Pelecopan

Pelecopan (BCX9930) is a potent, selective, orally active inhibitor of complement factor D with an IC50 value of 14.3 nM. Pelecopan can target factor D to prevent both intravascular and extravascular hemolysis in PNH. Pelecopan also be used for other alternative pathway (AP) mediated diseases[1][2].

Signaling Pathways >> Immunology/Inflammation >> Complement System

Research Areas >> Inflammation/Immunology

IC50: 14.3 nM (Complement factor D)[1]

BCX9930 has a potent, highly specific inhibitory activity for purified human factor D with an IC50 value of 14.3 nM and also inhibits its proteolytic activity against factor B bound to C3b with an IC50 of 28.1 nM. BCX9930 completely blocks hemolysis of PNH cells in vitro (with an IC50 value of 29.5 nM in rabbit erythrocytes) and suppresses the accumulation of C3 fragments on PNH erythrocytes. BCX9930 suppresses AP activity in rhesus monkeys ex vivo assays[1][3].

[1]. International Nonproprietary Names for Pharmaceutical Substances (INN)

[2]. AustinKulasekararajMD MRCP, FRCPath, et al. BCX9930, a Potent, Selective, Oral Factor D Inhibitor, Demonstrates Proof-of-Concept As Monotherapy in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH). Blood (2020) Volume 136, Supplement 1, 5 November 2020, Pages 14-15

[3]. XilinChenMDPhD, et al. Preclinical Characterization of BCX9930, a Potent Oral Complement Factor D Inhibitor, Targeting Alternative Pathway-Mediated Diseases Including Paroxysmal Nocturnal Hemoglobinuria (PNH). Blood (2020) Volume 136, Supplement 1, 5 November 2020, Page 32.