Business
BioCryst’s Oral Factor D Inhibitor, BCX9930, Shows Clinical Benefit as Monotherapy Through 400 mg bid in Treatment-naïve PNH Patients
RESEARCH TRIANGLE PARK, N.C., Sept. 30, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced new data from treatment-naïve
About this update from Biocryst Pharmaceuticals, Inc.
[{"type":"text","content":"RESEARCH TRIANGLE PARK, N.C., Sept. 30, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq:BCRX) today announced new data from treatment-naïve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients receiving doses through 400 mg bid of its oral Factor D inhibitor, BCX9930, as monotherapy in an ongoing dose-ranging trial.\n Oral BCX9930 is driving rapid and dose-dependent reductions in key biomarkers, including LDH, and increasing hemoglobin levels in all PNH patients in the trial. Increases in hemoglobin levels were maintained without transfusions. BCX9930 has been safe and well tolerated at all doses in the trial. No drug-related serious adverse events have been reported. “We are thrilled with the clinical benefits and safety profile of oral BCX9930 monotherapy that we continue to see in PNH patients with dosing up to 400 mg bid. With these excellent results, we plan to initiate advanced development trials next year in multiple complement-mediated hematology and nephrology diseases,” said Dr. William Sheridan, chief medical officer of BioCryst. The FDA has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. BioCryst has confirmed meetings with regulators in the 4th quarter of 2020 to discuss the advanced development program for BCX9930. Updated Data Through 400 mg bid All seven PNH patients in the trial were severely ill, with pre-treatment LDH from 3.8 to 11 × ULN, indicating active hemolysis. Four patients had a history of compromised bone marrow function, and two had thrombotic, lung or kidney complications from PNH.New data from the four treatment-naïve PNH patients who have received more than six weeks of therapy at 400 mg bid show significant clinical benefits for these patients.º Hemoglobin levels increased by a mean of 3.8 g/dL from baseline. These increases are being maintained without transfusions.º Three of four patients have responded with hemoglobin levels >11 g/dL to date. Hemoglobin in the fourth patient, who has compromised bone marrow function from aplastic anemia PNH, responded with an increase from 6 g/dL at baseline to 9.5 g/dL on treatment.º In all four patients, the size of the PNH red blood cell clone approached that of the PNH granulocyte clone, indicating near-complete control of complement-mediated hemolysis. The mean relative ...