Govorestat

Summary

Govorestat (CHEMBL4650327) is a phase-3 clinical-stage small molecule (ATC A16AX24) targeting AKR1B1; indicated across 3 conditions including galactosemia and peripheral neuropathy.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: A16AX24
• Targets: 1 (AKR1B1)
• Indications: 3 conditions
• Clinical trials: 5
• Chemistry: 425.4 Da · C17H10F3N3O3S2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650327
Name	Govorestat
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	132260161
ATC	A16AX24
Molecular formula	C17H10F3N3O3S2
Molecular weight	425.4
InChIKey	ORQGHAJIWGGFJK-UHFFFAOYSA-N

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

IUPAC name: 2-[4-oxo-3-[[5-(trifluoromethyl)-1,3-benzothiazol-2-yl]methyl]thieno[3,4-d]pyridazin-1-yl]acetic acid

Also known as: At-007, AT-007, AT007, Govorestat, GOVORESTAT

Patent coverage: 44 distinct patent families (110 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 104 (95%) 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
AKR1B1	aldo-keto reductase family 1 member B	Inhibition	10	1.1%	P15121

Bioactivity

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

Target pathways

Aggregated over 1 target gene(s): AKR1B1.

Top Reactome pathways

9 total, by targets touching each:

Pathway	Targets	Genes
Metabolism	1	AKR1B1
Metabolism of steroid hormones	1	AKR1B1
Pregnenolone biosynthesis	1	AKR1B1
Metabolism of lipids	1	AKR1B1
Fructose metabolism	1	AKR1B1
Fructose biosynthesis	1	AKR1B1
Galactose catabolism	1	AKR1B1
Metabolism of carbohydrates and carbohydrate derivatives	1	AKR1B1
Metabolism of steroids	1	AKR1B1

Dominant GO biological processes

GO term	Targets
retinoid metabolic process	1
epithelial cell maturation	1
renal water homeostasis	1
carbohydrate metabolic process	1
C21-steroid hormone biosynthetic process	1
regulation of urine volume	1
negative regulation of apoptotic process	1
daunorubicin metabolic process	1
doxorubicin metabolic process	1
fructose biosynthetic process	1
cellular hyperosmotic salinity response	1
metanephric collecting duct development	1
lipid metabolic process	1
prostaglandin metabolic process	1
retinol metabolic process	1

Indications & clinical

Indications

3 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
galactosemia	3	MONDO:0018116	MONDO:0009258
peripheral neuropathy	2	MONDO:0005244	EFO:0003100

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

Clinical trials

Total trials: 5.

Phase distribution

Phase	Trials
PHASE2/PHASE3	2
PHASE3	2
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05397665	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Pharmacodynamic EffIcacy and Clinical Benefit of AT 007 in Patients With Sorbitol Dehydrogenase (SORD) Deficiency
NCT04902781	PHASE2/PHASE3	COMPLETED	Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia
NCT05418829	PHASE3	UNKNOWN	AT-007 in Adult Subjects With Classic Galactosemia (CG)
NCT07191912	PHASE3	WITHDRAWN	Confirmatory Study of Govorestat in CMT-SORD
NCT04117711	PHASE1/PHASE2	COMPLETED	Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia

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.

37 molecules share ≥1 primary target. Top 37 by shared-target count:

Molecule	Source	Status	Shared targets
FOLIC ACID	ChEMBL + PubChem	Phase 4 (approved)	AKR1B1
EPALRESTAT	ChEMBL	Phase 4 (approved)	AKR1B1
ESTRADIOL	ChEMBL	Phase 4 (approved)	AKR1B1
ESTRONE	ChEMBL	Phase 4 (approved)	AKR1B1
INDOMETHACIN	ChEMBL	Phase 4 (approved)	AKR1B1
MEFENAMIC ACID	ChEMBL	Phase 4 (approved)	AKR1B1
NITAZOXANIDE	ChEMBL	Phase 4 (approved)	AKR1B1
SULINDAC	ChEMBL	Phase 4 (approved)	AKR1B1
TOLMETIN	ChEMBL	Phase 4 (approved)	AKR1B1
TOLRESTAT	ChEMBL	Phase 4 (approved)	AKR1B1
CAFFEIC ACID	ChEMBL	Phase 3	AKR1B1
CURCUMIN	ChEMBL	Phase 3	AKR1B1
GOSSYPOL	ChEMBL	Phase 3	AKR1B1
QUERCETIN	ChEMBL	Phase 3	AKR1B1
RANIRESTAT	ChEMBL	Phase 3	AKR1B1
RESVERATROL	ChEMBL	Phase 3	AKR1B1
SORBINIL	ChEMBL	Phase 3	AKR1B1
ALRESTATIN	ChEMBL	Phase 2	AKR1B1
AZD1981	ChEMBL	Phase 2	AKR1B1
CHLOROGENIC ACID	ChEMBL	Phase 2	AKR1B1
ELLAGIC ACID	ChEMBL	Phase 2	AKR1B1
FIDARESTAT	ChEMBL	Phase 2	AKR1B1
FLUFENAMIC ACID	ChEMBL	Phase 2	AKR1B1
GENISTEIN	ChEMBL	Phase 2	AKR1B1
HYMECROMONE	ChEMBL	Phase 2	AKR1B1
IMIRESTAT	ChEMBL	Phase 2	AKR1B1
ISOQUERCETIN	ChEMBL	Phase 2	AKR1B1
LIDORESTAT	ChEMBL	Phase 2	AKR1B1
LUTEOLIN	ChEMBL	Phase 2	AKR1B1
MINALRESTAT	ChEMBL	Phase 2	AKR1B1
OXEPINAC	ChEMBL	Phase 2	AKR1B1
PONALRESTAT	ChEMBL	Phase 2	AKR1B1
SEPRANOLONE	ChEMBL	Phase 2	AKR1B1
TIOPINAC	ChEMBL	Phase 2	AKR1B1
URSOLIC ACID	ChEMBL	Phase 2	AKR1B1
ZENARESTAT	ChEMBL	Phase 2	AKR1B1
ZOPOLRESTAT	ChEMBL	Phase 2	AKR1B1