BioCryst begins proof of concept trial in PNH patients with oral Factor D inhibitor, BCX9930

BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today 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.

Study Design

In this proof of concept study of the safety and effectiveness of oral BCX9930, up to 16 PNH patients will receive BCX9930 twice daily (BID) for 28 days. Patients in the first cohort will receive 50 mg BID for 14 days, then 100 mg BID for 14 days, for a total of 28 days. For patients in the second cohort, the planned dosing regimen is 200 mg BID for 14 days, followed by 400 mg BID for 14 days. Each cohort of up to eight patients can enroll up to four treatment-naïve patients and up to four poor responders to eculizumab or ravulizumab. Key markers of effectiveness to be evaluated include levels of LDH, hemoglobin and reticulocytes. BID treatment will continue for subjects who show benefit, such as improvement in LDH levels or hemoglobin.  Safety will be monitored clinically and with laboratory tests.

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