Cephalon Inc. called a halt to a late-stage study of a partnered Parkinson's disease drug, CEP-1347, as interim results revealed a lack of clinical benefit.
But the Frazer, Pa.-based company noted that the decision would not halt its collaboration with H. Lundbeck A/S, an alliance focused on developing drugs for neurodegenerative disorders. Nevertheless, the Phase II/III study stoppage was a letdown.
"We were disappointed, clearly," Robert Grupp, Cephalon's vice president of corporate affairs, told BioWorld Today. "But in this business, I suppose, results like this should not be a surprise when you're working with a new molecule and new endpoints."
An independent data monitoring committee recommended that the 800-patient trial be discontinued after completing a planned review of interim results. The evaluation came after the 200th patient received treatment in the study, which began two years ago to test the compound in early onset Parkinson's. Its primary endpoint was a measure of the time to initiation of dopaminergic therapy, the standard-of-care treatment that patients begin as their disease progresses beyond a certain point. A secondary endpoint evaluated disease progression through SPECT imaging.
The committee concluded that final data would not likely provide evidence of significant effect, though Grupp said specific findings have yet to be revealed to Cephalon. There were no safety concerns.
The study was being conducted by the Parkinson Study Group, a nonprofit, cooperative faction of Parkinson's disease practitioners in the U.S. and Canada. The drug, an apoptosis inhibitor of receptor tyrosine kinase (RTK), is designed to stop the death of neurons in the brain, the process by which Parkinson's leads to lower dopamine levels and causes its neurodegenerative effects. Grupp said CEP-1347 would have represented a first-in-class approach to treating the disease, had it reached the market. He added that he was unaware of other companies that had advanced similar compounds for Parkinson's into late-stage studies.
Nevertheless, the small molecule still could have life. Cephalon owns its rights, and while Grupp declined to say whether the company has researched its use in other programs, he said "we haven't determined its future."
Terms of the multiyear partnership with Lundbeck, of Copenhagen, Denmark, called for Cephalon to market CEP-1347 in the U.S. and Lundbeck to market it in Europe. The companies plan to continue their collaboration in other undisclosed areas.
When the companies first reached their agreement, Cephalon received $14.4 million in up-front funding for the potential $40 million deal. CEP-1347 is the RTK inhibitor program's lead molecule. (See BioWorld Today, June 2, 1999.)
"The partnership had been going well," Grupp said, "and we'd like to see that continue."
Beyond the setback, Cephalon recently reported positive news. Good Phase III results on Oravescent fentanyl prompted it to announce last week that plans are on track for filing a new drug application to the FDA next quarter. (See BioWorld Today, May 6, 2005.)
Cephalon markets three products in the U.S.: Provigil (modafinil), Gabitril (tiagabine hydrochloride) and Actiq (oral transmucosal fentanyl citrate), as well as more than 20 other products internationally. On Thursday, its shares (NASDAQ:CEPH) fell 44 cents to close at $45.13.