Details of the Drug

General Information of Drug (ID: DMQZXDW)

Drug Name

Lum001

Synonyms

SHP625

Indication

Disease Entry	ICD 11	Status	REF
Primary biliary cholangitis	DB96.1	Phase 2	[1]
Primary biliary cirrhosis	DB96.1	Phase 2	[2]
Primary sclerosing cholangitis	DB96.2	Phase 2	[3]
Progressive familial intrahepatic cholestasis	5C58.03	Phase 2	[3]
Structural developmental anomalies of liver	LB20.0	Phase 2	[3]

Structure

3D MOL is unavailable

2D MOL

#Ro5 Violations (Lipinski):
2

Molecular Weight

710.4

Topological Polar Surface Area

Not Available

Rotatable Bond Count

13

Hydrogen Bond Donor Count

1

Hydrogen Bond Acceptor Count

7

Chemical Identifiers

Formula: C40H56ClN3O4S
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;chloride
Canonical 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.[Cl-]
InChI: InChI=1S/C40H56N3O4S.ClH/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;1H/q+1;/p-1/t38-,39-;/m1./s1
InChIKey: POMVPJBWDDJCMP-RUKDTIIFSA-M

Cross-matching ID

PubChem CID: 9831642
CAS Number: 228113-66-4
TTD ID: D09JXH
VARIDT ID: DR01060

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Ileal sodium/bile acid cotransporter (SLC10A2)	TTPI1M5	NTCP2_HUMAN	Inhibitor	[4]
Sodium/bile acid cotransporter (SLC10A1)	TTWZRY5	NTCP_HUMAN	Inhibitor	[1]

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Molecular Interaction Atlas (MIA)

MIA Detail

Jump to Detail Molecular Interaction Atlas of This Drug

Molecular Expression Atlas of This Drug

The Studied Disease

Primary biliary cholangitis

ICD Disease Classification

DB96.1

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 (MEA)

MEA Detail

Jump to Detail Molecular Expression Atlas of This Drug

References

1	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
2	ClinicalTrials.gov (NCT02321306) An Open-label Study to Evaluate the Long-term Safety and Tolerability of LUM001 in Patients With Primary Biliary Cirrhosis. U.S. National Institutes of Health.
3	Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA)
4	Current research on the treatment of primary sclerosing cholangitis. Intractable Rare Dis Res. 2015 Feb; 4(1): 1-6.
5	Elobixibat for the treatment of constipation. Expert Opin Investig Drugs. 2013 Feb;22(2):277-84.
6	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.
7	Inhibition of ileal Na+/bile acid cotransporter by S-8921 reduces serum cholesterol and prevents atherosclerosis in rabbits. Arterioscler Thromb Vasc Biol. 1998 Aug;18(8):1304-11.
8	Inhibition of apical sodium-dependent bile acid transporter as a novel treatment for diabetes. Am J Physiol Endocrinol Metab. 2012 Jan 1;302(1):E68-76.
9	Discovery of a highly potent, nonabsorbable apical sodium-dependent bile acid transporter inhibitor (GSK2330672) for treatment of type 2 diabetes. J Med Chem. 2013 Jun 27;56(12):5094-114.
10	Expression and transport properties of the human ileal and renal sodium-dependent bile acid transporter. Am J Physiol. 1998 Jan;274(1 Pt 1):G157-69.
11	URL: http://www.guidetopharmacology.org Nucleic Acids Res. 2015 Oct 12. pii: gkv1037. The IUPHAR/BPS Guide to PHARMACOLOGY in 2016: towards curated quantitative interactions between 1300 protein targets and 6000 ligands. (Target id: 960).
12	Bulevirtide: First Approval. Drugs. 2020 Oct;80(15):1601-1605.