Taurolithocholic Acid

Summary

Taurolithocholic Acid (CHEMBL408701) is a phase-3 clinical-stage small molecule targeting TAS2R1, TAS2R14, and TAS2R46; indicated across 7 conditions including primary biliary cholangitis and amyotrophic lateral sclerosis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• Targets: 3 (TAS2R1, TAS2R14, TAS2R46)
• Indications: 7 conditions
• Chemistry: 483.7 Da · C26H45NO5S

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL408701
Name	Taurolithocholic Acid
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	439763
ChEBI	CHEBI:36259
Molecular formula	C26H45NO5S
Molecular weight	483.7
InChIKey	QBYUNVOYXHFVKC-GBURMNQMSA-N

SMILES: C[C@H](CCC(=O)NCCS(=O)(=O)O)[C@H]1CC[C@@H]2[C@@]1(CC[C@H]3[C@H]2CC[C@H]4[C@@]3(CC[C@H](C4)O)C)C

IUPAC name: 2-[[(4R)-4-[(3R,5R,8R,9S,10S,13R,14S,17R)-3-hydroxy-10,13-dimethyl-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1H-cyclopenta[a]phenanthren-17-yl]pentanoyl]amino]ethanesulfonic acid

ChEBI definition: The bile acid taurine conjugate of lithocholic acid.

Other ChEBI roles (chemical / environmental): human metabolite.

Also known as: Taurolithocholic acid, Taurolithocholate, lithocholic acid, TUDC (tauroursodeoxycholic acid), tauro-lithocolic acid, TAUROLITHOCHOLIC ACID

Parent form; salt/anhydrous children: CHEMBL2028239

Patent coverage: 419 distinct patent families (1,133 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).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
TAS2R1	TAS2R1	Agonist		0%	Q9NYW7
TAS2R14	TAS2R14	Agonist		0.1%	Q9NYV8
TAS2R46	TAS2R46	Agonist		2.2%	P59540

Broader ChEMBL bioactivity targets: 4 (assay-derived). Sample: ATP-binding cassette sub-family C member 4, Vitamin D3 receptor, Ephrin type-A receptor 2, G-protein coupled bile acid receptor 1.

Bioactivity

ChEMBL activities: 4 potent at pChembl ≥ 5 of 7 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GPBAR1	6.54	EC50	290	nM	CHEMBL_ACT_2117885
GPBAR1	6.48	EC50	330	nM	CHEMBL_ACT_19095353
GPBAR1	6.07	EC50	847	nM	CHEMBL_ACT_19095323
GPBAR1	6	EC50	1005	nM	CHEMBL_ACT_22903567

Target pathways

Aggregated over 3 target gene(s): TAS2R1, TAS2R14, TAS2R46.

Top Reactome pathways

9 total, by targets touching each:

Pathway	Targets	Genes
Signal Transduction	3	TAS2R1, TAS2R14, TAS2R46
Signaling by GPCR	3	TAS2R1, TAS2R14, TAS2R46
GPCR downstream signalling	3	TAS2R1, TAS2R14, TAS2R46
G alpha (i) signalling events	3	TAS2R1, TAS2R14, TAS2R46
Class C/3 (Metabotropic glutamate/pheromone receptors)	3	TAS2R1, TAS2R14, TAS2R46
GPCR ligand binding	3	TAS2R1, TAS2R14, TAS2R46
Sensory Perception	3	TAS2R1, TAS2R14, TAS2R46
Sensory perception of taste	3	TAS2R1, TAS2R14, TAS2R46
Sensory perception of sweet, bitter, and umami (glutamate) taste	3	TAS2R1, TAS2R14, TAS2R46

Dominant GO biological processes

GO term	Targets
detection of chemical stimulus involved in sensory perception of bitter taste	3
signal transduction	3
G protein-coupled receptor signaling pathway	3
sensory perception of taste	3

Indications & clinical

Indications

7 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
primary biliary cholangitis	3	MONDO:0005388	EFO:1001486
amyotrophic lateral sclerosis	3	MONDO:0004976	MONDO:0004976
type 1 diabetes mellitus	2	MONDO:0005147	MONDO:0005147
familial amyloid neuropathy	2	MONDO:0007100	EFO:0004129
asthma	1	MONDO:0004979	MONDO:0004979
multiple sclerosis	1	MONDO:0005301	MONDO:0005301
cirrhosis of liver	0	MONDO:0005155	EFO:0001422

Clinical trials

Total trials: 0.

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.

3 molecules share ≥1 primary target. Top 3 by shared-target count:

Molecule	Source	Status	Shared targets
ISOPROTERENOL	ChEMBL	Phase 4 (approved)	TAS2R1, TAS2R14, TAS2R46
DEXTROMETHORPHAN	ChEMBL	Phase 4 (approved)	TAS2R46
FLUFENAMIC ACID	ChEMBL	Phase 2	TAS2R14