BioCryst begins patient enrollment in REDEEM-1 trial of BCX9930 for rare blood disorder

ExperienceInteriors/E+ via Getty Images

• BioCryst Pharmaceuticals (BCRX -0.2%) said it enrolled the first patient in the REDEEM-1 pivotal trial of BCX9930 as standalone therapy for patients with paroxysmal nocturnal hemoglobinuria (PNH).
• PNH is a rare blood disorder characterized by destruction of red blood cells, blood clots, and impaired bone marrow function.
• REDEEM-1 will evaluate the efficacy and safety of BCX9930 (500 mg bid) monotherapy in ~81 PNH patients with an inadequate response to a C5 inhibitor.
• The company said that in a dose-ranging trial of BCX9930 in C5 inadequate response patients, BCX9930 (at doses of 400 mg or 500 mg bid) increased hemoglobin from baseline by a mean of 2.7 g/dL through weeks 12 to 24 with 80% of patients being transfusion-free over the same period. The drug was safe and generally well-tolerated in the trial.
• The U.S. Food and Drug Administration has granted the fast track status and orphan drug designation to BCX9930 for PNH.