gaboxadol   Click here for help

GtoPdb Ligand ID: 4322

Synonyms: LU-02030 | MK-0928 | MK0928 | OV101 | THIP
PDB Ligand
Compound class: Synthetic organic
Comment: GABAA agonist, with some preference for δ subunit-containing channels [6].
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 2
Hydrogen bond donors 2
Rotatable bonds 0
Topological polar surface area 58.03
Molecular weight 140.06
XLogP -0.63
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 O=c1[nH]oc2c1CCNC2
Isomeric SMILES O=c1[nH]oc2c1CCNC2
InChI InChI=1S/C6H8N2O2/c9-6-4-1-2-7-3-5(4)10-8-6/h7H,1-3H2,(H,8,9)
InChI Key ZXRVKCBLGJOCEE-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
Gaboxadol was investigated as a treatment for insomnia. More recently it has been repositioned for Fragile X and Angelman syndromes based on evidence that targeting GABAAergic system deficit can improve behavioural abnormalities in these syndromes [2,4-5,10].
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT00102167 Study of MK0928 in Healthy Adult Volunteers in a Model of Insomnia (0928-006)(COMPLETED) Phase 3 Interventional H. Lundbeck A/S
NCT03697161 A Study of OV101 in Individuals With Fragile X Syndrome Phase 2 Interventional Ovid Therapeutics Inc.
NCT06334419 Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS) Phase 2 Interventional Children's Hospital Medical Center, Cincinnati
NCT02996305 A Study in Adults and Adolescents With Angelman Syndrome (STARS) Phase 2 Interventional Ovid Therapeutics Inc. 1
NCT04106557 A Study of OV101 in Individuals With Angelman Syndrome (AS) Phase 3 Interventional Ovid Therapeutics Inc. This study in pediatric participants with angelman syndrome found no improvement in symptom severity scores between placebo and gaboxadol after 12 weeks of treament. 7