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Research & Development

Tenacity

At Akebia, we are inspired to think boldly and move bold thinking into action. We leverage our scientific expertise and this innovative thinking to develop clinical advances in areas that are important to people living with kidney disease.

We thrive as collaborators because we believe that we can go further together. We work with partners across the globe to pioneer and grow new areas of research and development.

We are optimistic and want to have a positive impact. Each day we bring our drive to life with the work we do.

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 alternative treatment options can broaden access to more patients with anemia. 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

Innovating to Protect the Kidneys

Akebia’s research and development team has deep experience in investigating oral hypoxia-inducible factor prolyl hydroxylase inhibitors (HIF-PHIs) and the Nobel Prize-winning science behind them. 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. 

Clinical Development

If you would like to learn more about our clinical trials, including becoming a participating  investigator or referring physician, please email [email protected] or visit www.clinicaltrials.gov

Akebia also seeks to partner and collaborate with healthcare professionals and researchers to investigate scientific hypotheses or questions related to our products and therapeutic areas of interest. Visit our External Sponsored Research page to learn more. 

 

Anemia due to CKD and Beyond

Leveraging our HIF expertise, we are focused on continuing our research in anemia due to CKD and working to identify and initiate development planning for other programs where HIF-PHIs 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, collaborations 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. 

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  (v4.0)   06/23

Akebia Therapeutics, Inc.
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Cambridge, MA 02142

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