MENU Research & Development Why Anemia? / Innovating to Protect the Kidneys / Clinical Development / Other Research Programs Why Anemia? Anemia is a condition in which a person does not have enough healthy red blood cells to carry adequate oxygen to the body’s tissues. It can commonly occur in people with chronic kidney disease (CKD) because their kidneys do not produce enough erythropoietin (EPO), which is a hormone released into the blood to help regulate the production of red blood cells. Anemia affects approximately 5.7 million people with CKD in the U.S. alone. Left untreated, anemia deteriorates patient health and is associated with increased morbidity and mortality in people with CKD. Injectable erythropoiesis-stimulating agents (ESAs) have been the standard of care for treating anemia due to CKD in both dialysis dependent and non-dialysis dependent patients since the early 1990s. While there are treatment options, unmet needs remain and undertreatment continues to be a challenge. We believe new treatment options for anemia are not only needed, but also possible. Here at Akebia, we are leading a change by working to advance innovative therapies to better the lives of people living with kidney disease. 5.7 million Approximately number* of people with CKD affected by anemia in the U.S. alone. *Based on third party prevalence data and company estimates Our lead product candidate, vadadustat, is part of a new class of investigational agents called oral hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHIs), which are based on Nobel Prize-winning science. HIF-PHIs are designed to mimic the body’s response to lower levels of oxygen, such as when a person is at high altitude. The body naturally responds to lower oxygen levels by increasing the availability of HIF, which is a protein that coordinates the expression of the genes responsible for erythropoietin synthesis and the regulation of iron metabolism. Inhibition of hypoxia-inducible factor prolyl hydroxylase (HIF-PH) can lead to increased red blood cell production and improved oxygen delivery to tissues. The discovery of HIF has laid the foundation to help understand the central role of oxygen sensing in many diseases, including anemia due to CKD. Following the completion of our global Phase 3 clinical development program for vadadustat, we have now filed a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for U.S. regulatory approval of vadadustat for the treatment of anemia due to CKD in adult patients on dialysis and adult patients not on dialysis. Within Japan, VafseoTM (vadadustat) is approved as a treatment for anemia due to CKD in both dialysis-dependent and non-dialysis dependent adult patients. Clinical Development Vadadustat is an oral HIF-PHI in clinical development for the treatment of anemia due to CKD in dialysis dependent and non-dialysis dependent adult patients. We are evaluating the safety and efficacy of vadadustat in two global Phase 3 programs – INNO2VATE (dialysis) and PRO2TECT (non-dialysis). Vadadustat is an investigational drug that has not been approved by the U.S. Food and Drug Administration (FDA) In 2020, we announced results from the INNO2VATE program for vadadustat in adult patients on dialysis (incident and prevalent dialysis patients). Additional information is available here: https://ir.akebia.com/news-releases/news-release-details/akebia-presents-results-its-inno2vate-global-phase-3-program. We also announced results from the PRO2TECT program for vadadustat in adult patients not on dialysis. Additional information is available here: https://ir.akebia.com/news-releases/news-release-details/akebia-presents-results-its-pro2tect-global-phase-3-program. For the latest Akebia corporate presentation, please visit Akebia’s Investors page. If you would like to learn more about our clinical trials, including becoming a participating investigator or referring physician, please email trials@akebia.com or visit www.clinicaltrials.gov, and input “vadadustat” into the “Other terms” search bar. VADADUSTAT EXPANDED ACCESS POLICY Anemia due to CKD and Beyond Leveraging our HIF expertise, we are focused on the development of vadadustat for the treatment of anemia due to CKD and are working to identify and initiate development planning for other programs where vadadustat may have therapeutic benefits. We also aim to add to our pipeline and portfolio of novel therapeutics through internal discovery and development, and through strategic transactions, such as in-licenses, collabroations and acquisitions. In addition, given our expertise in research and development, we believe there may be opportunities to leverage these assets and establish mutually beneficial relationships with other companies that are looking to enter the renal market or attempting to develop renal therapeutics. Vadadustat is an investigational drug that has not been approved by the U.S. Food and Drug Administration (FDA) or any regulatory authority for use outside of Japan. BIBLIOGRAPHY 1. National Institute of Diabetes and Digestive and Kidney Diseases. Anemia in Chronic Kidney Disease. Available at: https://www.niddk.nih.gov/health-information/kidney-disease/anemia. Accessed: September 20, 2019. 2. Stauffer ME, Fan T. Prevalence of Anemia in Chronic Kidney Disease in the United States. PLoS One 2014;9(1):e84943. DOI:10.1371/journal.pone.0084943. 3. Portoles J, Gorriz JL, Rubio E, et al. The development of anemia is associated to poor prognosis in NKF/KDOQI stage 3 chronic kidney disease. BMC Nephrol. 2013;14:2. DOI: 10.1186/1471-2369-14-2. 4. Kidney Disease: Improving Global Outcomes (KDIGO) Work Group. KDIGO Clinical Practice Guideline for Anemia in Chronic Kidney Disease. Kidney Int Suppl 2012;2(4):279–335. 5. Nakhoul G, Simon JF. Anemia of chronic kidney disease: Treat it, but not too aggressively. Cleve Clin J Med 2016;83(8):613-624. DOI: 10.3949/ccjm.83a.15065. 6. Besarab A, Bolton WK, Browne JK, et al. The effects of normal as compared with low hematocrit values in patients with cardiac disease who are receiving hemodialysis and epoetin. N Engl J Med 1998;339(9):584–590. 7. Singh AK, Szczech L, Tang KL, et al; CHOIR Investigators. Correction of anemia with epoetin alfa in chronic kidney disease. N Engl J Med 2006;355(20):2085–2098. 8. Drüeke TB, Locatelli F, Clyne N, et al; CREATE Investigators. Normalization of hemoglobin level in patients with chronic kidney disease and anemia. N Engl J Med 2006; 355(20):2071–2084. 9. Pfeffer MA, Burdmann EA, Chen CY, et al; TREAT Investigators. A trial of darbepoetin alfa in type 2 diabetes and chronic kidney disease. N Engl J Med 2009; 361(21):2019–2032. DOI: 10.1056/NEJMoa0907845. 10. Eltzschig HK, Bratton DL, Colgan SP. Targeting hypoxia signalling for the treatment of ischaemic and inflammatory diseases. Nat Rev Drug Discov 2014;13(11):852-869. DOI: 10.1038/nrd4422. 11. Haase VH. Hypoxia-inducible factors in the kidney. Am J Physiol Renal Physiol 2006;291(2):F271-281. 12. Majmundar AJ, Wong WJ, Simon MC. Hypoxia-inducible factors and the response to hypoxic stress. Mol Cell 2010;40(2):294-309. DOI: 10.1016/j.molcel.2010.09.022. 13. U.S. National Library of Medicine. Efficacy and Safety Study to Evaluate Vadadustat for the Correction of Anemia in Subjects With Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD) (PRO2TECT). Available at: https://clinicaltrials.gov/ct2/show/NCT02648347. Accessed: September 20, 2019. 14. U.S. National Library of Medicine. Efficacy and Safety Study to Evaluate Vadadustat for the Maintenance Treatment of Anemia in Subjects With Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD) (PRO2TECT). Available at: https://clinicaltrials.gov/ct2/show/NCT02680574. Accessed: September 20, 2019. 15. U.S. National Library of Medicine. Efficacy and Safety Study to Evaluate Vadadustat for the Correction of Anemia in Subjects With Non-dialysis-dependent Chronic Kidney Disease (NDD-CKD) (PRO2TECT). Available at: https://clinicaltrials.gov/ct2/show/NCT02648347. Accessed: September 20, 2019. 16. U.S. National Library of Medicine. Efficacy and Safety Study to Evaluate Vadadustat for the Correction or Maintenance Treatment of Anemia in Subjects With Incident Dialysis-dependent Chronic Kidney Disease (DD-CKD) (INNO2VATE). Available at: https://clinicaltrials.gov/ct2/show/NCT02865850. Accessed: September 20, 2019. 17. U.S. National Library of Medicine. Efficacy and Safety Study to Evaluate Vadadustat for the Maintenance Treatment of Anemia in Subjects With Dialysis-dependent Chronic Kidney Disease (DD-CKD) (INNO2VATE). Available at: https://clinicaltrials.gov/ct2/show/NCT02892149. Accessed: September 20, 2019. BC-0016 (v2.0) 4/21 Akebia Therapeutics, Inc. 245 First Street, Suite 1400 Cambridge, MA 02142 Driving Directions +1 617.871.2098 phone +1 617.871.2099 fax Contact Us © 2021 Akebia Therapeutics, Inc. All rights reserved. AURYXIA®, AKEBIA® Therapeutics and their associated logos are trademarks of Akebia and/or its affiliates. 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