BioCryst Pharmaceuticals Inc. (NASDAQ: BCRX) has announced that it has enrolled the first subject in its RENEW proof-of-concept basket trial with its oral Factor D inhibitor, BCX9930, in C3 glomerulopathy, primary membranous nephropathy, and immunoglobin A nephropathy patients.
RENEW study to evaluate BCX9930’s tolerability and efficacy
RENEW is multicenter open-label proof-of-concept research aiming to assess BCX9930’s (500 mg bid) tolerability, safety, and therapeutic potential given for 24 weeks to 42 adult subjects with C3G, IgAN, or PMN (14 patients per disease)According to validation of diagnostic and clinical signs in a previous kidney biopsy, all subjects will be recruited in one of three parallel trial groups and will be given BCX9930 for the 24 weeks therapy duration. The main RENEW objective is the Percentage change in the 24-hour urine protein-to-creatinine ratio (uPCR) from baseline, as measured after week 24.
Chief medical officer of BioCryst William Sheridan stated, “There are currently no targeted treatments available for patients living with these rare renal diseases, which underscores the importance of the RENEW study. We believe an oral Factor D inhibitor could be a groundbreaking therapeutic option for nephritis conditions that are driven by the alternative pathway of complement. Coupled with our recent initiations of the REDEEM-1 and REDEEM-2 pivotal trials in PNH, we are excited to advance our BCX9930 clinical program across four separate complement-mediated diseases.”
BioCryst evaluating BCX9930 in PNH
BioCryst recently disclosed that the REDEEM-1 and REDEEM-2 pivotal studies testing BCX9930 for individuals with PNH had begun recruiting participants. REDEEM-1 is an open-label, randomized, active comparator-controlled trial evaluating the safety and efficacy of ingesting BCX9930 alone in PNH patients who have failed to respond to a C5 inhibitor. REDEEM-2 is a randomized, double-blind, placebo-controlled trial that compares oral BCX9930’s safety and efficacy with placebo in PNH individuals who aren’t on complement inhibitor medication.
Within the next two years, BioCryst intends to enhance and broaden its Factor D program by finalizing REDEEM 1 & 2 data reporting and submitting regulatory approval applications in PNH.