Avoralstat
drug drugOn this page
Also known as BCX-4161Bcx4161
Summary
Avoralstat (CHEMBL4297502) is a phase-3 clinical-stage small molecule targeting KLKB1; indicated across 1 condition including hereditary angioedema.
At a glance
- Status: Max clinical phase 3 (not approved)
- Modality: Small molecule
- Targets: 1 (KLKB1)
- Indications: 1 condition
- Clinical trials: 4
- Chemistry: 513.5 Da · C28H27N5O5
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL4297502 |
| Name | Avoralstat |
| Type | Small molecule |
| Max phase | 3 |
| FDA approved | no |
| PubChem CID | 86566678 |
| Molecular formula | C28H27N5O5 |
| Molecular weight | 513.5 |
| InChIKey | TUWMKPVJGGWGNL-UHFFFAOYSA-N |
SMILES: COC1=CC(=C(C=C1C=C)C(=O)NC2=CC=C(C=C2)C(=N)N)C3=C(N=C(C=C3)C(=O)NCC4CC4)C(=O)O
IUPAC name: 3-[2-[(4-carbamimidoylphenyl)carbamoyl]-4-ethenyl-5-methoxyphenyl]-6-(cyclopropylmethylcarbamoyl)pyridine-2-carboxylic acid
Also known as: Avoralstat, BCX-4161, Bcx4161, AVORALSTAT
Patent coverage: 34 distinct patent families (91 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 90 (99%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.
Targets
Targets
Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).
| Gene | Target | Action | pAffinity | Cancer dependency | UniProt |
|---|---|---|---|---|---|
| KLKB1 | kallikrein B1 | Inhibition | 8.7 | 0% | P03952 |
Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Plasma kallikrein.
Bioactivity
ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| KLKB1 | 9.59 | Ki | 0.26 | nM | CHEMBL_ACT_23312843 |
Target pathways
Aggregated over 1 target gene(s): KLKB1.
Top Reactome pathways
14 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Hemostasis | 1 | KLKB1 |
| R-HSA-140837 | 1 | KLKB1 |
| R-HSA-140877 | 1 | KLKB1 |
| Degradation of the extracellular matrix | 1 | KLKB1 |
| Extracellular matrix organization | 1 | KLKB1 |
| Activation of Matrix Metalloproteinases | 1 | KLKB1 |
| Disease | 1 | KLKB1 |
| R-HSA-9651496 | 1 | KLKB1 |
| Defective factor XII causes hereditary angioedema | 1 | KLKB1 |
| Defective SERPING1 causes hereditary angioedema | 1 | KLKB1 |
| Diseases of hemostasis | 1 | KLKB1 |
| Regulation of FXIIa and plasma kallikrein activity | 1 | KLKB1 |
| FXIIa, PKa-dependent activation of coagulation pathway | 1 | KLKB1 |
| FXIIa activates plasma kallikrein-kinin system | 1 | KLKB1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| Factor XII activation | 1 |
| proteolysis | 1 |
| blood coagulation | 1 |
| zymogen activation | 1 |
| plasminogen activation | 1 |
| fibrinolysis | 1 |
| positive regulation of fibrinolysis | 1 |
| inflammatory response | 1 |
| hemostasis | 1 |
Indications & clinical
Indications
1 disease in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.
| Disease (in trials) | Phase | MONDO | EFO |
|---|---|---|---|
| hereditary angioedema | 3 | MONDO:0019623 | MONDO:0019623 |
Clinical trials
Total trials: 4.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE1 | 2 |
| PHASE2/PHASE3 | 1 |
| PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT02303626 | PHASE2/PHASE3 | COMPLETED | 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks |
| NCT01984788 | PHASE2 | COMPLETED | Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE |
| NCT02125162 | PHASE1 | COMPLETED | A Study of the Relative Bioavailability of a New Formulation of BCX4161 and the Effect of Food on BCX4161 |
| NCT02218294 | PHASE1 | COMPLETED | Study to Determine How BCX4161 is Metabolized and Eliminated by the Body |
Clinical evidence (CIViC)
No CIViC predictive evidence (expected for non-precision-medicine drugs).
Pharmacology
Pharmacogenomics
No PharmGKB pharmacogenomic data curated for this drug.
Related molecules
Related molecules
Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.
9 molecules share ≥1 primary target. Top 9 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| BEROTRALSTAT | ChEMBL + PubChem | Phase 4 (approved) | KLKB1 |
| DAREXABAN | ChEMBL | Phase 3 | KLKB1 |
| GABEXATE | ChEMBL | Phase 3 | KLKB1 |
| MILVEXIAN | ChEMBL | Phase 3 | KLKB1 |
| SEBETRALSTAT | ChEMBL | Phase 3 | KLKB1 |
| FENIRALSTAT | ChEMBL | Phase 2 | KLKB1 |
| GW813893 | ChEMBL | Phase 2 | KLKB1 |
| LETAXABAN | ChEMBL | Phase 2 | KLKB1 |
| Amiloride | PubChem | Approved | KLKB1 |
Related Atlas pages
- Genes: KLKB1
- In clinical trials for: hereditary angioedema
- Drugs: Berotralstat, Darexaban, Gabexate, Milvexian, Sebetralstat, Amiloride