CINCINNATI, Ohio — Akebia Therapeutics has successfully completed a Phase I trial of its anemia drug in healthy human volunteers, the company announced.
The drug, a pill known as AKB-6548, was shown to successfully and safely produce the erythropoietin (EPO) hormone that promotes the growth of red blood cells in bone marrow.
“The completion of this phase 1a study is clearly an important milestone for Akebia and we are very pleased to have human safety data and markers of efficacy in humans,” Dr. Robert Shalwitz, the company’s chief medical officer, said in a statement. The 1a trial was conducted in Cincinnati and involved 48 healthy volunteers, according to the statement.
2010 is shaping up to be a big year for Akebia. The company plans to begin Phase 1b and 2a clinical trials, and that’s only the beginning of what could turn into several major developments for the Cincinnati company this year. The primary difference between the 1a and 1b trials will be the number of doses each volunteer receives. In the 1a trial, patients received a single dose, while in the subsequent trial, patients will receive one dose a day over a seven-day period, according to Chief Financial Officer Ian Howes. Akebia hopes to complete the 1b trial in March and announce the results shortly thereafter.
Akebia was born three years ago when Procter & Gamble began dismantling its pharmaceuticals division. Joseph Gardner, a former Procter & Gamble executive and Akebia’s CEO, teamed up with Cincinnati venture-capital firm Triathlon Medical Ventures to buy two molecules — the anemia treatment and a therapy for preventing some heart-related side effects during cancer treatment.
The company pegs the worldwide market for chronic anemia drugs at $10 billion. The company believes its drug can be safer and more effective than existing therapies, most of which are administered via injection. Akebia is in a race to develop its drug against other treatments supported by larger competitors such as GlaxoSmithKline and Takeda Pharmaceuticals. The drug is targeted toward patients with chronic renal disease, as well as predialysis patients.
Akebia has received about $22 million thus far from investors, Howes said. The company could take on another $15 million in commitments this year, with most of that coming from existing investors, though it’s looking to add another one or two new investors, Howes said. He stressed Akebia has “plenty of cash” on hand and the new fund-raising is “nothing urgent.”
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.
More significantly, Akebia is in early talks with some large pharmaceutical firms that are interested in partnering with it on a licensing deal. The deal could happen as soon as the second half of the year, Howes said.
The company’s second drug, AKB-97778, aims to treat vascular leak, a condition that has life-threatening consequences in which blood components leak out of veins and into other systems. Akebia has collected “very encouraging preclinical data” associated with the vascular leak drug and expects to begin human trials this summer.
As for employment growth, 13-employee Akebia is looking to “stay lean,” but could add another one or two scientists later in the year, Howes said.
