK3CA, also known as PI3Kα, is a lipid kinase enzyme crucial in the PI3K/AKT/mTOR pathway, regulating cell growth, proliferation, survival, and metabolism. Dysregulation of this pathway is common across diseases.
Human genetics strongly support PIK3CA as a drug target (max score 0.96), with variants linked to conditions like megalencephaly and breast cancer (score 0.92). Loss-of-function variants are protective in breast cancer, suggesting inhibition is beneficial.
Two FDA-approved small molecule drugs, VIJOICE and PIQRAY, target PIK3CA, both developed by Novartis. Applications span oncology and other therapeutic areas.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Locally Advanced Malignant Solid Neoplasm with only 2 trials.
- phase2 represents biological uncertainty with 30% completion.
PIK3CA Genetic Evidence Strong
PIK3CA has strong genetic support with a maximum score of 0.96 across 31 diseases.
The strong genetic support suggests a higher probability of clinical trial success for PIK3CA-targeting drugs.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 100% directional consistency across 1 traits
- • Strong signal in integumentary system disease, reproductive system or breast disease, cancer or benign tumor pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link PIK3CA to 31 diseases.
Inhibiting this target may be therapeutic
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for PIK3CA 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 PIK3CA Drugs
Novartis is the only company with approved PIK3CA-targeting drugs.
The market is highly concentrated, presenting a barrier to entry for new companies.
PIK3CA Drug Modality Landscape
Modalities
Routes of Administration
PIK3CA is amenable to small molecule drugs, with oral options available for convenient dosing.
Exploring alternative modalities like antibodies or PROTACs could provide a competitive advantage.
PIK3CA Clinical Trials 59 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 18 | 7 | 3 | 8 | 70% |
| Phase 2 | 31 | 4 | 7 | 20 | 36% |
| Phase 3 | 9 | 2 | 2 | 5 | 50% |
| Phase 4 | 1 | 1 | 0 | 0 | 100% |
Top Sponsors
By Modality
Top Conditions
Top Drugs
Phase 3 Readout Calendar Pro
3 Phase 3 trials testing approved PIK3CA drugs across all sponsors.
Coverage: trials whose intervention is an approved drug targeting PIK3CA. Pre-approval candidates with development codes (e.g. AZD0901, MK-7240) are not yet linked. Anchored on CT.gov primary completion date.
PIK3CA Drug Approval Timeline (2019 - 2022)
The first PIK3CA-targeting drug was approved in 2019, with the most recent in 2022.
The recent approval suggests continued interest, but saturation may occur soon.
Pro Intelligence Preview
Deep insights for drug target analysis
Competitive Landscape
- • 1 companies competing
- • Market share by company
Full Drug Portfolio
- • All 2 approved drugs
- • Approval dates & indications
Genetic Validation
- • Full genetic evidence table
- • Effect sizes & directions
Approval Timeline
- • Full 2-drug timeline
- • First-of-modality markers
Clinical Trials Analysis
- • Competition: High (15 sponsors)
- • White space: 10 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 62 clinical trials targeting PIK3CA.
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