Coming in a few days under the wire, SuperGen Inc. said it has begun the rolling submission of a new drug application for Orathecin (rubitecan), an oral chemotherapy drug for refractory pancreatic cancer patients.
The submission, including data from more than 2,700 patients, had been expected by the end of this year and likely will finish by the end of the first quarter of next year, said Edward Jacobs, chief business officer and chief financial officer of Dublin, Calif.-based SuperGen.
"The FDA really prefers [a rolling submission] when they have a lot of patient data coming in," he said. "We're working with them on a weekly basis," providing the chemistry, manufacturing and controls part of the package first, with clinical data in February.
Supporting the NDA are data not only from the Phase III program - the largest randomized trials ever done worldwide in pancreatic cancer - but also from a Phase II study, results of which were presented at the American Society of Clinical Oncology meeting in May. Data from the Phase III trials are expected at the next ASCO meeting, in May 2003.
Orathecin was granted fast-track status by the FDA in November, but the drug has not had an easy road. In March, Abbott Laboratories Inc., of Abbott Park, Ill., ended its potential $150 million deal signed in 1999 for rubitecan, a topoisomerase I inhibitor extracted from the bark and leaves of the Camptotheca acuminata tree in China. (See BioWorld Today, March 6, 2002, and Dec. 23, 1999.)
"We have numerous suitors," Jacobs said, adding that the company also is studying options in non-equity financing. "Nobody's put an offer on the table, but we're lined up with meetings from Jan. 2 until the middle of February - the whole dance, as I call it."
Jacobs told BioWorld Today that "for once in the lifetime of this company, we're in the driver's seat. We feel we could do this [in the U.S.] alone, but we certainly need an international partner."
SuperGen also has Nipent, an approved small-molecule purine analogue for hairy-cell leukemia, for which the company maintained a distribution deal with Abbott. In December, at the annual meeting of the American Society of Hematology, SuperGen reported encouraging data from a Phase II combination trial that included Nipent in allogeneic bone marrow transplant patients with a variety of leukemias and lymphomas.
Another anticancer drug, decitabine, reverses DNA hypermenthylation and is being tested for myelodysplastic syndromes.
"With any kind of luck and the grace of God, we'd submit something on that in 2004," Jacobs said.
But Orathecin comes first in the pipeline, and the unmet medical need is significant. More than 75,000 people die worldwide from pancreatic cancer, which in the U.S. is the fourth leading cause of death by cancer and carries an average one-year survival rate of less than 5 percent.
"We're not curing anybody, but we have people who are survivors for many months and even years," Jacobs said, noting one man who has lived with pancreatic cancer for six years. Many others are have lasted into the "magic 12-to-24-month range," he said.
"This is not an easy game and we don't know how it's going to sort out [with the FDA], but we're much more confident than we were six months ago," Jacobs said. "We're a small company, 130 people, and 90 percent of them are working on this. We even have sales people retrieving data from the clinics."
Whether a partner will be chosen for the U.S., and which company might be signed up for overseas marketing, will be resolved later, he said. For now, the all-out goal is to present the FDA with data for prompt approval.
"It may sound corny, but we have an obligation to pancreatic cancer patients," Jacobs said. "That's what we're trying to do, and we bet the company on it."
SuperGen's stock (NASDAQ:SUPG) closed Monday at $3.29, up 61 cents, or 22.8 percent.