Akebia Therapeutics, Inc. is a biopharmaceutical company focused on the developing and commercializing small molecules to treat anemia and other diseases. The Company’s lead program, AKB-6548, is in a Phase 2b clinical trial for anemia associated with chronic kidney disease (CKD).
AKB-6548 is an orally available, hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor that stabilizes HIF2α and is currently in development by Akebia for the treatment of anemias secondary to CKD and end stage renal disease (ESRD or dialysis). These diseases are currently treated with injectable erythropoiesis stimulating agents (ESAs), which generated approximately $8 billion in global revenues in 2011, despite having “black box” warnings for increased cardiovascular risk in patients with CKD and increased rate of tumor growth and chance of death in patients with cancer.
By contrast, due to its different mechanism of action, AKB-6548 has demonstrated the potential to be a safer, more efficacious, less expensive, orally dosed alternative to the injectable ESAs that are currently used to treat a variety of anemias. Instead of binding directly to and saturating the EPO receptor for prolonged periods of time, AKB-6548 acts by simulating the body’s natural response to hypoxia that is carried out by stabilization of HIF2α. The drug response is similar to the physiological adjustment made by the body to an increase in altitude. In this way, once-daily dosing of this oral HIF-PH inhibitor can restore the normal diurnal variation of EPO for a patient with anemia in a way that an injectable ESA cannot. This approach leads to a safe, consistent, predictable and controllable rise in hemoglobin levels.