The U.S. Food and Drug Administration has awarded fast-track approval status for Keryx’s KRX-101.Israel’s Keryx Biopharmaceuticals is shepherding a drug through the final stages of U.S. clinical trials that gives hope to the three to four million U.S. diabetes sufferers who experience kidney failure along with their diabetes.
The prognosis for patients suffering from what’s known as end-stage kidney disease is grim at present and their only hope for survival is through a continuous regimen of kidney dialysis or from a kidney transplant.
Keryx didn’t develop the drug, which it calls KRX-101 and which is known generically as sulodexide. Sulodexide has been sold in Europe for 20 years as a treatment for coronary heart disease, but Keryx ran across some promising early test results for the drug for diabetes done by its Italian manufacturer, Alpha Wasserman. The Israeli startup snapped up the licensing rights to sulodexide for diabetic kidney failure, also known as diabetic nephropathy, with the intention of conducting clinical trials in the United States.
“(Alpha Wasserman) fielded a large stage two study and the results were very exciting,” said Barry Cohen, Keryx’s Cambridge, Mass.-based vice president of business development. “We licensed the drug from them since they had no intention of taking it to market.”
In the early-stage tests, KRX-101 successfully reduced the albumin that is released by the kidneys of nephropathy patients and continued to do so for a period of four months after the patients stopped taking the drug. Albumin is a protein that is retained if the kidneys are functioning properly and the restoration of albumin retention is a sign that the drug is repairing damage to the walls of the kidney, Cohen said.
Results in the first two stages of clinical trials have been so promising that the U.S. Food and Drug Administration has awarded so-called fast-track status to KRX-101 for Phase III clinical trials in order to speed approval.
Fast track means the drug can be approved for sale in the United States if Phase III patients experience the same improved albumin retention as the patients in earlier trials. Without fast-track status, Keryx would have to prove statistically that the drug prolonged patients’ lives, a process that could stretch the trials out many years. In the meantime, many patients would lack access to a drug that has been used safely in Europe for 20 years, albeit for a different disease.
“The only problem (with fast track) is that all the clinical trials patients want the drug and no one wants the placebo,” Cohen said, since many people think the FDA is tipping its hand that it thinks the potential benefits may far outweigh the dangers by granting fast-track status.
At this point, Keryx estimates that sales of KRX-101 would be $1 billion a year in the United States.
While KRX-101 is the Keryx drug that shows the most potential, the company was formed in Jerusalem in 1999 to do research and produce drugs using what’s known as kinase technology. Kinase technology involves manipulating the chemistry of the human body to direct drugs to the location in the body where they can best attack diseases.
Keryx is developing a drug based on kinase to treat the 20 percent of patients with prostate cancer whose cancers don’t respond to other treatments.
“We’ve tested it in animals with several rounds of experiments and we have seen that their tumors go away and stay away,” Cohen said. “We’ve seen powerful results, but only in animals and mice. We hope to be in Phase I (of FDA clinical trials) with this sometime in the middle of 2003.”
Keryx is led by founder Dr. Morris Laster, who started two other Israeli biotech firms, XPL and Neose.
“(Laster) because of his experience knew that kinase was in the early stages of development,” Cohen said. “He wanted to have a late-stage drug in the portfolio, which is why we bought KRX-101.”