BioCryst Outlines Pivotal Trial Designs For BCX9930 In Rare Blood Disorder

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.

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