US-based biopharmaceutical firm Akebia Therapeutics has reported positive top-line results from its Phase IIb placebo-controlled trial of its once-daily, oral therapy, AKB-6548, in non-dialysis patients with anaemia related to chronic kidney disease (CKD).
AKB-6548 is designed to stabilise 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 HIF prolyl hydroxylase (HIF-PH) enzyme.
The trial met its primary endpoint and the results showed that AKB-6548 was generally well tolerated, confirming that the once-daily, oral therapy can successfully increase and maintain haemoglobin (HGB) levels.
A total of 209 patients were enrolled in the 20-week trial and they were randomised 2:1 to active treatment or placebo.
Akebia senior vice-president and chief medical officer Dr Brad Maroni said: "These Phase IIb results are impressive, demonstrating a sustained effect on haemoglobin throughout the twenty weeks of treatment.
"The challenges associated with current treatment options are well documented. The data underscore the potential of AKB-6548 to effectively raise and maintain haemoglobin levels in a safe, predictable and controlled manner."
Results showed that 54.9% of patients who received AKB-6548 met the primary endpoint versus 10.3% in the placebo group.
The trial’s primary endpoint was defined as achieving or maintaining a mean HGB = 11.0g/dL or increasing HGB by = 1.2g/dL above the pre-treatment value as measured by the mean HGB value at weeks 19 and 20.
Commenting on the study results, Akebia president and chief executive officer John Butler said: "This marks a very important milestone for Akebia and sets a clear path forward for a global Phase III programme in renal anaemia.
"The strength of these data reinforces the best-in-class potential of AKB-6548 in anaemia 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 US and European regulatory agencies in preparation for launching Phase III registration studies next year."
AKB-6548 is also being evaluated in a Phase II clinical trial to treat dialysis patients with anaemia related to CKD. Results from that study are expected in the third quarter of 2015.