Maralixibat

Summary

Maralixibat (CHEMBL363392) is an approved small molecule targeting SLC10A2; indicated across 3 conditions including alagille syndrome and liver disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Targets: 1 (SLC10A2)
• Indications: 3 conditions
• Clinical trials: 14
• Chemistry: 675 Da · C40H56N3O4S+

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL363392
Name	Maralixibat
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	9831643
Molecular formula	C40H56N3O4S+
Molecular weight	675
InChIKey	STPKWKPURVSAJF-LJEWAXOPSA-N

SMILES: CCCCC1(CS(=O)(=O)C2=C(C=C(C=C2)N(C)C)[C@H]([C@H]1O)C3=CC=C(C=C3)OCC4=CC=C(C=C4)C[N+]56CCN(CC5)CC6)CCCC

IUPAC name: (4R,5R)-5-[4-[[4-(4-aza-1-azoniabicyclo[2.2.2]octan-1-ylmethyl)phenyl]methoxy]phenyl]-3,3-dibutyl-7-(dimethylamino)-1,1-dioxo-4,5-dihydro-2H-1lambda6-benzothiepin-4-ol

Also known as: Lopixibat, LUM-001 CATION, LUM001 CATION, Maralixibat, Maralixibat cation, TAK-625, MARALIXIBAT

Parent form; salt/anhydrous children: CHEMBL17879

Patent coverage: 83 distinct patent families (205 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
SLC10A2	Sodium/bile acid and sulphated solute cotransporter 2	Inhibition	9.55	0%	Q12908

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Ileal sodium/bile acid cotransporter.

Bioactivity

ChEMBL activities: 2 potent at pChembl ≥ 5 of 2 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
SLC10A2	9.55	IC50	0.28	nM	CHEMBL_ACT_1606399
SLC10A2	9.55	IC50	0.28	nM	CHEMBL_ACT_25091984

Target pathways

Aggregated over 1 target gene(s): SLC10A2.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Recycling of bile acids and salts	1	SLC10A2

Dominant GO biological processes

GO term	Targets
response to bacterium	1
bile acid and bile salt transport	1
monoatomic ion transport	1
sodium ion transport	1
lipid transport	1
transmembrane transport	1

Indications & clinical

Indications

3 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
Alagille syndrome	3	MONDO:0007318	MONDO:0007318
liver disorder	2	MONDO:0005154	EFO:0001421
biliary atresia	2	MONDO:0008867	MONDO:0008867

Clinical trials

Total trials: 14.

Phase distribution

Phase	Trials
PHASE3	6
PHASE2	4
Not specified	2
PHASE2/PHASE3	1
PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT06413368	PHASE2/PHASE3	RECRUITING	Maralixibat in Patients With Cystic Fibrosis and Constipation
NCT06553768	PHASE3	ACTIVE_NOT_RECRUITING	Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)
NCT03353454	PHASE3	WITHDRAWN	A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT03905330	PHASE3	COMPLETED	A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
NCT04185363	PHASE3	COMPLETED	An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT05543174	PHASE3	COMPLETED	A Study of TAK-625 for the Treatment of Alagille Syndrome (ALGS)
NCT05543187	PHASE3	COMPLETED	A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT07389031	PHASE2	NOT_YET_RECRUITING	Maralixibat for Intrahepatic Cholestasis of Pregnancy
NCT04168385	PHASE2	COMPLETED	MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study
NCT04524390	PHASE2	COMPLETED	Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
NCT04729751	PHASE2	COMPLETED	A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).
NCT02475317	PHASE1	COMPLETED	Study to Assess the Relative Potency of Multiple Oral Doses of LUM001 and SHP626 in Overweight and Obese Adults as Assessed by Fecal Bile Acid Excretion
NCT07293897	Not specified	RECRUITING	A Database Study of Maralixibat (TAK-625) in Participants With Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT04530994	Not specified	APPROVED_FOR_MARKETING	A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

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.

8 molecules share ≥1 primary target. Top 8 by shared-target count:

Molecule	Source	Status	Shared targets
CHENODIOL	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
CYCLOSPORINE	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
DEOXYCHOLIC ACID	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
URSODIOL	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
TAURURSODIOL	ChEMBL	Phase 4 (approved)	SLC10A2
LINERIXIBAT	ChEMBL	Phase 3	SLC10A2
TAURODEOXYCHOLIC ACID	ChEMBL	Phase 2	SLC10A2
Maralixibat Chloride	PubChem	Approved	SLC10A2