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Atlas / Drug / Arbaclofen

Arbaclofen

drug
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Also known as (r)-baclofenArbaclofeneArbaclofenoBaclofen (r)-formBaclofen, (r)-Baclofen, r(-)-Baclofenr-D-baclofenR-(-)-baclofenSTX-209STX209SID11113710(-)-baclofen

Summary

Arbaclofen (CHEMBL301742) is a phase-3 clinical-stage small molecule targeting GABBR1; indicated across 3 conditions including autism spectrum disorder and fragile x syndrome.

At a glance

  • Status: Max clinical phase 3 (not approved)
  • Modality: Small molecule
  • Targets: 1 (GABBR1)
  • Indications: 3 conditions
  • Clinical trials: 20
  • Chemistry: 213.66 Da · C10H12ClNO2

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL301742
NameArbaclofen
TypeSmall molecule
Max phase3
FDA approvedno
PubChem CID44602
Molecular formulaC10H12ClNO2
Molecular weight213.66
InChIKeyKPYSYYIEGFHWSV-QMMMGPOBSA-N

SMILES: C1=CC(=CC=C1[C@@H](CC(=O)O)CN)Cl

IUPAC name: (3R)-4-amino-3-(4-chlorophenyl)butanoic acid

Also known as: (r)-baclofen, Arbaclofen, Arbaclofene, Arbaclofeno, Baclofen (r)-form, Baclofen, (r)-, Baclofen, r(-)-, Baclofen, r-, D-baclofen, R-(-)-baclofen, STX-209

Patent coverage: 115 distinct patent families (253 SureChEMBL compound mentions), from 1 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
GABBR1GABAB1Full agonist4.60.2%Q9UBS5
GABAB receptorFull agonist8.5

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: GABA-B receptor, GABA B receptor, Cytochrome P450 2D6, Cytochrome P450 1A2, Cytochrome P450 2C9, Cytochrome P450 2C19.

Bioactivity

ChEMBL activities: 8 potent at pChembl ≥ 5 of 11 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
GABBR27.82IC5015nMCHEMBL_ACT_80260
O888717.5IC5032nMCHEMBL_ACT_80262
O888716.85IC50140nMCHEMBL_ACT_781694
CYP2D66.1Potency794.3nMCHEMBL_ACT_4949869
CYP2D66.1AC50794.3nMCHEMBL_ACT_6062802
GABBR26EC501000nMCHEMBL_ACT_12648722
CYP2C195.6Potency2512nMCHEMBL_ACT_4016485
CYP2C195.6AC502512nMCHEMBL_ACT_6030525

Target pathways

Aggregated over 1 target gene(s): GABBR1.

Top Reactome pathways

5 total, by targets touching each:

PathwayTargetsGenes
Activation of G protein gated Potassium channels1GABBR1
G alpha (i) signalling events1GABBR1
Class C/3 (Metabotropic glutamate/pheromone receptors)1GABBR1
GABA B receptor activation1GABBR1
Inhibition of voltage gated Ca2+ channels via Gbeta/gamma subunits1GABBR1

Dominant GO biological processes

GO termTargets
osteoblast differentiation1
adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway1
gamma-aminobutyric acid signaling pathway1
negative regulation of cell population proliferation1
positive regulation of glutamate secretion1
negative regulation of gamma-aminobutyric acid secretion1
negative regulation of epinephrine secretion1
negative regulation of dopamine secretion1
response to nicotine1
response to ethanol1
negative regulation of synaptic transmission1
synaptic transmission, GABAergic1
positive regulation of growth hormone secretion1
neuron-glial cell signaling1
signal transduction1

Indications & clinical

Indications

2 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
autism spectrum disorder3MONDO:0005258EFO:0003756
fragile X syndrome3MONDO:0010383MONDO:0010383

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

Clinical trials

Total trials: 20.

Phase distribution

PhaseTrials
PHASE39
PHASE28
Not specified2
EARLY_PHASE11

Top trials by phase / activity

NCTPhaseStatusTitle
NCT05179577PHASE3NOT_YET_RECRUITINGA Study to Investigate Arbaclofen ER Tablets for the Treatment of Spasticity in Patients With Multiple Sclerosis
NCT01282268PHASE3COMPLETEDEfficacy and Safety Study of STX209 (Arbaclofen) for Social Withdrawal in Adolescents and Adults With Fragile X Syndrome
NCT01325220PHASE3COMPLETEDEfficacy and Safety Study of STX209 (Arbaclofen) for the Treatment of Social Withdrawal in Children With Fragile X Syndrome
NCT01555333PHASE3TERMINATEDAn Open Label Extension Study in Subjects With Fragile X Syndrome
NCT01706523PHASE3TERMINATEDOpen Label Extension Study of STX209 (Arbaclofen) in Autism Spectrum Disorders
NCT01743651PHASE3COMPLETEDEfficacy Study of Arbaclofen to Treat Spasticity in Multiple Sclerosis
NCT01844232PHASE3COMPLETEDOne Year, Open Label, Dose Escalation Long-term Safety Study in Multiple Sclerosis (MS) Subjects With Spasticity
NCT03290131PHASE3COMPLETEDA Study to Investigate the Safety and Effectiveness of Arbaclofen Extended-Release Tablets for Patients With MS
NCT03319732PHASE3COMPLETEDA Study to Evaluate the Long-term Safety of Arbaclofen Extended-Release Tablets for Patients With Spasticity Due to MS
NCT04271332PHASE2ACTIVE_NOT_RECRUITINGSafety, Tolerability, and Efficacy of Arbaclofen in 16p11.2 Deletion
NCT00788073PHASE2COMPLETEDSafety, Tolerability and Efficacy Study of STX209 in Subjects With Fragile X Syndrome
NCT00846547PHASE2COMPLETEDOpen-Label Study of the Safety and Tolerability of STX209 in Subjects With Autism Spectrum Disorders
NCT01013480PHASE2TERMINATEDAn Open Label Extension Study of STX209 in Subjects With Fragile X Syndrome
NCT01064973PHASE2TERMINATEDAn Open Label Extension Study of STX209 in Subjects With Autism Spectrum Disorders
NCT01288716PHASE2COMPLETEDStudy of Arbaclofen for the Treatment of Social Withdrawal in Subjects With Autism Spectrum Disorders
NCT03682978PHASE2COMPLETEDArbaclofen in Children and Adolescents With ASD
NCT03887676PHASE2COMPLETEDArbaclofen vs. Placebo in the Treatment of Children and Adolescents With ASD (ARBA)
NCT02278328EARLY_PHASE1COMPLETEDMEG Study of Acute STX209 Effects in ASD
NCT00823368Not specifiedCOMPLETEDBiomarker Testing and DNA Collection in Subjects Participating in Protocol 22001
NCT00892580Not specifiedCOMPLETEDBiomarker and DNA Collection in Subjects Participating in Protocol 22003

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.

98 molecules share ≥1 primary target. Top 98 by shared-target count:

MoleculeSourceStatusShared targets
BACLOFENChEMBL + PubChemPhase 4 (approved)GABBR1
SGS-742ChEMBLPhase 2GABBR1
.gamma.-aminobutyric acidPubChemApprovedGABBR1
AcyclovirPubChemApprovedGABBR1
Alendronic AcidPubChemApprovedGABBR1
AlogliptinPubChemApprovedGABBR1
AmiloridePubChemApprovedGABBR1
AmlodipinePubChemApprovedGABBR1
AmoxapinePubChemApprovedGABBR1
AmoxicillinPubChemApprovedGABBR1
AspirinPubChemApprovedGABBR1
AtorvastatinPubChemApprovedGABBR1
AvatrombopagPubChemApprovedGABBR1
azithromycin, unspecified formPubChemApprovedGABBR1
BelinostatPubChemApprovedGABBR1
BenazeprilPubChemApprovedGABBR1
BicalutamidePubChemApprovedGABBR1
BosentanPubChemApprovedGABBR1
BosutinibPubChemApprovedGABBR1
BumetanidePubChemApprovedGABBR1
BupropionPubChemApprovedGABBR1
CaffeinePubChemApprovedGABBR1
CandesartanPubChemApprovedGABBR1
Candesartan CilexetilPubChemApprovedGABBR1
CarvedilolPubChemApprovedGABBR1
CelecoxibPubChemApprovedGABBR1
CimetidinePubChemApprovedGABBR1
CiprofloxacinPubChemApprovedGABBR1
ClozapinePubChemApprovedGABBR1
CodeinePubChemApprovedGABBR1
cyclosporinePubChemApprovedGABBR1
DiclofenacPubChemApprovedGABBR1
DiphenhydraminePubChemApprovedGABBR1
DonepezilPubChemApprovedGABBR1
DycloninePubChemApprovedGABBR1
EnalaprilPubChemApprovedGABBR1
ethinyl estradiolPubChemApprovedGABBR1
FenfluraminePubChemApprovedGABBR1
FluoxetinePubChemApprovedGABBR1
Fluticasone FuroatePubChemApprovedGABBR1
GabapentinPubChemApprovedGABBR1
GemfibrozilPubChemApprovedGABBR1
GlipizidePubChemApprovedGABBR1
HaloperidolPubChemApprovedGABBR1
HydrochlorothiazidePubChemApprovedGABBR1
IrbesartanPubChemApprovedGABBR1
LamivudinePubChemApprovedGABBR1
LansoprazolePubChemApprovedGABBR1
LeflunomidePubChemApprovedGABBR1
LiothyroninePubChemApprovedGABBR1
LosartanPubChemApprovedGABBR1
LoxapinePubChemApprovedGABBR1
MeloxicamPubChemApprovedGABBR1
ModafinilPubChemApprovedGABBR1
NefazodonePubChemApprovedGABBR1
NilotinibPubChemApprovedGABBR1
OlanzapinePubChemApprovedGABBR1
OsilodrostatPubChemApprovedGABBR1
OxazepamPubChemApprovedGABBR1
PalbociclibPubChemApprovedGABBR1
ParoxetinePubChemApprovedGABBR1
PasireotidePubChemApprovedGABBR1
PemigatinibPubChemApprovedGABBR1
PergolidePubChemApprovedGABBR1
PhenterminePubChemApprovedGABBR1
phenylbutyric acidPubChemApprovedGABBR1
PhenylephrinePubChemApprovedGABBR1
PioglitazonePubChemApprovedGABBR1
QuinaprilPubChemApprovedGABBR1
risedronic acidPubChemApprovedGABBR1
RisperidonePubChemApprovedGABBR1
RizatriptanPubChemApprovedGABBR1
RosiglitazonePubChemApprovedGABBR1
SertralinePubChemApprovedGABBR1
SildenafilPubChemApprovedGABBR1
SimvastatinPubChemApprovedGABBR1
SirolimusPubChemApprovedGABBR1
SitagliptinPubChemApprovedGABBR1
StavudinePubChemApprovedGABBR1
SumatriptanPubChemApprovedGABBR1
TacrolimusPubChemApprovedGABBR1
TegaserodPubChemApprovedGABBR1
TelmisartanPubChemApprovedGABBR1
TemozolomidePubChemApprovedGABBR1
TetracainePubChemApprovedGABBR1
ThalidomidePubChemApprovedGABBR1
ThiothixenePubChemApprovedGABBR1
Tiotropium Bromide MonohydratePubChemApprovedGABBR1
TofacitinibPubChemApprovedGABBR1
TolterodinePubChemApprovedGABBR1
TopiramatePubChemApprovedGABBR1
TriamterenePubChemApprovedGABBR1
Valproic AcidPubChemApprovedGABBR1
ViloxazinePubChemApprovedGABBR1
VorinostatPubChemApprovedGABBR1
WarfarinPubChemApprovedGABBR1
ZidovudinePubChemApprovedGABBR1
ZolpidemPubChemApprovedGABBR1

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