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Atlas / Drug / Gaboxadol

Gaboxadol

drug
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Also known as LU-02030MK-0928NSC-759585SID11113715SID11111837SID90341079SID11111836SID104171241SID170466512

Summary

Gaboxadol (CHEMBL312443) is a phase-3 clinical-stage small molecule targeting GABRA1, GABRA2, and GABRA3; indicated across 4 conditions including insomnia and angelman syndrome.

At a glance

  • Status: Max clinical phase 3 (not approved)
  • Modality: Small molecule
  • Targets: 10 (GABRA1, GABRA2, GABRA3…)
  • Indications: 4 conditions
  • Clinical trials: 13
  • Chemistry: 140.14 Da · C6H8N2O2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL312443
NameGaboxadol
TypeSmall molecule
Max phase3
FDA approvedno
PubChem CID3448
Molecular formulaC6H8N2O2
Molecular weight140.14
InChIKeyZXRVKCBLGJOCEE-UHFFFAOYSA-N

SMILES: C1CNCC2=C1C(=O)NO2

IUPAC name: 4,5,6,7-tetrahydro-[1,2]oxazolo[5,4-c]pyridin-3-one

Also known as: Gaboxadol, LU-02030, MK-0928, NSC-759585, SID11113715, SID11111837, SID90341079, SID11111836, SID104171241, GABOXADOL, SID170466512, gaboxadol

Parent form; salt/anhydrous children: CHEMBL1255746

Patent coverage: 1,535 distinct patent families (5,584 SureChEMBL compound mentions), from 2 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).

GeneTargetActionpAffinityCancer dependencyUniProt
GABRA1GABAA receptor α1 subunitAgonist0%P14867
GABRA2GABAA receptor α2 subunitAgonist0%P47869
GABRA3GABAA receptor α3 subunitAgonist0%P34903
GABRA4GABAA receptor α4 subunitAgonist0%P48169
GABRA5GABAA receptor α5 subunitAgonist0.9%P31644
GABRA6GABAA receptor α6 subunitAgonist0.1%Q16445
GABRDGABAA receptor δ subunitFull agonist1.2%O14764
GABRR1GABAA receptor ρ1 subunitAntagonist0%P24046
GABRR2GABAA receptor ρ2 subunitAntagonist0.2%P28476
GABRR3GABAA receptor ρ3 subunitAntagonist2.7%A8MPY1

Broader ChEMBL bioactivity targets: 5 (assay-derived). Sample: Nuclear receptor ROR-gamma, GABA-A receptor; anion channel, Menin/Histone-lysine N-methyltransferase MLL, GABA-A receptor; alpha-1/beta-2/gamma-2, Cytochrome P450 2C19.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 12 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
O090287.5IC5031.5nMCHEMBL_ACT_1214006
O090287.04IC5092nMCHEMBL_ACT_725250
O090287IC50100nMCHEMBL_ACT_927987
O090286.89IC50130nMCHEMBL_ACT_801235
O090286.89IC50130nMCHEMBL_ACT_926240
O090286.8Ki160nMCHEMBL_ACT_1739820

Target pathways

Aggregated over 10 target gene(s): GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD, GABRR1, GABRR2, GABRR3.

Top Reactome pathways

2 total, by targets touching each:

PathwayTargetsGenes
GABA receptor activation9GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRR1, GABRR2, GABRR3
Signaling by ERBB41GABRA1

Dominant GO biological processes

GO termTargets
gamma-aminobutyric acid signaling pathway10
synaptic transmission, GABAergic10
chloride transmembrane transport10
monoatomic ion transport10
chloride transport10
monoatomic ion transmembrane transport10
regulation of postsynaptic membrane potential8
inhibitory synapse assembly6
chemical synaptic transmission5
signal transduction4
regulation of presynaptic membrane potential3
central nervous system development1
behavioral fear response1
associative learning1
inner ear receptor cell development1

Indications & clinical

Indications

4 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)PhaseMONDOEFO
insomnia3MONDO:0013600EFO:0004698
Angelman syndrome3MONDO:0007113MONDO:0007113
major depressive disorder2MONDO:0002009MONDO:0002009
fragile X syndrome2MONDO:0010383MONDO:0010383

Clinical trials

Total trials: 13.

Phase distribution

PhaseTrials
PHASE310
PHASE23

Top trials by phase / activity

NCTPhaseStatusTitle
NCT00094627PHASE3COMPLETEDAdult Primary Insomnia Polysomnographic Study (0928-004)(COMPLETED)
NCT00095043PHASE3COMPLETEDSafety of MK0928 on Insomnia in the Elderly (0928-005)(COMPLETED)
NCT00102167PHASE3COMPLETEDStudy of MK0928 in Healthy Adult Volunteers in a Model of Insomnia (0928-006)(COMPLETED)
NCT00103818PHASE3COMPLETEDAn Investigational Study of MK0928 in Adult Outpatients With Insomnia (0928-014)(COMPLETED)
NCT00209846PHASE3COMPLETEDA Study of Gaboxadol in Primary Insomnia
NCT00209924PHASE3COMPLETEDA Study of Gaboxadol in Primary Insomnia
NCT00209937PHASE3COMPLETEDA Prospective Active-Reference Study of Gaboxadol in Primary Insomnia
NCT00209950PHASE3COMPLETEDA Prospective Open-Label Extension Study of Gaboxadol in Primary Insomnia
NCT00209963PHASE3COMPLETEDA Study of Gaboxadol in Primary Insomnia
NCT04106557PHASE3COMPLETEDA Study of OV101 in Individuals With Angelman Syndrome (AS)
NCT00807248PHASE2COMPLETEDShort-term Study of Combination Treatment of Escitalopram and Gaboxadol in Major Depressive Disorder
NCT04823052PHASE2WITHDRAWNInvestigation of Sulindac (HLX-0201) and Gaboxadol (HLX-0206) in Male Fragile X Syndrome Patients Aged 13-40
NCT06334419PHASE2COMPLETEDPlacebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

85 molecules share ≥1 primary target. Top 85 by shared-target count:

MoleculeSourceStatusShared targets
APALUTAMIDEChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
CLONAZEPAMChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
DIAZEPAMChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
ENZALUTAMIDEChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
FLUMAZENILChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
BREXANOLONEChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
GANAXOLONEChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
LIOTHYRONINEChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
ALFAXALONEChEMBL + PubChemPhase 2 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
ABECARNILChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
BAICALEINChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
DELORAZEPAMChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
FLAVONEChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
PROGABIDEChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6, GABRD
PROPOFOLChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4, GABRA5, GABRA6
.gamma.-aminobutyric acidPubChemApprovedGABRA4, GABRA6, GABRD, GABRR1, GABRR2, GABRR3
BRETAZENILChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA5, GABRA6
ALPRAZOLAMChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA5
CHLORDIAZEPOXIDEChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA5
ESZOPICLONEChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA4
FLUNITRAZEPAMChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA5
TRIAZOLAMChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3, GABRA5
AZD7325ChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA5
BASMISANILChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA5
MK-0777ChEMBLPhase 2GABRA1, GABRA2, GABRA3, GABRA5
LINDANEChEMBLPhase 4 (approved)GABRA1, GABRA6, GABRR1
ZOLPIDEMChEMBLPhase 4 (approved)GABRA1, GABRA2, GABRA3
ZURANOLONEChEMBLPhase 4 (approved)GABRA1, GABRA4, GABRD
DARIGABATChEMBLPhase 2GABRA1, GABRA3, GABRA5
PANADIPLONChEMBLPhase 2GABRA1, GABRA3, GABRA6
CaffeinePubChemApprovedGABRA4, GABRA6, GABRD
CiprofloxacinPubChemApprovedGABRA4, GABRA6, GABRD
FipronilPubChemApprovedGABRA4, GABRA6, GABRD
PhenytoinPubChemApprovedGABRA4, GABRA6, GABRD
valproatePubChemApprovedGABRA4, GABRA6, GABRD
CRIZOTINIBChEMBL + PubChemPhase 4 (approved)GABRA1, GABRA2
ASENAPINEChEMBLPhase 4 (approved)GABRA1, GABRA2
AZELASTINEChEMBLPhase 4 (approved)GABRA1, GABRA2
BENPERIDOLChEMBLPhase 4 (approved)GABRA1, GABRA2
CANDESARTAN CILEXETILChEMBLPhase 4 (approved)GABRA1, GABRA2
CHLORAMBUCILChEMBLPhase 4 (approved)GABRA1, GABRA2
CLOZAPINEChEMBLPhase 4 (approved)GABRA1, GABRA2
EPINASTINEChEMBLPhase 4 (approved)GABRA1, GABRA2
ETOMIDATEChEMBLPhase 4 (approved)GABRA1, GABRA2
GLAFENINEChEMBLPhase 4 (approved)GABRA1, GABRA2
SIMVASTATINChEMBLPhase 4 (approved)GABRA1, GABRA2
TIPRANAVIRChEMBLPhase 4 (approved)GABRA1, GABRA2
TROGLITAZONEChEMBLPhase 4 (approved)GABRA1, GABRA2
ZALEPLONChEMBLPhase 4 (approved)GABRA1, GABRA2
SURAMINChEMBLPhase 3GABRA1, GABRA2
TIRATRICOLChEMBLPhase 3GABRA1, GABRA2
SARIPIDEMChEMBLPhase 2GABRA1, GABRA5
ZOTEPINEChEMBLPhase 2GABRA1, GABRA2
AfatinibPubChemApprovedGABRA1, GABRA2
ApixabanPubChemApprovedGABRA1, GABRA2
BinimetinibPubChemApprovedGABRA1, GABRA2
chenodiolPubChemApprovedGABRA1, GABRA2
FulvestrantPubChemApprovedGABRA1, GABRA2
gentian violetPubChemApprovedGABRA1, GABRA2
ImipenemPubChemApprovedGABRA1, GABRA2
LactulosePubChemApprovedGABRA1, GABRA2
levocarnitinePubChemApprovedGABRA1, GABRA2
podofiloxPubChemApprovedGABRA1, GABRA2
Propylene GlycolPubChemApprovedGABRA1, GABRA2
PyrazinamidePubChemApprovedGABRA1, GABRA2
PyridoxinePubChemApprovedGABRA1, GABRA2
RiociguatPubChemApprovedGABRA1, GABRA2
saxagliptinPubChemApprovedGABRA1, GABRA2
ThiaminePubChemApprovedGABRA1, GABRA2
VorapaxarPubChemApprovedGABRA1, GABRA2
AMINOLEVULINIC ACIDChEMBL + PubChemPhase 4 (approved)GABRR1
PENTOBARBITALChEMBLPhase 4 (approved)GABRA2
STIRIPENTOLChEMBLPhase 4 (approved)GABRA1
ALISERTIBChEMBLPhase 3GABRA1
ALOGABATChEMBLPhase 2GABRA1
LORECLEZOLEChEMBLPhase 2GABRA1
PIPERINEChEMBLPhase 2GABRA1
REDAFAMDASTATChEMBLPhase 2GABRA1
SGS-742ChEMBLPhase 2GABRR1
ApremilastPubChemApprovedGABRA2
CocainePubChemApprovedGABRD
IdelalisibPubChemApprovedGABRA2
OfloxacinPubChemApprovedGABRD
OlodaterolPubChemApprovedGABRA2
TavaborolePubChemApprovedGABRA2

About this page

Generated deterministically by Sugi Atlas from primary biomedical databases via BioBTree. Every record on this page traces to a primary source.

Generated
Atlas version
v1.1.4
BioBTree
v2.0.2

Sources 30

This page pulls from 30 datasets indexed by BioBTree:

chebichembl_activitychembl_moleculechembl_targetcivic_evidenceclingen_dosageclinical_trialsdepmapefoensemblentrezgogoparentgtopdbgtopdb_interactiongtopdb_ligandhgncmeshmondomsigdbpatent_compoundpharmgkbpharmgkb_guidelinepubchempubchem_activityreactomereactomeparentrefseqtranscriptuniprot