A once-daily oral anemia drug from Akebia Therapeutics met its primary endpoint in a phase 2a trial of chronic kidney disease patients.
The successful 42-day trial of the drug, now called AKB-6548, sets the stage for a longer 2b trial that would follow patients for six months, said Bill Daly, Akebia’s senior vice president of business development.
“We couldn’t be more pleased” with the results of the latest trial, Daly said.
Cincinnati-based Akebia’s drug is designed to promote levels of erythropoietin (EPO) in kidney disease patients. EPO is a hormone that controls red blood cell production and causes a controlled rise in hemoglobin levels.
The endpoint of the recent trial was a dose-responsive increase in hemoglobin from baseline over the 42 days of the study, according to a statement from Akebia. The study enrolled 93 patients.
Akebia is racing against several competitors to fill what it sees as an unmet market need for an oral anemia drug. The $10 billion-a-year, worldwide market for chronic anemia drugs is dominated by injectable forms of recombinant EPO, which can cost thousands of dollars a year.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
The company says its drug is potentially safer and cheaper than traditional anemia treatments.
Akebia is looking to start its phase 2b trial this summer. It is funding the phase 2 trials via a $22 million series B round of investment that it closed last year.
However, to fund a phase 3 trial, Akebia will need another infusion of capital, Daly said. That could come from another venture round, a corporate partnership or an outright acquisition, for example.
Akebia expects to file a new drug application for the oral anemia drug in 2015, CEO Joseph Gardner said last year.
