By Brady Huggett

Continuing toward its goal of having four oncology products in Phase III studies, SuperGen Inc. began enrollment for a Phase III trial of its anticancer compound, decitabine, as treatment for advanced myelodysplastic syndrome.

The open-label, randomized study will pit decitabine against standard therapy in 160 patients split evenly into two groups. The primary endpoint of the trial is the number of months from enrollment it takes for patients' marrow to contain 30 percent leukemia cells.

It is difficult to pin a time frame on completed enrollment, said Robert Cohen, vice president, investor relations and finance at SuperGen, mainly because it's difficult to predict how quickly 160 patients can be found and registered. The trial will take place at 15 medical centers across the United States, and that should help recruitment, he said, but prognosticating beyond enrollment is just as difficult.

"It's not a lot of patients," he said. "But they have to meet a certain criteria. So I can't say when the study will be completed."

The trial is designed to support the filing of a new drug application with the FDA, which Cohen said would be done "as soon as possible" following the closing of the study.

In March 2000, results from a 66-patient, Phase II decitabine study were published in the Journal of Clinical Oncology, showing 49 percent had an observed response rate, with a 64 percent response rate in high-risk patients. In December 2000, at the 42nd Annual Meeting of the American Society of Hematology in San Francisco, data were presented on a study of 124 high-risk patients with advanced myelodysplastic syndrome, showing decitabine had anti-leukemic activity in 50 percent of them. The drug works by a dual mechanism of action; it is both an antimetabolite and a hypomethylation agent.

"It suppresses gene expression," Cohen said. "When you take away the methylation, the genes can properly express themselves and produce mature, healthy cells."

SuperGen, of Dublin, Calif., signed an $8 million Vascular Targeting Agent technology licensing agreement, related to vascular endothelial growth factor, with Peregrine Pharmaceuticals in mid-February, expanding an already formidable cancer portfolio. It also has rubitecan, for pancreatic cancer, in Phase III trials and is gathering data for an NDA filing, having closed enrollment in one of the studies. The opening of enrollment for the decitabine study gives the company half of what it expects to accomplish through 2001. (See BioWorld Today, Feb. 14, 2001.)

"We should have Avicine [vaccine for colorectal cancer] in Phase III by mid-year or sooner," Cohen said. "And Nipent for graft-vs.-host disease in Phase III sometime after the summer. So in the immediate future we will have four drugs in Phase III for cancer."

Avicine is licensed from AVI BioPharma Inc., of Portland, Ore., in a deal that entailed a $20 million payment for U.S. marketing rights. (See BioWorld Today, April 6, 2000.)

Cohen added that SuperGen has several combination studies under way for rubitecan and Phase I studies for inhaled versions of both paclitaxel and rubitecan. And there is more ahead for decitabine.

"We will be starting a Phase II study for non-small-cell lung cancer," said Cohen. "That's in formulation stages now. And we also have in formulation a Phase II study for sickle-cell anemia.

"It could be a very substantial drug for treating cancer," he said.

SuperGen's stock (NASDAQ:SUPG) moved up 12.5 cents Monday to close at $9.50. n

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