5-HT2A Inhibitors
2 drugsAbout 5-HT2A
The 5-HT2A receptor (serotonin 2A receptor) is a G protein-coupled receptor that mediates the effects of serotonin in the central nervous system. It plays a crucial role in neuronal signaling and various physiological processes.
While there is currently no genetic evidence directly linking 5-HT2A to specific diseases, its involvement in neuronal signaling makes it a compelling target for therapeutic intervention.
Two FDA-approved small molecule drugs target 5-HT2A: CAPLYTA (INTRA-CELLULAR) for CNS disorders and TONMYA (TONIX) for other therapeutic areas. These approvals highlight the potential of 5-HT2A modulation.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Schizophrenia or Schizoaffective with only 1 trials.
Human Genetic Evidence Moderate
There is no genetic evidence data available for the 5-HT2A target.
Lack of genetic support suggests higher risk in drug development; consider alternative targets with stronger genetic validation.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 100% directional consistency across 1 traits
- • Strong signal in psychiatric disorder, nervous system disease pathways
Cross-Disease Effects
Trade-off: LowDirection of Effect
100% alignedEvidence Across Diseases
16 totalGWAS and other genetic studies link HTR2A to 16 diseases.
Inhibiting this target may be therapeutic
🔗 Colocalization Evidence 1 strong
max H4: 0.98eQTL/pQTL signals for HTR2A 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 Drugs
The competitive landscape includes two companies with approved drugs: INTRA-CELLULAR and TONIX.
Low market concentration suggests an opportunity for new entrants, but also highlights potential challenges in establishing market share.
Drug Modality Landscape
Modalities
Routes of Administration
5-HT2A is amenable to small molecule drugs, with oral options available for convenient dosing.
Explore opportunities for novel modalities like antibodies or biologics to differentiate from existing small molecule drugs.
Clinical Trials 57 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 18 | 14 | 0 | 4 | 100% |
| Phase 2 | 9 | 6 | 2 | 1 | 75% |
| Phase 3 | 24 | 12 | 4 | 8 | 75% |
| Phase 4 | 6 | 2 | 2 | 2 | 50% |
Top Sponsors
By Modality
Top Conditions
Drug Approval Timeline (1996 - 2025)
The first drug, CAPLYTA, was approved in 2019, and the most recent, TONMYA, in 2025, spanning 7 years.
Recent approval indicates continued interest in this target, but further approvals may saturate the market.
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Deep insights for drug target analysis
Competitive Landscape
- • 2 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 42 clinical trials targeting 5-HT2A.
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