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Cincinnati’s Akebia Therapeutics races (as safely as possible) toward new anemia treatment

Akebia’s anemia drug, known only as AKB-6548, passed an important milestone recently when Akebia embarked on a Phase I clinical trial and the first steps toward bedside use. The pill manipulates genes to produce the erythropoietin (EPO) hormone that promotes the growth of red blood cells in bone marrow. It aspires to replace riskier injections and treat other anemia sufferers who can’t handle current treatments.

CINCINNATI, Ohio — As Procter & Gamble started the slow dismantling of its pharmaceutical division three years ago, Akebia Therapeutics was there to pick up some pieces.

At that time, Akebia was largely Joseph Gardner – who was leaving P & G after managing the company’s drug patent portfolio – and John Rice – who leads Cincinnati’s Triathlon Medical Ventures. They settled on a half-dozen potential therapies in P & G’s catalog and in a bidding process picked up two molecules: a unique therapy for anemia and a way to prevent some heart-related side-effects during cancer treatment.

The anemia drug, known only as AKB-6548, passed an important milestone recently when Akebia embarked on a Phase I clinical trial and the first steps toward bedside use. The pill manipulates genes to produce the erythropoietin (EPO) hormone that promotes the growth of red blood cells in bone marrow. It aspires to replace riskier injections and treat other anemia sufferers who can’t handle current treatments.

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Akebia is in a race to develop its drug against other treatments supported by the likes of GlaxoSmithKline and Takeda Pharmaceuticals that are further along in their trials. The company thinks it can make up ground. Plus, even if it isn’t first, Akebia thinks it can be the safiest of all possible treatments.

“There’s a pretty good understanding that the winner (in anemia) is the one with the best safety profile in addition to having some cost advantages,” Gardner said.

Gardner opted to leave P & G in 2006 as he witnessed the early phases of a process that ended this year when P & G sold its pharmaceutical unit. He and Rice picked their drugs because of a deal made that year by Fibrogen. A Japenese company paid $300 million for Fibrogen’s early-stage oral anemia drug — and offered $465 million for certain milestones during the drug-approval process (the drug has yet to come to market).

“I had to switch my mindset from a big-pharma mindset to a biotech mindset,” Gardner said on leaving to start Akebia. “I had to market to the venture capital community, and the VC community likes to follow big deals.”

Company leaders selected the name Akebia, a plant also known as the chocolate vine for its smell, for more direct and personal reasons: the name was available and they liked chocolate.

It’s been a fast five months for Akebia. The company added a new chief financial officer in May and then said it had raised $25 million — $10 million beyond what it had announced earlier. Last month, it added a new board memberDr. Victor Dzau, the president and CEO of the Duke University Health System — and a week later announced it was embarking on its clinical trial. It hopes to complete the trial by year’s end and move into Phase 2 next year.

Akebia’s drug could be an improvement for anemia patients, said Dr. Franklin Bunn, a professor at Harvard University and research director of the Hematology Division at Brigham and Women’s Hospital, who is on Akebia’s scientific advisory board. It would move the patients away from injections and is likely less expensive than current treatments, Bunn said.

But Akebia’s approach is different, too. Today’s injections provide erythropoietinin levels much higher than necessary. The treatment triggers unwanted side-effects, including a risk of increased heart attack. Akebia’s pill triggers genes in the body to produce its own EPO and in a much more acceptable amount to cut down on side-effects, Akebia executives said.

Anemia patients would likely have to take the pill once or twice daily.

“This treats anemia in a way that is very efficient and spares patients the injections,” Bunn said. “It would be an enormous benefit.”

But there is global competition to find new ways to treat anemia. For example, GlaxoSmithKline recently completed a Phase 1 trial on its own anemia drug, called only 1278863.

Also Affymax, a California drug developer that’s partnered with Takeda Pharmaceuticals, is in the midst of a Phase 3 clinical trial for its drug, Hematide, which treats anemia associated with chronic kidney failure. Drug treatment in the trials should be done by year’s end. Also, Affymax said it has enough money to fund its Phase 3 trials, but also has the ability to raise up to $60 million more through a stock sale. It expects to file a new drug application sometime next year.

Hematide would be injected once a month — nowhere near as frequently as Akebia’s drug or other current treatments.

Akebia executives think their approach will be much safer than Hematide. Affymax and Takeda last year stopped developing the drug to treat anemia caused by chemotherapy in the wake of federal safety concerns over drugs that increased the risk of blood clots when they are prescribed at higher-than-recommended levels. Affymax stated that regulatory changes and the “commercial environment” made that treatment too difficult to develop.

But the companies said in July that independent monitors approved continued studies for the drug for kidney failure.

The trick, though, is for Akebia to be so much safer and cheaper that physicians would be open to replacing their current treatments with a new drug. Akebia has yet to answer all its safety questions, too. Primarily, the company needs to determine what other genes its drug will stimulate and what those changes could do to a human body.

“I can’t stress enough that it’s too early to predict which way this will go,” Bunn said.

The safer the drug the more lucrative it becomes. Akebia estimates the current American anemia market at $8 billion, though it also thinks it drug can attract an even larger population made up of untreated dialysis patients, anemia patient resistant to current treatments and age-related anemias. With an attractive safety profile, the potential market for AKB-6548 could be much larger than the current market, Akebia executives said.

Gardner also thinks the company can gain ground on its larger competitors because of the eight-member company’s nimble and streamlined business approach. Also, because of layoffs or changes at big pharmaceutical companies, Gardner also thinks Akebia could add heavyweight expertise quickly.

“That’s a key component,” he said. “We do a lot of project  planning and making sure we move programs as efficiently as possible with few mistakes. That is an inherent part of our strategy.”

The company is fully funded to complete the Phase I trial and do additional studies. Its investors include Novartis Bioventures, Kearny Venture Partners, Triathlon and Venture Investors. The company has also received funding from Ohio’s publicly funded Third Frontier program.

While it could in about a year need money to support more tests, Akebia is also exploring licensing rights with a Japanese company to sell the anemia drug in Asia, which would fund continued research for the company, Gardner said. It’s also likely to grow up to as many to 20 employees depending on the results of clinical trials in the next year.

While Akebia’s most advanced drug is its anemia treatment, the company also is excited about its second drug that prevents vascular leak syndrome, in which blood components leak out of veins and into other systems — often during cancer treatment. The drug, if successful, could be used in several multibillion markets, Akebia executives said.