Drug intelligence / Profile preview

HSK39297

Development stage
Phase 3
Lead developer
Haisco Pharmaceutical Group
Modality
Small Molecules
Administration
Oral
01

Overview

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].

Other names
[14C]HSK39297Carbon-14 labeled HSK39297Carbon14 labeled HSK39297Carbon 14 labeled HSK39297
02

Targets

CFB (Complement Factor B)

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