govorestat   Click here for help

GtoPdb Ligand ID: 13307

Synonyms: AT-007 | AT007
PDB Ligand
Compound class: Synthetic organic
Comment: Govorestat (AT-007) is anorally active, CNS penetrant aldose reductase inhibitor [2]. It blocks the conversion of galactose into toxic galactitol in patients with inherited galactosemia.
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species

govorestat is commercially available from our sponsors

2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/). All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 6
Hydrogen bond donors 1
Rotatable bonds 5
Topological polar surface area 132.93
Molecular weight 425.41
XLogP 1.73
No. Lipinski's rules broken 0

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)

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Canonical SMILES C1=CC2=C(C=C1C(F)(F)F)N=C(CN3C(=O)C4=CSC=C4C(=N3)CC(=O)O)S2
Isomeric SMILES C1=CC2=C(C=C1C(F)(F)F)N=C(S2)CN3C(=O)C4=CSC=C4C(=N3)CC(=O)O
InChI InChI=1S/C17H10F3N3O3S2/c18-17(19,20)8-1-2-13-12(3-8)21-14(28-13)5-23-16(26)10-7-27-6-9(10)11(22-23)4-15(24)25/h1-3,6-7H,4-5H2,(H,24,25)
InChI Key ORQGHAJIWGGFJK-UHFFFAOYSA-N

Generated using the Chemistry Development Kit (CDK) (Willighagen EL et al. Journal of Cheminformatics vol. 9:33. 2017, doi:10.1186/s13321-017-0220-4; https://cdk.github.io/)
No information available.
Summary of Clinical Use Click here for help
Govorestat (AT-007) is a clinical candidate for the treatment of type I (classic) galactosemia [1]. Type I galactosemia is a rare genetic metabolic condition caused by galactose-1-phosphate uridyltransferase (GALT) deficiency. Govorestat for the treatment of galactosemia has received Orphan Medicinal Product Designation from the EMA and Orphan Drug Designation, Pediatric Rare Disease Designation and Fast Track Designation from the FDA.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT04902781 Clinical Benefit, Safety, PK and PD Study of AT-007 in Pediatric Subjects With Classic Galactosemia Phase 2/Phase 3 Interventional Applied Therapeutics, Inc.
NCT05418829 AT-007 in Adult Subjects With Classic Galactosemia (CG) Phase 3 Interventional Applied Therapeutics, Inc.
NCT04117711 Safety and Pharmacokinetics of AT-007 in Healthy Subjects and in Adult Subjects With Classic Galactosemia Phase 1/Phase 2 Interventional Applied Therapeutics, Inc.