ILEX Oncology Inc.'s drug eflornithine (DFMO) failed to reach statistical significance in a Phase III trial with superficial bladder cancer patients, resulting in the company's decision to discontinue development.
"We're obviously disappointed because it was a pivotal Phase III trial," said Laura Boscarino, spokeswoman for the San Antonio-based company. "However, as a company, we are focused on cancer treatments and this was the only drug in our pipeline that was a cancer-prevention agent. So, given that distinction, it was not a core product in the company for a very long time. From a financial point of view, we never accounted for this in our financial model, and it certainly has no material impact on us."
ILEX's stock (NASDAQ:ILXO) closed Friday at $24.72, up 40 cents.
The Phase III study of DFMO was conducted in collaboration with the National Cancer Institute in Bethesda, Md., which is continuing to study the agent in breast cancer, colon cancer and nonmelanoma skin cancer. An independent data monitoring board analyzed the data, which showed that DFMO did not prevent the recurrence of cancer more so than placebo.
"They almost exactly mirrored each other," Boscarino told BioWorld Today. "It showed no statistical significance."
Boscarino was unable to give exact data or details about the Phase III trial, which enrolled about 450 patients, but she said the company is discontinuing development in bladder cancer.
Brian Rye, a biotech analyst with Janney Montgomery Scott LLC in Philadelphia, said the failure is insignificant.
"I think the company had been fairly up front the last couple of years," he told BioWorld Today. "For valuation purposes, we had attached maybe a 5 percent chance of success.
"It was really no surprise. It does not impact any of our estimates going forward."
Bladder cancer patients are a "tough-to-treat" population, and no drug yet has been able to prevent recurrence of the disease, Rye said.
Aside from DFMO, ILEX has one marketed product - Campath - which had sales of $53.3 million in the first nine months of 2003. Campath is partnered with Berlin-based Schering AG and its U.S. affiliate Berlex Laboratories Inc., of Montville, N.J. Millennium Pharmaceuticals Inc., of Cambridge, Mass., co-developed the drug.
ILEX submitted a rolling new drug application for clofarabine at the end of 2003 to treat pediatric acute leukemia. (See BioWorld Today, Oct. 23, 2003.)
"We hope to have that submission fully in by mid-this year," Boscarino said, "so that's our next drug approval, if all goes well."
Clinical results of clofarabine showed a 34 percent overall response rate in 29 children with acute myelogenous leukemia, and a 25 percent overall response rate in 36 children with acute lymphoblastic leukemia. ILEX is the North American co-development partner of Bioenvision Inc., of New York, which holds the compound's rights outside the U.S.
ILEX also has Synthadotin (ILX-651) in a Phase II melanoma trial that is ongoing. The company expects to begin a clinical study of the drug in non-small-cell lung cancer this year, as well as a third, undetermined indication. Synthadotin is a third-generation synthetic pentapeptide analogue of the natural substance dolastatin. It targets tubulin.