Arrowhead applies for clearance to begin Phase 1/2a trial of RNAi therapeutic

Arrowhead Pharmaceuticals announced that it has filed an application for clearance to initiate a Phase 1/2a clinical trial of ARO-CFB, the company’s investigational RNA interference, RNAi, therapeutic being developed as a potential treatment for complement mediated renal disease, such as immunoglobulin A nephropathy IgAN , which is the most common glomerular disease worldwide and carries a high lifetime risk of progression to end-stage renal disease. Additionally, ARO-CFB may have clinical applications in non-renal diseases involving complement activation. ARO-CFB is designed to reduce hepatic expression of complement factor B CFB , which plays an important regulatory role in amplifying complement alternative pathway activation and has been identified as a promising therapeutic target. James Hamilton, M.D., MBA, Chief of Discovery and Translational Medicine at Arrowhead, said: “IgAN is the most common glomerular disease worldwide, accounting for up to 40% of all cases of glomerulonephritis. …we believe ARO-CFB has the potential to be a powerful treatment option for complement mediated renal disease.” An application for approval of the clinical trial was submitted to a local Ethics Committee and to the New Zealand Medicines and Medical Devices Safety Authority for review by the Standing Committee on Therapeutic Trials.