Washington Editor

In reporting positive results from a Phase III study of Oravescent fentanyl, Cephalon Inc. said it is on track to submit a new drug application to the FDA next quarter.

The product produced a statistically significant and clinically relevant benefit to breakthrough cancer pain patients who already were receiving around-the-clock, pain-controlling medication for their underlying chronic pain. Specifically, the double-blinded, placebo-controlled, variable-dose trial included 123 patients, and those treated with Oravescent fentanyl showed a statistically significant improvement on the primary endpoint, a measure of the Sum of Pain Intensity Difference scale (p<0.01).

"The patients received a blinded titration packet with the drug, which will either have an active dose or placebo," explained Robert Grupp, Cephalon's vice president of corporate affairs. "After taking that, their pain relief is noted, and that allows for the statistical measurement."

Prior to that step, patients were titrated with a range of Oravescent fentanyl doses: 100 mcg, 200 mcg, 400 mcg, 600 mcg and 800 mcg. Grupp told BioWorld Today that one dose stood out as more efficacious than the others, though those data and other findings from a full analysis are to be reported at a yet-to-be-determined medical meeting. Secondary endpoints include measures of three other subjective scales: the Pain Intensity Difference, the Total Pain Relief and the Global Medicine Performance assessments.

Patients who failed to achieve pain relief were allowed to take a rescue dose of their underlying medication. Oravescent fentanyl was generally well tolerated in the study, which began a year ago. (See BioWorld Today, May 10, 2004.)

Cephalon, headquartered in Frazer, Pa., added that it would complete the long-term safety portion of the program by the middle of the year. Should it reach the market, the drug would complement Actiq (fentanyl citrate), Cephalon's already-marketed therapy for cancer pain that generated $101.9 million in first-quarter sales.

Both fentanyl products are delivered transmucosally, though Oravescent fentanyl comes as a tablet that is placed under the tongue while Actiq is applied as a lozenge on a handle.

Oravescent fentanyl was acquired through Cephalon's $515 million buyout of CIMA Labs Inc., of Eden Prairie, Minn. The oravescent delivery technology underlying the product allows it to provide pain relief quicker than the 15 minutes it takes for Actiq. (See BioWorld Today, Nov. 5, 2003.)

Additional studies into Oravescent fentanyl are aimed at broadening a label beyond the breakthrough cancer pain indication it initially would share with Actiq, Grupp said. Down-the-road development could position Oravescent fentanyl for the entire acute pain market.

Beyond those plans, Cephalon awaits FDA action on a version of its marketed sleep disorder drug modafinil for attention deficit/hyperactivity disorder in children, as well as for Nuvigil, a single-isomer version of modafinil. Seeking another indication for Gabitril, which already is approved for epilepsy, the drug is in a Phase III study that should end by the end of this year. Another late-stage product in the company's portfolio is CEP-1347, which is halfway through a four-year Phase III trial to test its ability to slow the progression of Parkinson's disease. An interim analysis is scheduled for this month.

Grupp added that Cephalon could look to expand its oncology franchise through mergers and acquisitions.

On Thursday, the company's shares (NASDAQ:CEPH) gained 56 cents to close at $45.07.