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

General Information of Drug (ID: DM9EZM0)

Drug Name

Iptacopan Drug Info

Synonyms

LNP-023; UNII-8E05T07Z6W; 8E05T07Z6W; 1644670-37-0; 4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl)benzoic acid; 4-[(2~{S},4~{S})-4-ethoxy-1-[(5-methoxy-7-methyl-1~{H}-indol-4-yl)methyl]piperidin-2-yl]benzoic acid; 4-[(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl]benzoic acid; Iptacopan [INN]; CHEMBL4594448; SCHEMBL16400416; GTPL10710; US9682968, Example-26a; BDBM160475; ZINC223246892; compound 41 [PMID: 32073845]; HY-127105; CS-0093107; 4-((2S,4S)-(4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl))benzoic acid; Benzoic acid, 4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)-2-piperidinyl)-; JGQ

Indication

Disease Entry	ICD 11	Status	REF
Paroxysmal nocturnal haemoglobinuria	3A21.0	Approved	[1]
IgA nephropathy	MF8Y	Phase 3	[2]

Cross-matching ID

PubChem CID: 90467622
CAS Number: CAS 1644670-37-0
TTD Drug ID: DM9EZM0

Molecule(s) Related to This Drug

Drug Therapeutic Target (DTT)

DTT Name	DTT ID	UniProt ID	MOA	REF
Complement factor B (CFB)	TTA0P7K	CFAB_HUMAN	Inhibitor	[3]

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

Molecule	Molecule Type	Gene Name	Liver	Colon	Kidney	Small Intestine
Complement factor B (CFB)	DTT	CFB	10.419	4.895	7.235	6.78

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

Paroxysmal nocturnal haemoglobinuria

ICD Disease Classification

3A21.0

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Complement factor B (CFB)	DTT	CFB	1.63E-02	0.07	0.13

Molecular Expression Atlas between Disease Section and Healthy Individual Tissue

References

1	FDA Approved Drug Products from FDA Official Website. 2023. Application Number: 218276
2	ClinicalTrials.gov (NCT04578834) Study of Efficacy and Safety of LNP023 in Primary IgA Nephropathy Patients (APPLAUSE-IgAN). U.S. National Institutes of Health.
3	Discovery of 4-((2 S,4 S)-4-Ethoxy-1-((5-methoxy-7-methyl-1 H-indol-4-yl)methyl)piperidin-2-yl)benzoic Acid (LNP023), a Factor B Inhibitor Specifically Designed To Be Applicable to Treating a Diverse Array of Complement Mediated Diseases. J Med Chem. 2020 Jun 11;63(11):5697-5722.