Chemistry: Generation 2.5
ION736 (formerly IONIS-AZ7-2.5Rx), also known as AZD8701, is an investigational antisense medicine designed to reduce the production of Forkhead Box P3 (FOXP3) for the treatment of patients with cancer. FOXP3 is a protein involved in the function of immuno-suppressive T regulatory cells (Tregs). Tregs, which are found at high levels in various types of cancers, often predict poor survival and poor response to immune checkpoint therapeutics. Preclinical studies have demonstrated that FOXP3 downregulation resulted in an increased immune response and anti-tumor activity. Moreover, the combination of antisense inhibition of FOXP3 with other immune-oncology drugs led to enhanced anti-tumor activity.
Safety and efficacy have not been evaluated by any regulatory authorities for the indications described.