West Coast Editor
With approval pending that would grant Thalomid a label for multiple myeloma, Celgene Corp. said two Phase III trials with Revlimid in the same indication "overwhelmingly exceeded" the prespecified efficacy endpoint for stopping the trials.
Celgene's stock (NASDAQ:CELG) closed Monday at $33.88, up $5.20, or 18.1 percent. Analysts at Prudential Financial upgraded the stock to "overweight" but Robert W. Baird & Co. remained neutral pending more detailed data from the trials, though Baird did raise its price target on Celgene's shares to $29. C.E. Unterberg, Towbin was even more optimistic, upping the price target from $35 to $41.
"The results today certainly exceeded my expectations," said Matt Osborne, analyst with C.E. Unterberg, Towbin in Boston. An independent data monitoring committee found data from both Phase III trials surpassed the p<0.0015 value in the primary endpoint, which is time to disease progression.
Celgene will continue the trials to measure the secondary endpoint, survival, with more data due in the second half of the year. Top-line results might be offered at the American Society of Clinical Oncology meeting in May, Osborne said.
"Certainly, last year was kind of a coming-out party for myeloma at ASCO, and this could work its way into a late-breaker session," he said.
The trials, being conducted under a special protocol assessment with the FDA, compared results in patients getting Revlimid (lenalidomide) plus dexamethasone with those given dexamethasone alone for relapsed or refractory MM.
Revlimid's preliminary safety profile was said to be favorable, and talks have begun with the FDA regarding the next steps.
Conducted at 97 international sites, the trials enrolled 705 patients. The primary endpoint was calculated as the time from randomization to the first documentation of progressive disease, based on the Blade myeloma response criteria.
Last fall, the FDA sent Celgene an approvable letter for Thalomid (thalidomide) in multiple myeloma. Physicians long have prescribed the drug, approved for leprosy, for MM. Celgene asked for official approval in late 2003, but Thalomid - even without the label - has been competing well with Velcade (bortezomib) from Cambridge, Mass.-based Millennium Pharmaceuticals Inc. (See BioWorld Today, Oct. 26, 2004, and March 3, 2005.)
Celgene is expected to reply to the FDA's approvable letter this quarter, and the drug could be cleared for marketing in the second half of the year.
"The question is how quickly and to what extent Revlimid will cannibalize Thalomid," Osborne told BioWorld Today. "Feedback from physicians suggests that, early on at least, they're willing to try Revlimid," and the drug likely will "be used pretty quick" for relapsed/refractory disease.
"Personally, my numbers were a little heavier for cannibalization, in terms of the rest of The Street," he said, noting that the company expects the drugs to be used in combination so that "Thalomid will be around for a while."
He pointed to the STEPS (Systems for Thalomid Education and Prescription Safety) program, with which doctors must sign up in order to use Thalomid.
"They have to continually enroll every month, which is difficult for physicians, especially if they have a drug with fewer side effects," he said.
Yaron Werber, analyst with Citigroup Smith Barney in New York, said the data so far suggest Revlimid can be used after a patient has failed Thalomid, "and that's probably how it's going to be used initially."
Tests are under way to determine whether Revlimid is appropriate as a front-line therapy, in which (in combination with dexamethasone) it will probably be used, Werber told BioWorld Today.
"Chances are, Thalomid is going to be kicked out," he said, and given to patients who failed Revlimid or Velcade.
Thalomid also is being tested against renal-cell carcinoma.
"We haven't heard much from that area," Osborne said. "It was the second strategy for getting Thalomid approved." About 16 percent of Thalomid is used off label for renal-cell cancer, he said.
In any case, the most recent disclosure about trial data impressed Werber.
"I did not think the trial was powered to show this magnitude of difference," he said, citing the "fairly high" statistical bar.
Revlimid with dexamethasone is showing "more than a doubling of the time to disease progression," he said, and analysts will be watching for the trial's secondary endpoint, although "there's going to be a crossover that will complicate the survival outlook a little bit," since all patients will be transferred to the combination arm.
MM, also known as myeloma or plasma-cell myeloma, is the second most common blood cancer, characterized by overproduction of malignant plasma cells in the bone marrow. Also this quarter, the company plans to file a biologics license application for Revlimid against myelodysplastic syndrome.