Aminocaproic Acid

Summary

Aminocaproic Acid (CHEMBL1046) is an approved small-molecule antifibrinolytic drug (ATC B02AA01) targeting PLG; indicated across 5 conditions including craniosynostosis and aortic disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: B02AA01
• Targets: 1 (PLG)
• Indications: 5 conditions
• Clinical trials: 12
• Chemistry: 131.17 Da · C6H13NO2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL1046
Name	Aminocaproic Acid
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	564
ChEBI	CHEBI:16586
ATC	B02AA01
Molecular formula	C6H13NO2
Molecular weight	131.17
InChIKey	SLXKOJJOQWFEFD-UHFFFAOYSA-N

SMILES: C(CCC(=O)O)CCN

IUPAC name: 6-aminohexanoic acid

ChEBI definition: An ε-amino acid comprising hexanoic acid carrying an amino substituent at position C-6. Used to control postoperative bleeding, and to treat overdose effects of the thrombolytic agents streptokinase and tissue plasminogen activator.

Pharmacological roles (ChEBI): antifibrinolytic drug, hematologic agent.

Other ChEBI roles (chemical / environmental): metabolite.

Also known as: .epsilon.-aminocaproic acid, 177 J.D, 177 J.D., 177 JD, Acide aminocaproique, Acido aminocaproico, Amicar, Aminocaproic, Aminocaproic acid, CL 10304, CL-10304, CY-116

Patent coverage: 31,063 distinct patent families (95,343 SureChEMBL compound mentions), from 1 matched compound structure(s). 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
PLG	plasminogen	Binding	4.4	4.6%	P00747

Broader ChEMBL bioactivity targets: 10 (assay-derived). Sample: Prelamin-A/C, ATP-dependent DNA helicase Q1, Ferritin light chain, Plasminogen, Thyroid hormone receptor beta, Menin/Histone-lysine N-methyltransferase MLL, Sodium-dependent noradrenaline transporter, Sodium-dependent dopamine transporter, Polyunsaturated fatty acid lipoxygenase ALOX15, Aldehyde dehydrogenase 1A1.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 11 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
ALOX15	8.3	Potency	5	nM	CHEMBL_ACT_4463461
THRB	6.4	Potency	398.1	nM	CHEMBL_ACT_4005856
LMNA	5.9	Potency	1259	nM	CHEMBL_ACT_3641529
RECQL	5.2	Potency	6310	nM	CHEMBL_ACT_4727395
PLG	5.05	Kd	9000	nM	CHEMBL_ACT_19054657

Target pathways

Aggregated over 1 target gene(s): PLG.

Top Reactome pathways

6 total, by targets touching each:

Pathway	Targets	Genes
Platelet degranulation	1	PLG
Degradation of the extracellular matrix	1	PLG
Activation of Matrix Metalloproteinases	1	PLG
Signaling by PDGF	1	PLG
Regulation of Insulin-like Growth Factor (IGF) transport and uptake by Insulin-like Growth Factor Binding Proteins (IGFBPs)	1	PLG
Dissolution of Fibrin Clot	1	PLG

Dominant GO biological processes

GO term	Targets
proteolysis	1
blood coagulation	1
negative regulation of cell population proliferation	1
negative regulation of cell-substrate adhesion	1
protein processing	1
extracellular matrix disassembly	1
tissue regeneration	1
fibrinolysis	1
positive regulation of blood vessel endothelial cell migration	1
myoblast differentiation	1
muscle cell cellular homeostasis	1
tissue remodeling	1
biological process involved in interaction with symbiont	1
negative regulation of fibrinolysis	1
positive regulation of fibrinolysis	1

Indications & clinical

Indications

5 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
craniosynostosis	1	MONDO:0015469	MONDO:0015469
aortic disorder	0	MONDO:0005561	EFO:0005775
rosacea	0	MONDO:0006604	EFO:1000760

2 further indication records had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 12.

Phase distribution

Phase	Trials
Not specified	4
PHASE4	3
PHASE2	2
PHASE2/PHASE3	1
PHASE1/PHASE2	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT02030821	PHASE4	COMPLETED	TXA vs. Amicar in Total Knee and Hip Arthroplasty
NCT02238288	PHASE4	UNKNOWN	Randomized Clinical Trial With Aminocaproic Acid in the Prevention of Exodontic Bleeding in Anticoagulants Patients
NCT04814433	PHASE4	TERMINATED	Evaluating Postoperative Non-Opioid Pain Management Utilizing Local Anesthetics Coupled With Modulated Coagulation
NCT00223704	PHASE2/PHASE3	COMPLETED	Bradykinin Receptor Antagonism During Cardiopulmonary Bypass
NCT00103545	PHASE1/PHASE2	COMPLETED	Use of ACA 125 in Patients With Ovarian Cancer: Safety and Immune Response
NCT02074436	PHASE2	TERMINATED	PRevention Of BLeeding in hEmatological Malignancies With Antifibrinolytic (Epsilon Aminocaproic Acid)
NCT03365999	PHASE2	COMPLETED	Oral Tranexamic Acid vs. Oral Aminocaproic Acid to Reduce Blood Loss and Transfusion After Total Knee Replacement.
NCT00912119	PHASE1	COMPLETED	Amicar Pharmacokinetics of Children Having Craniofacial Surgery
NCT00617955	Not specified	COMPLETED	Effects of Aprotinin During Cardiac Surgery/Long Term Death Rates
NCT01248104	Not specified	COMPLETED	Comparison of Tranexamic to Epsilon Aminocaproic Acid: a Prospective Analysis of Blood Conservation in Cardiac Surgery
NCT01391182	Not specified	COMPLETED	Epsilon Aminocaproic Acid (EACA) for the Reduction of Blood Loss in Total Hip Arthroplasty (THA)
NCT04424563	Not specified	COMPLETED	Activated Factor Seven in Traumatic Retro Peritoneal Hematoma

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

10 molecules share ≥1 primary target. Top 10 by shared-target count:

Molecule	Source	Status	Shared targets
BEROTRALSTAT	ChEMBL	Phase 4 (approved)	PLG
MELAGATRAN	ChEMBL	Phase 4 (approved)	PLG
PENTAMIDINE	ChEMBL	Phase 4 (approved)	PLG
TELAPREVIR	ChEMBL	Phase 4 (approved)	PLG
TRANEXAMIC ACID	ChEMBL	Phase 4 (approved)	PLG
DABIGATRAN	ChEMBL	Phase 3	PLG
GABEXATE	ChEMBL	Phase 3	PLG
MILVEXIAN	ChEMBL	Phase 3	PLG
NAFAMOSTAT	ChEMBL	Phase 3	PLG
EFEGATRAN	ChEMBL	Phase 2	PLG