Vertex has announced several important advancements across its programmes in immunoglobulin A nephropathy (IgAN), APOL1-mediated kidney disease (AMKD) and autosomal dominant polycystic kidney disease (ADPKD). These updates represent significant progress toward reaching the company’s goal of bringing forward first-in-class or best-in-class therapies that target the underlying cause of these serious kidney diseases.
Povetacicept in IgAN
Vertex announced today that the Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to povetacicept (pove) for the treatment of IgAN. Pove is an investigational recombinant fusion protein therapeutic and dual antagonist of the BAFF (B cell activating factor) and APRIL (a proliferation inducing ligand) cytokines with best-in-class potential in IgAN and other B cell-driven diseases. BTD is for a drug that treats a serious condition, and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on one or more clinically significant endpoints over existing treatments.
Pove is currently being studied in Rainier, a global phase 3 clinical trial in patients with IgAN. The study is designed to have a pre-planned interim analysis (IA) evaluating urine protein to creatinine ratio (UPCR) for the pove arm versus placebo after a specified number of patients reach 36 weeks of treatment. The IA cohort has been fully enrolled, and Vertex remains on track to file for accelerated approval in the US in H1 2026 if results are supportive.
Inaxaplin in AMKD
Vertex also announced today enrolment completion of the IA cohort of Amplitude, a global phase 2/3 clinical trial designed to assess the impact of inaxaplin on kidney function and proteinuria for people living with AMKD. Inaxaplin is a first-in-class, investigational small molecule inhibitor of APOL1 aimed at addressing the underlying cause of AMKD. The Amplitude study is designed with an interim analysis at week 48 in a pre-specified number of patients to assess percent change from baseline in proteinuria and eGFR slope in the inaxaplin arm versus placebo. If positive, the IA will serve as the basis for Vertex to seek accelerated approval of inaxaplin in the US.
