Akebia Therapeutics, Inc. Announces Presentation Of Phase 2b Data For AKB-6548 In Non-Dialysis Patients With Anemia Related To Chronic Kidney Disease At The 52nd ERA-EDTA Congress

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Akebia Therapeutics, Inc. (NASDAQ:AKBA), a biopharmaceutical company focused on delivering innovative therapies to patients with kidney disease through the biology of hypoxia inducible factor (HIF), announced that data for AKB-6548, a once-daily, oral therapy for the treatment of anemia related to chronic kidney disease (CKD), were presented today at the 2015 European Renal Association-European Dialysis and Transplant Association (ERA-EDTA) 52nd Congress, which is being held from May 28 – 31, 2015 in London. Pablo E. Pergola, M.D., Ph.D., Director of the Clinical Advancement Center at Renal Associates PA and Clinical Associate Professor of Medicine at the University of Texas Health Science Center at San Antonio, delivered an oral presentation featuring the data from the Phase 2b clinical study of AKB-6548 in non-dialysis CKD patients. A copy of the presentation is available online at www.akebia.com.

“The Phase 2b data demonstrate the potential of AKB-6548 to tightly control hemoglobin levels within a clinically desired range, a critical goal for the next generation of anemia treatments”

The placebo-controlled, 20-week Phase 2b study enrolled 210 patients with CKD stages 3, 4 and 5 who were randomized 2:1 to receive once-daily AKB-6548 (N=138) or placebo (N=72). Patients were assigned to one of three study groups based on recombinant erythropoiesis stimulating agent (rESA) treatment exposure: rESA treatment naïve, rESA previously treated, or rESA actively treated. The initial 450mg daily dose of AKB-6548 was adjusted in accordance with the patient's hemoglobin response using a dose titration algorithm designed to minimize HGB excursions of greater than 13.0 g/dL.

As previously reported, in this Phase 2b trial, treatment with AKB-6548 controlled hemoglobin (HGB) levels in a sustained manner and in the clinically relevant range, producing a coordinated physiologic response to resolve anemia while avoiding excessive fluctuations in HGB levels which have been associated with increased cardiovascular risks. AKB-6548 was generally well tolerated in the Phase 2b trial and overall adverse events were balanced between the treatment and placebo groups (74.6% vs. 73.6%).

New data presented today showed that the group of patients converted from active rESA therapy to AKB-6548 maintained their mean baseline HGB level of 10.5 g/dL throughout the study. In contrast, patients who were switched from active rESA therapy to placebo experienced a decline in the mean HGB level from 10.4 g/dL to 9.8 g/dL within the first two weeks of randomization. These results support a starting dose of 450 mg once daily for patients converting from rESAs to AKB-6548.

“The Phase 2b data demonstrate the potential of AKB-6548 to tightly control hemoglobin levels within a clinically desired range, a critical goal for the next generation of anemia treatments,” stated Dr. Pergola. “It is particularly noteworthy that patients who were converted from rESA therapy to AKB-6548 treatment maintained their hemoglobin levels throughout the study, providing additional evidence that 450 mg once daily is an appropriate starting dose to carry forward into future trials.”

“The Phase 2b data reinforces the best-in-class potential of AKB-6548 in renal anemia and provides a clear roadmap as we move into our Phase 3 program in the non-dialysis setting later this year,” said John P. Butler, President and Chief Executive Officer of Akebia. “AKB-6548 is designed to offer patients a more effective, safer and more convenient treatment option compared to current therapies, and the data across our entire AKB-6548 development program underscore the potential of this once-daily, oral treatment to transform the anemia treatment landscape.”

About AKB-6548

AKB-6548 is a once-daily, oral therapy currently in development for the treatment of anemia related to CKD. AKB-6548 is designed to stabilize HIF, a transcription factor that regulates the expression of genes involved with red blood cell (RBC) production in response to changes in oxygen levels, by inhibiting the hypoxia-inducible factor prolyl hydroxylase (HIF-PH) enzyme. AKB-6548 exploits the same mechanism of action used by the body to naturally adapt to lower oxygen availability associated with a moderate increase in altitude. At higher altitudes, the body responds to lower oxygen availability with increased production of HIF, which coordinates the interdependent processes of iron mobilization and erythropoietin (EPO) production to increase RBC production and, ultimately, improve oxygen delivery.

As a HIF stabilizer with best-in-class potential, AKB-6548 raises hemoglobin levels predictably and sustainably, with a dosing regimen that allows for a gradual and controlled titration. AKB-6548 has been shown to improve iron mobilization, potentially eliminating the need for intravenous iron administration and reducing the overall need for iron supplementation.

About Anemia Related to CKD

Approximately 30 million people in the United States have CKD, with an estimated 1.8 million of these patients suffering from anemia. Anemia results from the body's inability to coordinate RBC production in response to lower oxygen levels due to the progressive loss of kidney function, which occurs in patients with CKD. Left untreated, anemia significantly accelerates patients' overall deterioration of health with increased morbidity and mortality. Renal anemia is currently treated with injectable recombinant erythropoiesis-stimulating agents, or rESAs, which are associated with inconsistent hemoglobin responses and well-documented safety risks.

About Akebia Therapeutics

Akebia Therapeutics, Inc. is a biopharmaceutical company headquartered in Cambridge, Massachusetts, focused on delivering innovative therapies to patients with kidney disease through HIF biology. Akebia's lead product candidate, AKB-6548, is a once-daily, oral therapy, which has completed a Phase 2b study for the treatment of anemia related to chronic kidney disease in non-dialysis patients and is also being tested in a Phase 2 study for the treatment of anemia in patients undergoing dialysis.

Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements include those about Akebia's strategy, future plans and prospects, including statements regarding the potential indications and benefits of AKB-6548, the potential of AKB-6548 to be a "best-in-class" product, the development plan for AKB-6548 and the commencement of Phase 3 clinical studies of AKB-6548 in non-dialysis patients with renal-anemia. The words "anticipate," "appear," "believe," "estimate," "expect," "intend," "may," "plan," "predict," "project," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions 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 risk that existing preclinical and clinical data may not be predictive of the results of ongoing or later clinical trials; the ability of Akebia to successfully complete the clinical development of AKB-6548; the funding required to develop Akebia's product candidates and operate the company, and the actual expenses associated therewith; the timing and content of decisions made by the FDA and other regulatory authorities; the actual time it takes to prepare for and initiate the Phase 3 clinical studies; the success of competitors in developing product candidates for diseases for which Akebia is currently developing its product candidates; and Akebia's ability to obtain, maintain and enforce patent and other intellectual property protection for AKB-6548. 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 March 31, 2015, and other filings that Akebia may make with the Securities and Exchange Commission in the future. Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release.