- Akebia Therapeutics reported success in the first of two large Phase 3 programs testing its anemia drug vadadustat in adults with damaged kidneys.
- Akebia disclosed results from the two INNO2VATE studies, in which vadadustat had to match a standard of care biologic in nearly 4,000 chronic kidney disease patients on dialysis. The drug showed to be just as effective at boosting hemoglobin levels, and patients on it had numerically fewer heart attacks and strokes, a key safety goal.
- Akebia is part of a high-stakes race to use pills, not injectable biologic drugs, to treat anemia. It's trying to catch up to FibroGen, which has a rival drug that's approved in China and Japan and under review in the U.S. Akebia still awaits results from a second Phase 3 program, in non-dialysis patients, that should produce data soon.
The next few years could bring significant changes for chronic kidney disease patients with anemia, a condition that, for years, has been treated with injectable drugs like Amgen's Epogen and Aranesp.
Akebia, FibroGen, GlaxoSmithKline and others are trying to upend the market for those drugs — which have generated billions in yearly sales, but don't work for everyone and carry risks of serious heart problems. They're attempting to do so with a group of pills that trick the body into thinking it's in low-oxygen conditions, which in turn makes the body produce more red blood cells. And they're trying to prove that these drugs can be just as effective as their biologic counterparts, but potentially safer and more convenient.
The true test will likely come next year, when these drugs start to get in the hands of clinicians in the massive U.S. and European markets. FibroGen's drug roxadustat has already won approvals in China and Japan, but could be approved by the Food and Drug Administration by late December. A regulatory filing in Europe is underway as well.
Tuesday's news helps Akebia keeps pace. The company has two large Phase 3 programs underway in anemic chronic kidney disease patients who are either on dialysis, in the case of the INNO2VATE program, or not on dialysis, in the case of the PRO2TECT program.
Akebia has now reported results from the INNO2VATE studies. One study tested vadadustat in 369 patients who recently started dialysis for failing kidneys and had limited exposure to biologics. The other, much larger trial enrolled 3,554 patients who had been taking biologics and were switched to either Aranesp or vadadustat.
In both trials, vadadustat was non-inferior to Aranesp in helping patients produce the oxygen-carrying protein hemoglobin, on average, at two different time ranges — both 24 to 36 weeks and 40 to 52 weeks after the start of treatment.
Vadadustat was also numerically superior to Aranesp on a key safety measure, the time to death from any cause or a major cardiovascular event such as a heart attack or stroke. The results match data from FibroGen: Both vadadustat and roxadustat had a hazard ratio of 0.96 in studies pitting their drugs against Aranesp in dialysis-dependent patients, which in a statistical sense, means participants on the FibroGen and Akebia medicines were slightly more likely to have better outcomes.
FibroGen, however, was unable to replicate those results in pre-dialysis patients. That gives vadadustat the chance to separate itself if it produces similar findings in the PRO2TECT trials, wrote RBC Capital Markets analyst Kennen MacKay.
Serious treatment-related side effects were more common in patients on Aranesp, at 58.3%, than patients on vadadustat, at 55%. The most frequent side effects tied to treatment were diarrhea, pneumonia, high blood pressure and abnormally high potassium levels.
The PRO2TECT trial data are expected in mid-2020. The company plans to file approval applications in the U.S. and Europe "as quickly as possible" pending positive results, CEO John Butler said in a statement. The drug is already under review in Japan. Akebia shares rights to vadadustat with Otsuka and Mitsubishi Tanabe Pharma.
Akebia shares ticked up about 37%, to $11.90 apiece, in early trading Tuesday.