BioCryst Pharmaceutical Inc. (NASDAQ: BCRX) has announced the enrolment of the first subject in the REDEEM-1 pivotal study with BCX9930, its oral Factor D inhibitor, in paroxysmal nocturnal hemoglobinuria (PNH) patients.
REDEEM to assess BCX9930 as monotherapy in PNH
REDEEM-1 is an open-label, randomized, active comparator-controlled study that looks at the safety and effectiveness of BCX9930 (500 mg bid) monotherapy in 81 PNH patients who had not responded well to a C5 inhibitor. Patients who have not had an adequate reaction to a C5 inhibitor will be randomized 2:1 to stop taking their C5 inhibitor and take BCX9930 as monotherapy or to keep taking their C5 inhibitor for another 24 weeks in part 1 of this trial. In part 2 (weeks 25-52) of the study, all subjects will receive BCX9930 to analyze the drug’s long-term safety, tolerability, and efficacy.
BioCryst Chief Medical Officer William Sheridan said, “On the heels of recently beginning enrollment in our REDEEM-2 pivotal trial, today’s announcement marks another important milestone as we advance BCX9930 closer to registration for patients living with PNH. Given the unmet need patients have related to the current standard of care, we aim to demonstrate in the REDEEM-1 pivotal trial the potential of BCX9930 as an oral monotherapy that could represent significant improvement for patients compared to their experiences with C5 inhibitor therapies.”
ORLADEYO reports full-year 2021 ORLADEYO revenue of $122 million
Also, the company has announced that its fourth-quarter and full-year 2021 ORLADEYO revenue was $45.6 million and $122 million, respectively. For full-year 2022 the company expects ORLADEYO net revenue to be $250 million. BioCryst anticipates ORLADEYO to be a market leader in HAE prophylaxis treatment with sales of $1 billion.
CEO BioCryst Jon Stonehouse said, “Building on our substantial 2021 patient base, we are confident that ORLADEYO will achieve no less than $250 million of net revenue in 2022 and that ORLADEYO will become the market leader as the most prescribed prophylactic therapy with peak sales of $1 billion.”
