Iptacopan (hydrochloride hydrate)

Iptacopan (LNP023) hydrochloride hydrate is an effective and orally-active highly selective factor B inhibitor with an IC50 value of 10 nM and KD of 7.9 nM. Iptacopan hydrochloride hydrate exerts a proximal effect in the complement cascade reaction, preventing the destruction (hemolysis) of red blood cells in PNH and the damage of renal cells in IgAN and C3G. Iptacopan hydrochloride hydrate can be used for the study of complement-mediated diseases, particularly paroxysmal nocturnal hemoglobinuria (PNH), primary immunoglobulin A nephropathy (IgAN), and complement 3 glomerulopathy (C3G) [1][2][3].

Product Specifications

CAS Number

[2447007-60-3]

Product Name Alternative

LNP023 (hydrochloride hydrate)

UNSPSC

12352005

Target

Complement System

Related Pathways

Immunology/Inflammation

Field of Research

Metabolic Disease; Inflammation/Immunology

Smiles

CC(C=C1OC)=C(NC=C2)C2=C1CN3[C@H](C4=CC=C(C=C4)C(O)=O)C[C@H](CC3)OCC.Cl.O

Molecular Formula

C25H33ClN2O5

Molecular Weight

476.99

References & Citations

[1]Dimitrios C Mastellos, et al. Expanding Complement Therapeutics for the Treatment of Paroxysmal Nocturnal Hemoglobinuria. Semin Hematol. 2018 Jul;55 (3) :167-175.|[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.|[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.