BioCryst Begins Proof of Concept Trial in PNH Patients With Oral Factor D Inhibitor, BCX9930

BioCryst Pharmaceuticals, Inc. announced the first patients have been dosed with BCX9930 in a proof of concept trial in paroxysmal nocturnal hemoglobinuria (PNH). These patients were naïve to eculizumab and ravulizumab.

BCX9930 is an oral Factor D inhibitor discovered and developed by BioCryst for the treatment of complement-mediated diseases. The company expects to report data from the proof of concept study in PNH patients in the second quarter of 2020.

“In PNH, our goal for BCX9930 is to develop a safe, well-tolerated, oral monotherapy with excellent efficacy, whether or not patients have ever been treated with a C5 inhibitor,” said Dr. William Sheridan, chief medical officer of BioCryst.

“Based on the outstanding safety and PK/PD profile we have seen with BCX9930 in healthy subjects, and the historically excellent translation of complement markers into clinical results in PNH patients, we are confident that we will demonstrate proof of concept in PNH, and other alternative pathway complement-mediated diseases,” Sheridan added.

Because Factor D is essential for alternative pathway overactivity in PNH, and all other complement diseases of the alternative pathway, successful proof of concept data with BCX9930 would enable BioCryst to advance the program across other target indications.