IOA-360

IOA-360 is a novel, highly selective small molecule inhibitor of the Transforming Growth Factor-beta (TGF-β) receptor I kinase, also known as ALK5. Developed by iOnctura, it is designed to address bone marrow failure and anemia associated with overactivation of the TGF-β superfamily in conditions such as Myelodysplastic Syndromes (MDS) and Acute Myeloid Leukemia (AML). By blocking ALK5, IOA-360 inhibits the phosphorylation and activation of downstream SMAD2/3 proteins, which are known to suppress erythropoiesis. Preclinical studies have demonstrated that IOA-360 promotes erythroid colony formation and differentiation in primary MDS patient samples, both as a monotherapy and in combination with luspatercept.