Benzinga | Nov 29, 2021 08:31AM EST

BioCryst Begins Patient Enrollment In REDEEM-2 Pivotal Trial Evaluating BCX9930 As Oral Monotherapy For Patients With PNH

BioCryst Pharmaceuticals, Inc. (NASDAQ:BCRX) today announced the enrollment of the first patient in the REDEEM-2 pivotal trial with its oral Factor D inhibitor, BCX9930, in patients with paroxysmal nocturnal hemoglobinuria (PNH).



REDEEM-2 is a randomized, placebo-controlled trial evaluating the efficacy and safety of BCX9930 (500 mg bid) as monotherapy versus placebo in approximately 57 PNH patients not currently receiving complement inhibitor therapy. In part 1 of this trial, patients will be randomized 2:1 to receive BCX9930 or placebo under double-blind conditions for 12 weeks. All patients will receive BCX9930 in part 2 (weeks 13-52) to assess the long-term safety, tolerability and effectiveness of BCX9930, with patients randomized to placebo in part 1 switching to BCX9930 at the week 12 visit. The primary endpoint of REDEEM-2 is change from baseline in hemoglobin, as assessed at week 12.

"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," said Dr. William Sheridan, chief medical officer of BioCryst.

"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," Sheridan added.

REDEEM-2 is powered at 90 percent to detect a difference in mean change from baseline of hemoglobin of ? 2.15 g/dL at 12 weeks. In a dose-ranging trial of BCX9930 in treatment-na?ve patients, the company previously reported that BCX9930 (at doses of 400 mg or 500 mg bid) increased hemoglobin from baseline by a mean of 3.7 g/dL at week 12 and eliminated transfusions. BCX9930 was safe and generally well-tolerated in the trial.

BioCryst also is preparing to enroll patients in the REDEEM-1 pivotal trial, a randomized, open-label, active, comparator-controlled comparison of the efficacy and safety of BCX9930 (500 mg bid) monotherapy in approximately 81 PNH patients with an inadequate response to a C5 inhibitor. Additionally, the company is initiating a proof-of-concept trial of BCX9930 in renal complement-mediated diseases.

The U.S. Food and Drug Administration has granted both Fast Track status and Orphan Drug Designation to BCX9930 for PNH. For more information about REDEEM-2, visit ClinicalTrials.gov and search NCT number NCT05116787.