maralixibat   Click here for help

GtoPdb Ligand ID: 11708

Synonyms: compound 74 [PMID: 16134951] | Livmarli® | lopixibat (deleted INN) | LUM 001 | LUM-001 | LUM001
Approved drug
maralixibat is an approved drug (FDA (2021), EMA (2022))
Compound class: Synthetic organic
Comment: This is the parent molecule of maralixibat chloride as described in the INN. Maralixibat (LUM001) is a clinical stage, orally administered ileal bile acid transporter (IBAT; SLC10A2) inhibitor [1]. It was developed for therapeutic potential to treat cholestatic pruritus.
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
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 CDK toolkit. 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 5
Hydrogen bond donors 1
Rotatable bonds 13
Topological polar surface area 78.46
Molecular weight 674.4
XLogP 6.93
No. Lipinski's rules broken 2
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.

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Canonical SMILES CCCCC1(CCCC)CS(=O)(=O)c2c([C@H]([C@H]1O)c1ccc(cc1)OCc1ccc(cc1)C[N+]13CCN(CC1)CC3)cc(cc2)N(C)C
Isomeric SMILES CCCCC1(CS(=O)(=O)c2c(cc(cc2)N(C)C)[C@H]([C@H]1O)c1ccc(cc1)OCc1ccc(cc1)C[N+]12CCN(CC1)CC2)CCCC
InChI InChI=1S/C40H56N3O4S/c1-5-7-19-40(20-8-6-2)30-48(45,46)37-18-15-34(41(3)4)27-36(37)38(39(40)44)33-13-16-35(17-14-33)47-29-32-11-9-31(10-12-32)28-43-24-21-42(22-25-43)23-26-43/h9-18,27,38-39,44H,5-8,19-26,28-30H2,1-4H3/q+1/t38-,39-/m1/s1
InChI Key STPKWKPURVSAJF-LJEWAXOPSA-N
No information available.
Summary of Clinical Use Click here for help
Maralixibat was evaluated in clinical studies to evaluate its efficacy as a therapy for cholestatic liver disease to reduce both the associated pruritus and bile acid-induced liver damage. The EMA issued orphan designations to allow maralixibat to be used for the treatment of primary biliary cirrhosis, primary sclerosing cholangitis, Alagille syndrome and progressive familial intrahepatic cholestasis (January 2014), and biliary atresia (July 2020), An oral formulation of maralixibat (Livmarli®) was granted FDA approval in September 2021, as a treatment of cholestatic pruritus in patients with Alagille syndrome ≥1 year old.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT02057692 Evaluation of LUM001 in the Reduction of Pruritus in Alagille Syndrome Phase 2 Interventional Mirum Pharmaceuticals, Inc. 3
NCT01904058 Phase 2 Study to Evaluate LUM001 in Combination With Ursodeoxycholic Acid in Patients With Primary Biliary Cirrhosis Phase 2 Interventional Mirum Pharmaceuticals, Inc. 2
NCT04168385 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 Phase 2 Interventional Mirum Pharmaceuticals, Inc.
NCT04729751 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). Phase 2 Interventional Mirum Pharmaceuticals, Inc.
NCT03905330 A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC) Phase 3 Interventional Mirum Pharmaceuticals, Inc.