Benzinga | Mar 22, 2021 08:01AM EDT

BioCryst's Oral Factor D Inhibitor, BCX9930, Advancing to Pivotal Trials in PNH Following Successful Proof of Concept Trial

--Following doses of 400 mg bid or 500 mg bid of oral BCX9930, 100 percent of treatment-na?ve patients and 83 percent of C5 inadequate response patients were transfusion-free--

--Mean hemoglobin increased from 8.3 g/dL to 11.8 g/dL in treatment-na?ve patients and from 8.9 g/dL to 12.2 g/dL in C5 inadequate response patients, demonstrating control of hemolysis--

--Pivotal trials in PNH and proof of concept trials in renal complement-mediated disease expected to begin in 2H 2021 --

RESEARCH TRIANGLE PARK, N.C., March 22, 2021 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced that its oral Factor D inhibitor, BCX9930, significantly increased hemoglobin and reduced transfusions in an ongoing dose-ranging trial in treatment-na?ve (no prior treatment with C5 inhibitors) paroxysmal nocturnal hemoglobinuria (PNH) patients, and in PNH patients with an inadequate response to C5 inhibitors. BCX9930 was safe and generally well-tolerated in the trial.

Based on these results, and recent interactions with U.S. and European regulators, the company plans to advance directly into pivotal trials in PNH and proof of concept trials in renal complement-mediated diseases in the second half of 2021.

PNH patients in the trial also experienced reductions in key laboratory biomarkers, such as reticulocyte count, lactate dehydrogenase (LDH) (treatment-na?ve patients) and percentage of C3 opsonization (patients with inadequate C5 response) following dosing at 400 mg bid or 500 mg bid.

"The significant reduction in transfusions and increases in hemoglobin seen in this trial with an oral medicine address an unmet need for patients and physicians -- a PNH therapy that can maximize hematological benefit through the control of both intravascular and extravascular hemolysis," said Antonio Risitano, M.D., Ph.D., San Giuseppe Moscati Hospital, Avellino, Italy, and principal investigator of the trial.

"I am very encouraged by these results which may position proximal inhibitors, and in particular this oral anti-Factor D agent, as medications changing the treatment paradigm of PNH, and possibly of other alternative pathway mediated diseases," he added.