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HSK39297 is an orally administered small molecule drug developed by Tibet Haisco Pharmaceutical and Haisco Pharmaceutical Group. It acts as a complement factor B (CFB) inhibitor, targeting the alternative pathway of the complement system. The drug is being investigated primarily for paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic disorder characterized by uncontrolled complement activation leading to hemolysis, as well as for IgA nephropathy and C3 glomerulopathy. Clinical trials have reached phase 3 in PNH and phase 2 in IgA nephropathy. The mechanism of action involves inhibition of CFB, which plays a key role in amplifying the alternative pathway of complement activation, thereby reducing hemolysis and related symptoms in PNH patients[1][2][3][4].
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