By Kim Coghill

Washington Editor

BioChem Pharma Inc. said the M.D. Anderson Cancer Center in Houston is initiating an exploratory Phase III study of Troxatyl as a component of first-line therapy in previously untreated patients with poor-prognosis acute myelogenous leukemia (AML).

Troxatyl (troxacitabine) is an investigational compound currently in clinical trials for the treatment of several forms of cancer. BioChem Pharma, of Laval, Quebec, discovered the product, which is a complete DNA chain terminator and DNA polymerase inhibitor. It acts by incorporating itself into the growing DNA chain of cells, interfering with their ability to replicate further.

Jacques Jolivet, BioChem Pharma's director of clinical development, oncology, said the trial will begin with three arms, from which the best two will be selected for a larger, more standard trial.

"Anderson will conduct the trial because they have been involved with us since the development of this drug and they are the largest leukemia center in the world," he said. "They have about 1,200 new cases a year and that is much more than their closest competitor, which is about 300 cases a year. They are by far the largest and most active group and we are very honored to be associated with them."

The study will compare Troxatyl administered in association with either ara-c or idarubicin to a control regimen of ara-c and idarubicin using a Bayesian design (adaptive randomization). The exploratory phase will include 100 patients and results should be complete in about two years.

AML is the most common type of leukemia in adults, with a median age of 65 at the time of diagnosis. AML is not a single disease but a group of neoplastic disorders characterized by proliferation and accumulation in the bone marrow and peripheral blood of immature hematopoietic cells. Untreated, AML is usually fatal within weeks to months after diagnosis.

However, for patients treated with presently available chemotherapy, the five-year survival rate is about 10 percent to 15 percent.

Troxatyl is being evaluated as a single agent or in combination therapy in a number of ongoing single-site and multicenter clinical trials in hematologic malignancies, including AML, chronic myeloid leukemia (blastic phase), and in lymphoproliferative disorders such as lymphoma, chronic lymphocytic leukemia and myeloma. It also is being evaluated as a single agent in pancreatic cancer and in combination therapy in a number of solid tumors.

BioChem and Shire Pharmaceuticals Group plc, of Andover, UK, in December entered an agreement to merge, forming a global specialty pharmaceutical company. (See BioWorld Today, Dec. 12, 2000.)

BioChem's stock (NASDAQ:BCHE) closed Tuesday at $33.062, up $1.312. n