Being first isn’t the only goal in the drug-development business.
Sometimes, an unmet patient need is so great, companies that are second or third to market stand a good chance of success. That’s what Akebia Therapeutics Inc. near Cincinnati is hoping for as it prepares to do Phase 2 clinical trials of its drug to treat chronic anemia.
Millions of Americans who have heart, kidney, inflammatory bowel or other chronic diseases, are undergoing chemotherapy to treat cancer, or are elderly suffer from chronic anemia, according to the National Anemia Action Council. For many of these patients, anemia has progressed from simple fatigue to a life-threatening condition.
Pharmaceutical companies have developed injectable drugs, called erythropoietin-stimulating agents (ESAs), to treat chronic anemia. But the drugs that boost erythropoietin — the hormone that stimulates the bone marrow to make more red blood cells, solving anemia — also cause an unacceptably high rate of heart attacks and strokes among some patients. These drugs can cost up to $13,000 a year.
The Food and Drug Administration has required drugmakers to warn patients about these risks. The federal regulator also has called on researchers and drug developers to come up with safer drugs.
Enter, Akebia.
More than three years ago, Joseph Gardner, who was leaving Cincinnati consumer products giant Procter & Gamble (P&G) after managing the company’s drug patent portfolio, and John Rice, who leads Cincinnati’s Triathlon Medical Ventures, snagged a half-dozen potential therapies as P&G wound down its pharmaceuticals business. Gardner — now Akebia’s president and CEO — and Rice created Akebia to commercialize those therapies, one of which is an oral erythropoietin-stimulating drug known as AKB-6548.
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.
Last week, the small molecule discovery and development company in Blue Ash, Ohio, reported positive results from a second Phase 1 clinical trial of its anemia drug. In 33 healthy volunteers, the drug increased erythropoietin and immature red blood cells known as reticulocytes. Doses of the drug were safe and well tolerated by the volunteers.
Akebia also announced last week the second closing of a $16 million financing round announced almost a year ago, boosting the round to $17 million.
By the end of the year, the company’s executives likely will decide to partner with a large pharmaceutical company to finish development of AKB-6548 or start a second fundraising round to pay for that development, said Ian Howes, the company’s chief financial officer and vice president of corporate development.
At the moment, large pharma companies are licking their chops at a banquet of partnering opportunities with biotechnology companies. “That’s probably driven by a shortage of venture capital and private equity,” Howes said. “Biotech companies are generally left with programs that are unfunded. So, they are trying to find homes for them.”
Most big drug companies are looking for later-stage drug development programs than those Akebia has, he said. But drug companies always are hunting for potentially game-changing therapies to license from biotech companies.
Now, AKB-6548 does not have a novel mechanism, Howes said. It’s also not first-in-class — a couple of companies (GlaxoSmithKline (NYSE: GSK) and FibroGen) are “between six and 12 months ahead of us in the clinic,” he said.
But the opportunities of entering a broadening — and some would say, underserved — market for a safe, effective, affordable oral treatment for chronic anemia could outweigh the benefits of being first in the market.
“If you look at the anemia space as a whole, there are only three marketed anemia drugs in the United States, and they generate about $11 billion a year — $8 billion in the U.S. And they’re all unsafe drugs,” Howes said. “So, the FDA is looking for new drugs that are safer and easier to use.”
If the new class of drugs to which AKB-6548 belongs is successful, “You’ll see a couple of things,” he said. “You’ll see the injectable erythropoietin-stimulating agents lose a lot of market share. But I think you’ll see the overall market expand.”
The market for anemia-fighting drugs likely would get bigger as patients who can’t be treated by the risky injectable drugs, such as the elderly or those on chemotherapy, begin to be treated with the new class of oral drugs. That’s attracting the interest of large drug companies, Howes said.
Price pressure also is expected. Beginning in January, “ESA injectables are going to be bundled with all the other dialysis supplements” for insurance reimbursement purposes, he said. “So, we’re going to see the prices of ESAs tumble significantly next year.”
The tumble likely would put a lid on the price of Akebia’s AKB-6548. But if more patients are treated with the new class of drugs, the increased volume “would make up for the decrease in prices,” Howes said. “The bottom line is, with an easier-to-use, safer drug, you’ll see a lot more of it being used, then it will become cheaper.”
