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MY008211 is an investigational, orally bioavailable small molecule inhibitor of Complement Factor B, developed by Shanghai Meiyue Biotechnology Development Co., Ltd. It is primarily being evaluated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). By selectively targeting Factor B, a key serine protease in the alternative complement pathway, MY008211 prevents the formation of the C3 convertase (C3bBb). This action effectively blocks the amplification loop of the complement cascade, thereby inhibiting both intravascular hemolysis (mediated by the membrane attack complex) and extravascular hemolysis (mediated by C3b opsonization). The drug is currently in Phase 2 clinical development for PNH and has undergone Phase 1 drug-drug interaction studies with clopidogrel and rifampin.
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