Press Release
Akebia Announces Positive Top-Line Results from its Phase 2b Study of AKB-6548 in Non-Dialysis Patients with Anemia Related to Chronic Kidney Disease
Study Meets Primary Endpoint
Company Plans to Initiate Global Phase 3 Registration Studies in 2015
Conference Call at
The 20-week study enrolled 209 patients, who were randomized 2:1 to active treatment or placebo. The results showed that 54.9 percent of patients who received AKB-6548 met the primary endpoint versus 10.3 percent in the placebo group (p < 0.0001). The primary endpoint was defined as achieving or maintaining a mean HGB ≥ 11.0 g/dL or increasing HGB by ≥ 1.2 g/dL above the pre-treatment value as measured by the mean HGB value at weeks 19 and 20.
The study also was designed to evaluate the ability of the dose titration algorithm to minimize HGB excursions ≥ 13.0 g/dL. Only six patients (4.4 percent) treated with AKB-6548 experienced an excursion above this threshold. This result supports the adaptive dosing approach used in the study, which enabled robust HGB increases while minimizing excursions.
"These Phase 2b results are impressive, demonstrating a sustained effect
on hemoglobin throughout the twenty weeks of treatment," said
AKB-6548 was generally well tolerated. Treatment-emergent adverse events (TEAEs) with AKB-6548 were consistent with those reported in past studies and were well balanced overall between the active and placebo treatment groups (74.6 percent and 73.6 percent, respectively). There was a higher incidence of serious adverse events (SAEs) reported in the active treatment group versus the placebo group (23.9 percent and 15.3 percent, respectively), the most common being renal-related. Of the 49 SAEs reported in the active treatment group, one was considered probably related to active treatment and two were considered possibly related, including one death. There were two additional deaths in the treatment group, neither of which was considered drug-related.
Patients at this advanced stage of CKD have many associated comorbidities and are progressing toward end stage renal disease and dialysis. The overall profile of study participants was consistent with that seen in the general population in the later stages of CKD.
"There is a clear need for new treatment options for patients with renal
anemia," said
"We are extremely pleased with the results of our Phase 2b study. This marks a very important milestone for Akebia and sets a clear path forward for a global Phase 3 program in renal anemia," said John P. Butler, President and Chief Executive Officer of Akebia. "The strength of these data reinforces the best-in-class potential of AKB-6548 in anemia related to CKD and brings us a significant step closer to achieving our goal of bringing innovative therapies to renal patients who are in need of new treatment options. We look forward to discussing these data with U.S. and European regulatory agencies in preparation for launching Phase 3 registration studies next year."
Complete efficacy and safety data from this Phase 2b study will be presented at an upcoming medical meeting. Akebia is also evaluating AKB-6548 in a Phase 2 clinical study for the treatment of dialysis patients with anemia related to CKD. Results from that study are expected in the third quarter of 2015.
About AKB-6548 Phase 2b Study Design
The randomized, double-blind, placebo-controlled study evaluated the HGB response to orally administered AKB-6548, compared to baseline and to placebo treated subjects, in non-dialysis subjects with CKD stages 3, 4 and 5 dosed for 20 weeks. Secondary objectives were to evaluate the hematologic pharmacodynamic response, safety, and tolerability of orally administered AKB-6548. The study enrolled 209 non-dialysis patients with CKD stages 3, 4 and 5, who were randomized 2:1 to receive AKB-6548 or placebo. Patients initiated treatment with either 450 mg of AKB-6548 or placebo, administered once daily for 20 weeks. The dose was adjusted in accordance with the patient's hemoglobin response based on the AKB-6548 dose titration algorithm. Patients were assigned to one of three study groups: recombinant erythropoietin stimulating agents (rESAs) treatment naïve, rESAs previously treated or rESAs actively treated.
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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 may raise hemoglobin levels and RBC count predictably and sustainably, with a positive safety profile and a dosing regimen that allows for a gradual and controlled titration. Furthermore, AKB-6548 may improve iron mobilization, potentially eliminating intravenous iron administration and reducing the overall need for iron supplementation.
About Anemia Related to CKD
Approximately 30 million people in
About
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, plans for presenting a more
detailed analysis of the data from the Phase 2b study, the development
plan for the Phase 3 study including discussions with regulatory
authorities, the expected timing of results from the Phase 2 study in
dialysis patients with anemia related to CKD, and the potential of
AKB-6548 to be a "best-in-class" drug. 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 acceptance of Akebia's abstract for presentation at a medical
meeting; the timing and content of decisions made by the FDA and other
regulatory authorities; the rate of enrollment in the Phase 2 study; the
actual time it takes to complete the Phase 2 study and analyze the data;
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
Investors:
Senior Director, Investor Relations and Corporate
Communications
njones@akebia.com
or
Media:
liz.falcone@fkhealth.com
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Akebia Therapeutics, Inc.
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Cambridge, MA 02142
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