By Bill Berkrot
(Reuters) - Celgene Corp said its Abraxane drug helped patients with advanced pancreatic cancer live an average of two months longer than chemotherapy and significantly increased the percentage of those who survived with the disease for up to two years, according to a late-stage study.
Based on the results, Celgene said it expects to file applications in the United States and Europe in the first half of this year seeking approval for the use of Abraxane for pancreatic cancer.
Celgene previously said Abraxane met the main survival goals of the study of 861 patients who had not previously been treated for their cancer. It released the details on Tuesday ahead of a medical meeting where the study will be presented this week.
Patients who received Abraxane in combination with the chemotherapy agent gemcitabine had a median overall survival of 8.5 months, compared with 6.7 months for patients who received standard therapy of gemcitabine alone. The result was deemed to be statistically significant, researchers said.
"It's such a tough disease that moving the needle on survival at all is a major accomplishment," Dr. Daniel Von Hoff, the study's lead investigator, said in a telephone interview.
"It's been a pretty tough, discouraging area," he said, adding that the overwhelming majority of studies of similar patients have failed to improve survival at all.
Abraxane plus gemcitabine demonstrated a 59 percent increase in one-year survival - 35 percent vs 22 percent - and doubled the rate of survival at two years from the start of treatment, with 9 percent still alive compared with 4 percent for gemcitabine alone.
Von Hoff said this was the first study in such advanced pancreatic cancer to report two-year survivors.
"People will look at it and say, 'I have more of a chance than I thought,'" he said. He added that if people can live for 24 months with the disease, Abraxane might enable them to survive until better options evolve.
"It gives you more of a chance to get to hopefully a real breakthrough," said Von Hoff. "You just don't know what's coming."
He will present the data on Friday at the American Society of Clinical Oncology (ASCO) Gastrointestinal Cancers Symposium in San Francisco.
Abraxane is already approved to treat breast and lung cancer and has also demonstrated efficacy against melanoma.
Advanced pancreatic cancer has the lowest survival rate among all cancer types, with a five-year survival rate of less than 2 percent. With few recent advances in the field, it remains one of the most critical unmet needs in oncology.
The survival extension seen in the Abraxane study is likely to be viewed by regulators as clinically meaningful and result in an expanded approval and a significant sales boost for Abraxane.
The drug had sales of $106 million in the third quarter of 2012. Celgene has targeted Abraxane sales of $1 billion to $1.25 billion in 2015, reaching as high as $2 billion in 2017, helped by use of the drug against pancreatic cancer and melanoma.
Abraxane also met secondary goals of the study. It demonstrated an improvement in a median progression-free survival, or the time it took for the disease to worsen, at 5.5 months versus 3.7 months, and had an overall response rate, or those who experienced tumor shrinkage, of 23 percent compared with 7 percent in the control group.
Time to treatment failure was also improved with Abraxane plus gemcitabine, Celgene said.
Patients in the study had the most advanced form of the disease, in which the cancer had already spread to other organs, such as the liver and lungs.
Von Hoff characterized the side effect profile of the combination therapy as "acceptable and manageable."
Deaths connected to adverse events were 4 percent in both arms of the study.
There was a significantly higher incidence of peripheral neuropathy, a numbness in the fingers or toes, seen in the Abraxane group, at 17 percent versus less than 1 percent in the control group, researchers said.
"One thing we worry about is febrile neutropenia," or a low white blood cell count with fever, Von Hoff said. The incidence was 3 percent for the Abraxane combination and 1 percent for gemcitabine. "That is very comforting," he said.
"I'm glad to see we've got something that has an impact on survival," Von Hoff said. "It's something to build on."
(Reporting by Bill Berkrot; editing by John Wallace)