BioCryst Outlines Pivotal Trial Designs For BCX9930 In Rare Blood Disorder

BioCryst Pharmaceuticals Inc BCRX has announced the designs for REDEEM-1 and REDEEM-2, two upcoming pivotal trials for its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH).
REDEEM-1 will compare the efficacy and safety of BCX9930 monotherapy versus C5 inhibitor in approximately 81 PNH patients.
REDEEM-2 will evaluate the efficacy and safety of BCX9930 as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy.
The primary endpoint for both trials is the change from baseline in hemoglobin (Hb), assessed at weeks 12 to 24 in REDEEM-1 and at week 12 in REDEEM-2.
Key secondary endpoints for both trials are the proportion of transfusion-free subjects, the number of units of packed red blood cells (pRBC) transfused, and change from baseline in FACIT-Fatigue score.
Enrollment will start in the second half of 2021.
Price Action: BCRX shares are down 0.32% at $15.58 during the market session on the last check Thursday.