Beyond EPO injections: Anemia treatment that comes in a pill

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The pharmaceutical company Affymax just got $10 million for moving forward with an anemia treatment that would require one injection a month as opposed to as many as several shots a week.

Imagine the opportunity if someone can take a similar anemia treatment and let patients swallow it?

Startups and well-established drugmakers are competing to develop the first orally administered anemia treatment. At least five companies are working on an oral small-molecule solution to coax the body to produce enough healthy red blood cells (anemia is often caused by kidney failure or iron deficiency).

The anemia market could be worth up to $10 billion annually. It’s dominated by injectable biologic drugs, such as Amgen’s Epogen and Aranesp and Johnson & Johnson’s Procrit, with Amgen holding most of the market. Those drugs generally work by mimicking the effects of erythropoietin (EPO), a hormone that promotes the growth of red blood cells in bone marrow.

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Many of the current innovations, like the effort of Affymax and rival Celgene, focus on making those injections less frequent and safer.

Anemia pill companies, meanwhile, are developing drugs that are known as HIF-PHIs designed to increase natural production of EPO in anemic patients. These drugs work through a different mechanism than biologic anemia drugs, and the companies developing them believe that oral drugs can make anemia treatment safer, faster, cheaper and more convenient.

Look for these drugs to proliferate the market several years from now. Here’s a look at the major players and where the competition stands today.

GlaxoSmithKline and Bayer: These are clearly the biggest names around oral anemia treatment.

Bayer has begun recruiting kidney disease patients for a phase 1 trial of its anemia drug. The study, with an estimated enrollment of 48 patients, was slated to begin in April and be completed this month, according to the National Institutes of Health.

A Germany-based spokeswoman for the company declined to say when Bayer might file a New Drug Application (NDA) with the U.S. Food and Drug Administration for the drug, called BAY85-3934. “We have a project in the field of anemia currently underway in phase 1,” she stated. “As this stage is still very early, we cannot speculate on any timings regarding filing, etc. The phase 1 is still going and we will update on the progress for this project via Bayer’s regular quarterly and annual updates.”

A GSK spokeswoman similarly declined to provide information beyond what’s on the United Kingdom-based company’s website. The drug, GSK1278863, has undergone three completed phase 1 trials, according to the site. One was a dose-escalation study to evaluate the drug’s mechanism of action in single oral doses in healthy patients. A fourth phase 1 trial, which would evaluate the drug’s action in healthy patients as well as patients with impaired kidney function, hasn’t started recruiting patients.

FibroGen: This San Francisco-based company is currently enrolling chronic kidney disease patients in phase 2a and 2b clinical trials of its anemia drug candidate, FG-4592. FibroGen reported in early May that an interim analysis of data from a phase 2 trial “suggest a favorable safety profile” for the drug.

FibroGen has one key factor in its favor: a deep-pocketed development partner. Tokyo-based Astellas Pharma has licensed the rights to the drug in Japan, Europe and the Middle East. As part of the deal, Astellas shoulders half of the drug’s development costs in the United States and Europe and makes various milestone payments to FibroGen.

The San Francisco company retains marketing rights in North America, South America and the Asia-Pacific region aside from Japan. When the companies announced the deal in 2006, they said it could eventually be worth up to $2 billion for FibroGen if sales goals are achieved, which certainly is a big “if.”

A FibroGen spokeswoman offered the following statement regarding the drug’s timeline: “Final phase 3 requirements will be discussed with the FDA at our end-of-phase 2 meeting targeted for mid-2012; after that, we will have a clearer picture of time to NDA.”

Akebia Therapeutics: The Cincinnati-based startup got its start by licensing a small-molecule anemia drug candidate from Procter & Gamble. The company recently raised a $22 million series B round of investment to take it through a pair of phase 2b trials. The second of those trials is expected to begin in January, and will involve following about 100 patients for 90 days. The once-daily drug, called AKB-6548, will initially be targeted at chronic kidney disease patients.

CEO Joseph Gardner anticipates filing an NDA in 2015. However, to pay for expensive phase 3 clinical trials, the company will need to either find a development partner or land another large venture round. “We’ve been talking to several [potential partners] for some time, and those conversations are ongoing,” Gardner said.

Keeping in mind that Amgen now dominates the anemia drug market, it’s fair to speculate whether it’s one of those potential partners that Akebia is in talks with. It would seem surprising if Amgen was willing to cede its dominant position in the anemia market, meaning the company would have to be looking at partnership opportunities, or working on an oral drug it’s not publicly discussed. Or Amgen has decided that anemia drugs aren’t worth the trouble and doesn’t care if its market-leading position erodes over time.

Ligand: San Diego-based Ligand is developing an oral, small-molecule anemia drug that’s still in the preclinical phase. The EPO-stimulating drug, LG5640, would be targeted to patients suffering from anemia caused by kidney failure, chemotherapy or inflammation. A company spokesman didn’t respond to two requests for comment.

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Brandon Glenn

By Brandon Glenn MedCity News

Brandon Glenn is the Ohio bureau chief for MedCity News.
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