Washington Editor

SuperGen Inc. said it plans to begin filing a rolling new drug application by the end of the year for Orathecin, an oral chemotherapy compound for the treatment of pancreatic cancer.

The news is the outgrowth of a positive meeting between SuperGen, of Dublin, Calif., and the FDA, Joseph Rubinfeld, SuperGen's chairman and CEO, told BioWorld Today. He said the company will seek approval for patients who are refractory to available therapies.

"This filing is crucial for SuperGen because it ratifies our existence in a sense because people have discounted us in the marketplace, saying we would never be able to do a full NDA [new drug application] single-handedly," Rubinfeld said. "But in a rather small company, we've been able to take a major drug in the largest trial ever undertaken in this disease and bring it to the filing stage, so I think this is a very dramatic watershed endpoint for our company."

SuperGen's stock (NASDAQ:SUPG) closed Thursday at $2.70, up 46 cents, or 20.4 percent.

The FDA has awarded Orathecin (rubitecan) fast-track status, which leads Rubinfeld to believe that the NDA could be before the Oncologic Drugs Advisory Committee as early as June, with an approval quickly thereafter.

If Orathecin makes it to market, it would be the first oral chemotherapy agent in the camptothecin class. Rubinfeld said camptothecin in the form of injections is available. However, they generally have side effects. Orathecin, on the other hand, is a safer pill, he said.

"The benefit of Orathecin is that patients live longer with less pain and fewer side effects and they have a good quality of life," he said. "There were patients [in the trials] that were in bed on another drug who started taking this drug and jumped out of bed to play golf - not all of them, but there is a certain subset that benefited tremendously. Some are alive two or three years later, when normally they would be dead in four months. This is cancer, and there is no cure, but at least we are on the road."

While the company hasn't published detailed data from its Phase III trials, Rubinfeld said SuperGen's 1,000-patient clinical program is the largest randomized pancreatic cancer program ever initiated worldwide. He said the trials have a series of endpoints, including survival.

In addition to the Phase III studies, the filing will include a 58-patient multicenter Phase II trial. In April, the company reported that a long-term follow-up review of the study confirmed the drug's activity in patients with refractory pancreatic cancer who had failed prior treatments.

SuperGen no longer has a partner to help bring Orathecin to market. However, Rubinfeld expects that to change quickly, saying, "Companies will probably be lined up at the door today and tomorrow, once this news gets out."

SuperGen's former partner, Abbott Laboratories, of Abbott Park, Ill., returned rights to the compound to SuperGen several months ago when it ended their $150 million collaboration signed in 1999. (See BioWorld Today, Dec. 23, 1999, and March 6, 2002.)

Abbott said it was dissolving the worldwide sales and marketing agreement in order to conserve its resources.

However, there had been a few problems along the way in the clinical studies. In September 2000, SuperGen's stock tanked 29 percent on news that it would delay filing the NDA. (See BioWorld Today, Sept. 5, 2000.)

Apparently, Abbott had expressed some concerns about trials in which comparison therapies were allowed. SuperGen, in turn, hired a research firm to conduct the Phase III trial.

Rubinfeld said SuperGen has a "robust" pipeline that includes Nipent (generically known as pentostatin or deoxycoformycin), a treatment for hairy cell leukemia, that is being studied in other leukemias.