BioCryst Pharmaceuticals, Inc. announced that preclinical data on BCX9930, an oral Factor D inhibitor under development as monotherapy for paroxysmal nocturnal hemoglobinuria and other complement-mediated diseases, showed complete in vitro blockade of both hemolysis of PNH erythrocytes and accumulation of C3 fragments on PNH erythrocytes, indicating that BCX9930 monotherapy has the potential to inhibit both intravascular and extravascular hemolysis.
December 6, 2020
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