Iptacopan hydrochloride

• CAT Number: I014978
• CAS Number: 1646321-63-2
• Molecular Formula: C25H31ClN2O4
• Molecular Weight: 458.98
• Purity: 98%
• Synonyms: 4-[(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl]benzoic acid;hydrochloride
• Target: CFB
• Target Protein: P00751
• Appearance: Solid
• Storage: Dry, dark and at 2 - 8 °C for six months or -20°C for two years.
• IC50: KD: 7.9 nM (factor B)IC50: 10 nM (factor B)
• InChI: InChI=1S/C25H30N2O4.ClH/c1-4-31-19-10-12-27(22(14-19)17-5-7-18(8-6-17)25(28)29)15-21-20-9-11-26-24(20)16(2)13-23(21)30-3;/h5-9,11,13,19,22,26H,4,10,12,14-15H2,1-3H3,(H,28,29);1H/t19-,22-;/m0./s1
• InChIKey: SEZXOFFLNHXEJE-CQERKEQDSA-N
• SMILES: CCO[C@H]1CCN([C@@H](C1)C2=CC=C(C=C2)C(=O)O)CC3=C(C=C(C4=C3C=CN4)C)OC.Cl

Iptacopan hydrochloride（Cat No.:I014978）is a first-in-class oral factor B inhibitor designed to target the alternative complement pathway, providing therapeutic benefits in conditions like paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy. By selectively inhibiting factor B, iptacopan reduces excessive complement activation, which is linked to the destruction of red blood cells and kidney damage in these diseases. Its oral administration offers a more convenient treatment option compared to existing therapies. Iptacopan hydrochloride is currently being studied in clinical trials for its potential to improve patient outcomes in complement-mediated disorders.

Reference

[1]. Dimitrios C Mastellos, et al. Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Semin Hematol. 2018 Jul;55(3):167-175.
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[2]. Anna Schubart, et al. Small-molecule Factor B Inhibitor for the Treatment of Complement-Mediated Diseases. Proc Natl Acad Sci U S A. 2019 Apr 16;116(16):7926-7931.
[Content Brief]

[3]. Nello Mainolfi, et al. 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.
[Content Brief]