ION532 (formerly IONIS-AZ5-2.5Rx ), also known as AZD2373, is an investigational medicine designed to reduce the production of apolipoprotein L1 (APOL1) for the treatment of APOL1-associated chronic kidney disease (CKD). Genetic studies have shown that two risk alleles (termed G1 and G2) are highly associated with nondiabetic CKD and increased rate of progression towards kidney failure, providing an exciting opportunity for personalized medicine in CKD. These two variants are more frequently found in individuals of West-African ancestry. Reduction of APOL1 with ION532 in a preclinical mouse model of APOL1-related kidney disease demonstrated significantly reduced proteinuria, a hallmark of CKD. ION532 is being developed for the treatment of APOL1-associated nephropathies.
About Chronic Kidney Disease
CKD is a worldwide public health problem, affecting about 10% of the population. Within United States, over 37 million adults have CKD and millions of others are at increased risk. CKD may be caused by diabetes, high blood pressure and other disorders. Without treatment, CKD may progress and eventually lead to kidney failure, which requires dialysis or a kidney transplant to maintain life.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.