Bioenvision Inc. started dosing the first adult patients in a pivotal European Phase II study of clofarabine in acute myeloid leukemia.
Company officials expect the trial, designed to include more than 64 patients, to take about nine months.
Clofarabine has been granted an orphan designation by the European Agency for the Evaluation of Medicinal Products as a treatment for acute lymphoblastic leukemia (ALL) and acute myeloid leukemia (AML).
Meanwhile, San Antonio-based ILEX Oncology Inc. owns rights to clofarabine in cancer indications in the U.S. and Canada. ILEX, which is merging with Cambridge, Mass.-based Genzyme Corp., completed its new drug application in April for the product in the treatment of refractory or relapsed acute leukemia in children.
The FDA is evaluating the NDA under priority-review guidelines, meaning action should come six months after the filing is completed. Clofarabine also has been granted orphan drug designation in adult and pediatric ALL and AML.
David Luci, chief financial officer at Bioenvision, of New York, told BioWorld Today that clofarabine is expected to make it to market on both sides of the Atlantic, first as a pediatric product.
Should the European Phase II end in success, Bioenvision expects to file a supplemental application in Europe, and may share data with ILEX (or Genzyme) for North American markets.
In Europe, adult AML is a $250 million annual market, Luci said.
Clofarabine is a next-generation purine nucleoside antimetabolite that affects DNA synthesis. ILEX licensed exclusive North American development, marketing and manufacturing rights for the product in March 2001. (See BioWorld Today, March 16, 2001.)
The first patient dosed in Bioenvision's adult Phase II trial is in Edinburgh, Scotland. Subsequent patients will be located in up to 11 other UK centers, the company said.
The trial is designed to offer patients who are unable to tolerate intensive courses of chemotherapy with a treatment option. Accelerated enrollment in the trial follows favorable results of a preliminary, investigator-led study evaluating clofarabine as a single agent in the same patient category. That closed ahead of time, with the primary objective of the study being exceeded, with a complete response rate of 64 percent observed in the interim analysis, the company said.
Luci said Bioenvision anticipates studying clofarabine in noncancer indications, including autoimmune diseases.
Bioenvision's stock (AMEX:BIV) gained 51 cents Tuesday to close at $6.61; ILEX's stock (NASDAQ:ILXO) lost 3 cents to close at $24.80; and Genzyme's stock (NASDAQ:GENZ) was up 76 cents to close at $49.33.