Company to Present Data from Phase 2a Study of Lead Clinical Compound, AKB-6548, a Potentially Novel Approach to Treat Anemia Related to Chronic Kidney Disease
The oral presentation entitled, "Controlled Hemoglobin Response in a
Double-Blind, Placebo-Controlled Trial of AKB-6548 in Subjects with
Chronic Kidney Disease," will take place on
Maintaining hemoglobin (HGB) levels within the target range is important for optimal clinical outcomes. In the previously presented double-blind, placebo-controlled Phase 2a trial, AKB-6548 effectively increased HGB levels in patients with CKD in a controlled manner while keeping the maximum HGB level below 13 g/dL throughout the dosing period. AKB-6548 was generally well-tolerated across the dosing groups with no drug-related serious adverse events.
"There is a clear need for new therapies to treat the increasing patient
population impacted by anemia related to chronic kidney disease," said
AKB-6548 is currently in Phase 2b development for the treatment of anemia related to CKD in patients who are not dependent on dialysis, with results expected in the fourth quarter of 2014. A Phase 2 study of AKB-6548 in patients who are undergoing dialysis is slated to start mid-year 2014.
About the Phase 2a Study Design and Results
The Phase 2a double-blind, placebo-controlled study enrolled patients with CKD stage 3, 4 or 5, not on dialysis, with hemoglobin (HGB) < =10.5 g/dL. Patients were randomized to receive either placebo (n=19) or AKB-6548 (n=72) 240 mg, 370 mg, 500 mg or 630 mg once daily for six weeks. HGB was monitored at each study visit during dosing and was used to determine if the dose of study medication was to be reduced or discontinued. A one-time dose reduction was allowed during the study for any subject responding in excess of protocol-defined HGB values.
As previously presented, AKB-6548 increased total iron-binding capacity and increased HGB levels with a clear dose-response relationship. There was no observed negative impact on key biomarkers of kidney health, with no change from baseline in vascular endothelial growth factor (VEGF), C-reactive protein and Cystatin-C, and there were no drug-related serious adverse events.
About Anemia Related to Chronic Kidney Disease
Representing a growing global health concern, patients with chronic kidney disease progressively lose kidney function resulting in the development of several serious medical conditions including anemia. Left untreated, anemia significantly accelerates patients' overall deterioration of health with increased morbidity and mortality. Anemia results from the body's inability to coordinate red blood cell production in response to lower oxygen levels due to the progressive loss of kidney function.
AKB-6548 is a once-daily, oral therapy currently in development for the treatment of anemia related to chronic kidney disease (CKD). AKB-6548 is designed to stabilize hypoxia-inducible factor (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-PHD) 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. Due to its specific HIF effect, AKB-6548 has the potential to restore coordination of the interdependent processes of iron mobilization and EPO production that are disrupted in patients with anemia related to CKD.
A HIF stabilizer with best-in-class potential, AKB-6548 may raise hemoglobin levels and RBC count predictably and sustainably, with an optimal 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.
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 and the expected timing of the
announcement of data from the Phase 2b study and the commencement of the
Phase 2 dialysis study. 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 actual time it takes to
complete clinical trials and analyze the data; the rate of enrollment in
the Phase 2 dialysis study; the ability of Akebia to successfully
complete the clinical development of AKB-6548 or any other product
candidate; the funding required to develop Akebia's product candidates
and operate the company, and the actual expenses associated therewith;
the content of decisions made by the FDA and other regulatory
authorities; 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 or 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
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