Amyloid beta Inhibitors
4 drugsAbout Amyloid beta
Amyloid beta (Aβ) is a protein fragment that forms amyloid plaques in the brain, a hallmark of Alzheimer's disease. While its precise function is still under investigation, Aβ's role in plaque formation is well-documented.
Human genetic studies provide strong validation for APP (amyloid precursor protein) as a therapeutic target in Alzheimer's disease (score 0.95). Loss-of-function variants are protective against Alzheimer's disease (score 0.87), supporting inhibition-based therapies.
Four FDA-approved drugs target amyloid beta, including KISUNLA, LEQEMBI, VIZAMYL, and LEQEMBI IQLIK. These drugs are biologics (75%) and small molecules (25%) indicated for CNS and other conditions.
Strategic Insights
ℹ️ How we calculate- White space opportunity in Preclinical Alzheimer's Disease with only 1 trials.
- phase3 represents biological uncertainty with 50% completion.
Amyloid beta Genetic Evidence Strong
APP has strong genetic support with a max score of 0.95 linking it to Alzheimer's disease.
Strong genetic support increases the probability of clinical success for APP-targeting drugs.
💡 Why inhibition?
- • Loss-of-function variants reduce disease risk (OR < 1)
- • 50% directional consistency across 2 traits
- • Strong signal in psychiatric disorder, nervous system disease, phenotype pathways
Cross-Disease Effects
Trade-off: HighDirection of Effect
50% alignedEvidence Across Diseases
20 totalGWAS and other genetic studies link APP to 34 diseases.
Inhibiting this target may be therapeutic
🔗 Colocalization Evidence 20 strong
max H4: 1.00eQTL/pQTL signals for APP 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 Amyloid beta Drugs
Three companies, GE HEALTHCARE, Eli Lilly, and EISAI INC, have approved drugs targeting amyloid beta.
High market concentration suggests significant barriers to entry for new amyloid beta drugs.
| Drug | Company | Approved | Indications |
|---|---|---|---|
| LEQEMBI IQLIK | EISAI INC | 2025 | 1 |
Amyloid beta Drug Modality Landscape
Modalities
Routes of Administration
Amyloid beta is druggable by both biologics (3) and small molecules (1), indicating broad therapeutic accessibility.
Consider developing small molecule Aβ inhibitors to differentiate from existing biologic therapies.
📈 Modality Evolution
Small molecules pioneered Amyloid beta targeting (2013), with other biologics entering more recently (2023).
Amyloid beta Clinical Trials 29 trials
Completion by Phase
| Phase | Total | Completed | Failed | Active | Completion |
|---|---|---|---|---|---|
| Phase 1 | 4 | 4 | 0 | 0 | 100% |
| Phase 2 | 11 | 4 | 0 | 7 | 100% |
| Phase 3 | 12 | 1 | 1 | 10 | 50% |
| Phase 4 | 2 | 0 | 0 | 2 | - |
Top Sponsors
By Modality
Top Conditions
Amyloid beta Drug Approval Timeline (2013 - 2025)
The first drug was approved in 2013, and the most recent in 2025, spanning 13 years.
The recent approval suggests renewed interest, but also increasing competition in the amyloid beta space.
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
- • 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 32 clinical trials targeting Amyloid beta.
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