CAMBRIDGE - Akebia Therapeutics, Inc. (Nasdaq: AKBA), today announced the presentation of clinical data from its PRO2TECT global Phase 3 program that evaluated the efficacy and safety of vadadustat, Akebia's investigational oral hypoxia-inducible factor prolyl hydroxylase inhibitor (HIF-PHI), versus darbepoetin alfa for the treatment of anemia due to chronic kidney disease (CKD) in adult patients not on dialysis, at American Society of Nephrology Kidney Week 2020 Reimagined (ASN Kidney Week).
As previously announced, vadadustat achieved the primary and key secondary efficacy endpoints in each of the two studies in the program, but did not meet the primary safety endpoint. Newly presented pre-specified regional analyses of the PRO2TECT program show vadadustat demonstrated no clinically meaningful increase in cardiovascular risk compared to darbepoetin alfa in analyses of MACE, expanded MACE and all-cause mortality in U.S. patients treated to a target hemoglobin (Hb) range of 10 to 11 g/dL, consistent with U.S. treatment guidelines.
'The PRO2TECT results show that orally administered vadadustat achieved both primary and secondary efficacy endpoints in patients not on dialysis with anemia associated with CKD. The newly presented analyses showed that there were regional differences with respect to MACE, expanded MACE and all-cause mortality, consistent with well-known, differing regional hemoglobin treatment target guidelines,' said Glenn Chertow, M.D., M.P.H., Professor of Medicine, Chief, Division of Nephrology at Stanford University, and Co-Chair of the independent Executive Steering Committee for PRO2TECT and INNO2VATE. 'The analyses also confirm there was no increased cardiovascular risk associated with vadadustat across the U.S. patients treated to a target hemoglobin range of 10 to 11 g/dL.'
The PRO2TECT data are being presented today during a late-breaking oral presentation, titled 'Global Phase 3 Clinical Trials of Vadadustat vs Darbepoetin Alfa for Treatment of Anemia in Patients with Non-Dialysis-Dependent Chronic Kidney Disease' (Abstract FR-OR54) at ASN Kidney Week. Akebia's vadadustat development program also includes INNO2VATE, the global Phase 3 program for the treatment of anemia due to CKD in adult patients on dialysis. Results from this program were presented at ASN Kidney Week in an oral presentation on October 22, 2020.
About the PRO2TECT Global Phase 3 Program of Vadadustat
Akebia's global PRO2TECT program included two separate Phase 3 studies of ESA-untreated patients (Correction) and ESA-treated patients (Conversion), which collectively enrolled 3,476 adult patients not on dialysis with anemia due to CKD. Both PRO2TECT studies were global, multicenter, open label (sponsor blind), active-controlled (darbepoetin alfa - an injectable erythropoiesis stimulating agent (ESA)), non-inferiority studies. In both studies, patients were randomized 1:1 to receive either vadadustat or darbepoetin alfa. Efficacy and safety results were measured against non-inferiority margins agreed upon with the FDA and the EMA.
About Anemia due to Chronic Kidney Disease (CKD)
Anemia is a condition in which a person lacks enough healthy red blood cells to carry adequate oxygen to the body's tissues. It commonly occurs in people with CKD because their kidneys do not produce enough erythropoietin (EPO), a hormone that helps regulate production of red blood cells. Anemia due to CKD can have a profound impact on a person's quality of life as it can cause fatigue, dizziness, shortness of breath and cognitive dysfunction. Left untreated, anemia leads to deterioration in health and is associated with increased morbidity and mortality in people with CKD.
About Vadadustat
Vadadustat is an oral hypoxia-inducible factor prolyl hydroxylase (HIF-PH) inhibitor designed to mimic the physiologic effect of altitude on oxygen availability. At higher altitudes, the body responds to lower oxygen availability with stabilization of hypoxia-inducible factor, which can lead to increased red blood cell production and improved oxygen delivery to tissues. Vadadustat is in global Phase 3 development for the treatment of anemia due to CKD and is not approved by the U.S. Food and Drug Administration (FDA) or any regulatory authority with the exception of Japan's Ministry of Health, Labour and Welfare (MHLW). In Japan, vadadustat is approved as a treatment for anemia due to CKD in both dialysis-dependent and non-dialysis dependent adult patients.
About Akebia Therapeutics
Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. The Company was founded in 2007 and is headquartered in Cambridge, Massachusetts.
Forward Looking Statements
Statements in this press release regarding Akebia's strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, including but not limited to statements regarding the expectation that the potential approval of vadadustat in the U.S. as a treatment for patients not on dialysis will be a review issue for the FDA; the belief that newly presented analyses of PRO2TECT and the totality of the data from the global Phase 3 program of vadadustat will inform the FDA's review of treatment of patients not on dialysis with vadadustat; the potential for obtaining approval of vadadustat in dialysis; the potential indications for and benefits of vadadustat; submitting filings for marketing approval of vadadustat, and the timing thereof and the clinical opportunity for vadadustat. The terms 'believe,' 'confident,' 'expect,' 'plan,' 'potential,' 'will,' 'working' and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including the timing and content of advice given and decisions made by health authorities, including approval and labeling decisions; the actual time it takes to make regulatory submissions for vadadustat to health authorities, including the submission of the NDA to the FDA and the submission of the MAA to the EMA; risks associated with the Priority Review Voucher for vadadustat; the potential direct or indirect impact of the COVID-19 pandemic on our business, operations, and the markets and communities in which we and our partners, collaborators, vendors and customers operate; manufacturing and quality risks; risks associated with management and key personnel changes and transitional periods; the actual funding required to continue to commercialize our commercial product, to develop and commercialize vadadustat, and to operate the Company; market acceptance and coverage and reimbursement of our commercial product and vadadustat, if approved; the risks associated with potential generic entrants for our commercial product and vadadustat, if approved; early termination of any of Akebia's collaborations; Akebia's and its collaborators' ability to satisfy their obligations under Akebia's collaboration agreements; the competitive landscape for our commercial product and vadadustat; the scope, timing, and outcome of any legal, regulatory and administrative proceedings; changes in the economic and financial conditions of the businesses of Akebia and its collaborations partners and vendors and Akebia's ability to obtain, maintain and enforce patent and other intellectual property protection for our commercial product, vadadustat and any other product candidates. Other risks and uncertainties include those identified under the heading 'Risk Factors' in Akebia's Quarterly Report on Form 10-Q for the quarter ended June 30, 2020 and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.
Contact:
Kristen K. Sheppard
Email: Ir@akebia.com
AKEBIA THERAPEUTICS, INC. 
