Akebia Therapeutics CEO On Controlling Anemia Safely
Biopharmaceutical Akebia Therapeutics has a laser focus on innovative therapies for treating patients with CKD (Chronic Kidney Disease).
Akebia President and CEO John Butler spoke with Benzinga about the company’s leading compound, AKB-6548, under development as a once-daily oral therapy for treatment of anemia related to CKD.
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Benzinga: First, to set the stage, can you talk a little about HIF and the role it plays in our health and for Akebia?
John Butler: Hypoxia-inducible factor (HIF) is the body's primary sensing mechanism for a change in oxygen levels, or hypoxia.
Relating this to altitude, if you or I were to go to Denver, our body would quickly recognize the lower oxygen level.
HIF has a five-minute half-life. It's constantly being made and destroyed. At altitude, the body senses the lower oxygen level, and inhibits the enzymes that break down HIF. That allows HIF to stabilize.
Our drug is doing, pharmacologically, what our body naturally does when we go to altitude. We inhibit the inhibitor. Managing this in a much more physiologic way, we think, will be much safer.
BZ: Why is safely treating anemia so important?
JB: I worked for Amgen in the early '90s. I went to a dialysis center where patients had hemoglobin’s of six. You and I walk around at 14, 15.
EPOGEN absolutely changed their lives. It was wonderful to be a part of that. Now, 24 years later, we've found that we haven't moved forward, we've actually moved backward.
(Following) larger outcomes trials (we realized) that cardiovascular events in patients increased when treated with EPOGEN.
Patients receive many benefits from having their anemia managed, but you have to do it the right way. That's the attempt here. (By doing it) in a more physiologic way, you lower cardiovascular events.
BZ: How could AKB 6548 be an improvement over other therapies?
JB: First, it is about safety. Our clinical data show this wonderful, gradual rise in hemoglobin.
Sometimes you want to solve a problem quickly. That's not the case here. You want to manage anemia, but one of the places where those increased risks have been identified is also in letting hemoglobin go too high, and what they call cycling of hemoglobin – where it's up and it's down and up and down.
With AKB 6548, we see this very stable hemoglobin control. Physicians are looking to manage the anemia but they also see the safety, because it's that ability to control it in a range that people worried about.
In addition, this is an oral therapy. Patients not yet on dialysis represent the biggest growth opportunity for the product. To have a once-a-day oral product that physicians can prescribe for anemia that they feel is safer should be able to expand the market significantly. We really have that potential.
BZ: What's the status of AKB 6548 therapy?
JB: Last October, we announced phase IIb data in patients who were not on dialysis. Now we're in the middle of regulatory conversations around what the phase III program will look like. We hope to wrap those up soon.
Our plan currently is to start phase III before the end of the year in non-dialysis and we need to start the dialysis trial at some point in 2016.
From a business perspective, we're also in the middle of working on geographic partnerships for Japan and Europe.
We plan to hold on to the U.S. and commercialize the product in the U.S. ourselves, but those two partnerships will be critical in financing phase III, the large outcomes trial.
BZ: Are there any other anticipated uses for AKB 6548 besides CKD (Chronic Kidney Disease)?
JB: There are other opportunities – anywhere you want to manage anemia.
One area we're interested in is the anemia of aging. Millions of older individuals walk around with chronically low hemoglobin levels, in nursing homes etc., and we feel a low dose daily therapy could be an interesting opportunity for those patients to have a significant benefit.
Congestive heart failure is another area and in addition, chemotherapy can have a negative impact on red blood cells as well as white. So, billions of dollars in (potential) sales in oncology.
BZ: What other therapies are you working on?
JB: What they found with EPOGEN was that it actually had an impact on growing the tumor; so once again, a more physiologic approach could be interesting there.
We have a second compound, AKB 6899, which is pre-clinical right now. It's very similar, structurally to AKB 6548.
AKB 6899 decreases vascular endothelial growth factor, or VEGF, levels, which makes it not only a potential treatment for the anemia of cancer, but also an actual anti-tumor agent.
What's interesting, pre-clinically, is that it only really works on hypoxic cells, which tumor cells generally are that is, they have low oxygen levels.
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BZ: What are some of the challenges Akebia has faced in developing these new therapies?
JB: Well, the good thing, obviously, is that we have a high need in this space.
Issues with increased cardiovascular events have made it a very closely watched area from a regulatory perspective. For a small company that is more challenging from a financing perspective.
I think Wall Street is looking for a conclusion to the regulatory conversations we have ongoing. Originally, it was looking for the dialysis data and we really knocked the cover off the ball on that data.
Third is the first geographic partnership that we sign. We expect to have a Japanese deal done by the end of the year based on where things are now.
That, I think, reduces any concerns about our ability to execute the trials, the financing of the company. That will allow us to be in a very different place than we are today, I think.
At the time of this writing, Jim Probasco
Image Credit: Public Domain
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