Details of the Drug-Related molecule(s) Expression Atlas

General Information of Drug (ID: DMJCF1O)

Drug Name
Maralixibat Drug Info
Synonyms
Lopixibat; UNII-UYB6UOF69L; Maralixibat [USAN]; 716313-53-0; UYB6UOF69L; CHEMBL363392; Maralixibat (USAN); Lopixibat cation; CHEMBL17879; 1-(4-((4-((4R,5R)-3,3-dibutyl-7-(dimethylamino)-4-hydroxy-1,1-dioxido-2,3,4,5-tetrahydrobenzo[b]thiepin-5-yl)phenoxy)methyl)benzyl)-1,4-diazabicyclo[2.2.2]octan-1-ium; LUM001 CATION; LUM-001 CATION; SCHEMBL10013954; BDBM50140282; 4-Aza-1-azoniabicyclo(2.2.2)octane, 1-((4-((4-((4R,5R)-3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl)phenoxy)methyl)phenyl)methyl)-; D10951; Q27291331; (4R,5R)-5-[4-[[4-(1-aza-4-azoniabicyclo[2.2.2]octan-4-ylmethyl)phenyl]methoxy]phenyl]-3,3-dibutyl-7-(dimethylamino)-1,1-dioxo-4,5-dihydro-2H-1$l^{6}-benzothiepin-4-ol; 1-{4-[4-((4R,5R)-3,3-Dibutyl-7-dimethylamino-4-hydroxy-1,1-dioxo-2,3,4,5-tetrahydro-1H-1lambda*6*-benzo[b]thiepin-5-yl)-phenoxymethyl]-benzyl}-4-aza-1-azonia-bicyclo[2.2.2]octane; chloride
Indication
Disease Entry ICD 11 Status REF
Progressive familial intrahepatic cholestasis 5C58.03 Phase 3 [1]
Alagille syndrome LB20.0Y Phase 2 [2]
Cross-matching ID
PubChem CID
9831643
CAS Number
CAS 716313-53-0
TTD Drug ID
DMJCF1O

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Ileal sodium/bile acid cotransporter (SLC10A2) TTPI1M5 NTCP2_HUMAN Inhibitor [3]

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule Molecule Type Gene Name Liver Colon Kidney Small Intestine
Ileal sodium/bile acid cotransporter (SLC10A2) DTT SLC10A2 5.752 5.641 5.904 6.506
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

The Studied Disease Progressive familial intrahepatic cholestasis
ICD Disease Classification 5C58.03
Molecule Name Molecule Type Gene Name p-value Fold-Change Z-score
Ileal sodium/bile acid cotransporter (SLC10A2) DTT SLC10A2 2.54E-01 -0.06 -0.17
Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT04185363) An Extension Study of Maralixibat in Patients With Progressive Familial Intrahepatic Cholestasis (PFIC). U.S. National Institutes of Health.
2 ClinicalTrials.gov (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). (RISE). U.S. National Institutes of Health.
3 Ileal bile acid transporter inhibition as an anticholestatic therapeutic target in biliary atresia and other cholestatic disorders. Hepatol Int. 2020 Sep;14(5):677-689.