Caficrestat

Summary

Caficrestat (CHEMBL4297455) is a phase-3 clinical-stage small molecule; indicated across 2 conditions including severe acute respiratory syndrome.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Indications: 2 conditions
• Clinical trials: 4
• Chemistry: 421.4 Da · C17H10F3N5O3S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297455
Name	Caficrestat
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	56639723
Molecular formula	C17H10F3N5O3S
Molecular weight	421.4
InChIKey	YRGPAXAVTDMKDK-UHFFFAOYSA-N

SMILES: C1=CC2=C(C=C1C(F)(F)F)N=C(S2)CN3C(=O)C4=NC=CN=C4C(=N3)CC(=O)O

IUPAC name: 2-[5-oxo-6-[[5-(trifluoromethyl)-1,3-benzothiazol-2-yl]methyl]pyrazino[2,3-d]pyridazin-8-yl]acetic acid

Also known as: Aldose reductase-in-1, At 001, At-001, AT-001, Caficrestat, CAFICRESTAT

Patent coverage: 160 distinct patent families (414 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 411 (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

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

2 indications (0 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
severe acute respiratory syndrome	2	MONDO:0005091	MONDO:0100096

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

Clinical trials

Total trials: 4.

Phase distribution

Phase	Trials
PHASE3	1
PHASE2	1
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04083339	PHASE3	ACTIVE_NOT_RECRUITING	Safety and Efficacy of AT-001 in Patients With Diabetic Cardiomyopathy
NCT04365699	PHASE2	COMPLETED	Cardiovascular Effects of COVID-19
NCT01731093	PHASE1	COMPLETED	Safety and Tolerability of Antioxidant (AT-001)for Reducing Brain Oxidative Stress
NCT03062384	Not specified	COMPLETED	AT-001 for Long-term Preservation of Brain Health in Aging

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

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).