Benzinga | Sep 30, 2020 07:10AM EDT

BioCryst Highlights Data Showing Its Oral Factor D Inhibitor, BCX9930, 'Shows Clinical Benefit as Monotherapy Through 400 mg bid in Treatment-nave PNH Patients'

RESEARCH TRIANGLE PARK, N.C., Sept. 30, 2020 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced new data from treatment-na?ve (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.

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-na?ve PNH patients who have received more than six weeks of therapy at 400 mg bid show significant clinical benefits for these patients. ? Hemoglobin levels increased by a mean of 3.8 g/dL from baseline. These increases are being maintained without transfusions. ? 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. ? 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 (red blood cells/granulocytes) PNH red blood cell clone size increased from 48 percent at baseline to 94 percent on treatment. ? All four patients have shown reductions in LDH. Three of four patients show average serial LDH of <1.5? ULN. In the fourth patient, the LDH has decreased from a pretreatment baseline of 11 ? ULN to 2.2 ? ULN on treatment to date. ? All four patients have continued on therapy with BCX9930 based on the investigators' assessment of clinical benefit.

* The most common adverse event was mild to moderate headache lasting one to three days. One patient had a mild rash that resolved after continued BCX9930 dosing at 100mg bid. One patient had a mild rash at 200 mg bid that is resolving during uninterrupted dosing after dose escalation to 400 mg bid.

In addition to the ongoing dose-ranging trial in treatment-na?ve PNH patients, the company plans to report data from PNH patients with an inadequate response to C5 inhibitors receiving at least 400 mg bid of BCX9930 by the end of 2020.

Additional details can be found on slides, which can be accessed at the Investors' section of BioCryst's website at http://www.biocryst.com.