BioCryst Pharmaceutical Inc. (NASDAQ: BCRX) has announced the enrolment of its first patient in the REDEEM-2 vital study with the Oral Factor D inhibitor BCX9930 in paroxysmal nocturnal hemoglobinuria patients.
REDEEM-2 Study to evaluate safety and efficacy of BCXX9930
The REDEEM-2 study will be a double-blind, randomized, placebo-controlled trial that compares the safety and efficacy of BCX9930 (500 mg bid) compared to placebo in 57 PNH patients who aren’t on complement inhibitor therapy. Notably, in the first phase of this study, patients will be randomized to 2:1 and given BCX9930 or a placebo for 12 weeks under double-blind settings. Then, in part 2 (weeks 13-52), all patients will receive BCX9930 to examine the drug’s long-term tolerability, safety, and effectiveness, with patients who were randomized to placebo in part 1 switching to BCX9930 during their 12th-week visit. REDEEM-2’s primary objective is hemoglobin change from baseline, as measured at week 12.
Chief Medical Officer William Sheridan said, “There are important unmet needs among patients living with PNH based on the current standard of care, specifically related to efficacy and burden of therapy. As an oral monotherapy with proof-of-concept data showing control of both intravascular and extravascular hemolysis, BCX9930 has the potential to help patients live their lives with more freedom from their disease.”
“Beginning patient enrollment in our REDEEM-2 pivotal trial marks a critical step forward as BCX9930 advances closer to registration for patients living with PNH,” added Sheridan.
BioCryst to enroll patient in REDEEM 1 study
The REDEEM-1 pivotal trial, an open-label, randomized, active, comparator-controlled assessment of the safety and efficacy of BCX9930 (500 mg bid) monotherapy in around 81 PNH patients with an inadequate response to a C5 inhibitor, is also getting ready to enroll participants. In addition, BCX9930 is being tested in a proof-of-concept experiment in renal complement-mediated disorders.
BCX9930 for PNH has been given both Fast Track and Orphan Drug Designation by the US Food and Drug Administration. The company previously reported that BCX9930 increased hemoglobin from baseline by a mean of 3.7 g/dL at week 12 and eliminated transfusions in treatment-naive patients.
