Updated 6:47 p.m.
Small molecule discovery and development company Akebia Therapeutics Inc. reported positive results from a second Phase 1 clinical trial of its anemia drug, as well as the second closing of a $16 million financing round announced almost a year ago, boosting the round to $17 million.
Startup Akebia is racing to the finish line with big drug companies toward a $10 billion-a-year worldwide market for a safe, effective, affordable oral treatment for chronic anemia. So far, there are no oral small-molecule drugs to treat chronic anemia, though expensive injectable drugs that have serious side effects are being used.
On Wednesday, the Cincinnati, Ohio, company’s president and chief executive, Joseph Gardner, revealed the likelihood of a second investigational new drug (IND) application for his company “later this year.” The application would be for AKB-9778 — a drug to treat vascular leak syndrome, a side effect of cancer treatments that represents “multi-billion-dollar markets,” according to Ian Howes, the company’s chief financial officer.
During Akebia’s Phase 1b trial for its anemia drug, 33 healthy volunteers responded favorably to three escalating doses of the company’s leading drug candidate — AKB-6548 — which is an “hypoxia-inducible factor-prolyl hydroxylase inhibitor” designed to boost production of erythropoietin (EPO) in anemic patients. EPO is a hormone that regulates red blood cell production.
In the healthy volunteers, the drug succeeded in increasing EPO and immature red blood cells known as reticulocytes, Akebia said in a release. Doses of the drug also were safe and well tolerated by the volunteers. The trial was conducted at Medpace Inc. in Cincinnati.
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.
“We are very pleased with the results of this Phase 1b study,” said Dr. Robert Shalwitz, chief medical officer for Akebia, in the release. “After 10 days of dosing, we saw a controlled, dose-dependent increase in erythropoietin and reticulocytes, and no serious adverse events at any of the doses tested. We look forward to moving AKB-6548 into Phase 2a clinical trials in July.”
The company is developing its drug to help fight chronic anemia in kidney failure patients. But eventually, it could also help heal bone fractures, wounds and other conditions, according to Akebia.
The worldwide market for chronic anemia drugs is dominated by injectable forms of genetically engineered EPO, Akebia said. These injections can cost thousands of dollars a year. Some large drugmakers like GlaxoSmithKline and Takeda Pharmaceuticals, as well as biotech companies like Akebia and FibroGen Inc., are working on oral small-molecule drugs to treat chronic anemia.
Akebia also announced the completion of a second tranche of the Series A $16 million financing round announced in July 2009. The tranche rose to $5 million from $4 million, which increased the round size to $17 million from $16 million.
The latest funding will be used to take AKB-6548 (the anemia drug) through a Phase 2 clinical study by the end of the year and AKB-9778 (the vascular leak drug) through its IND application and into clinical trials, said Howes, who also is Akebia’s vice president of corporate development.
Akebia has raised $28 million during its Series A round. Novartis Bioventures Ltd., Venture Investors LLC, Triathlon Medical Ventures, Kearny Venture Partners, Athenian Venture Partners and Sigvion Capital — all existing investors — contributed to the latest tranche, the company said.
“Achieving the additional financing and increasing its scheduled size is particularly gratifying,” Gardner said, in the release. “Expanding the tranche is indicative of the strong support we have from our investors and their desire to move the company forward.”
