West Coast Editor
If a recent survey of 100 hematologists and oncologists is any indicator, then Vidaza (azacitidine), Pharmion Corp.'s pyrimidine nucleoside analogue approved in May, could be poised to snag a major share of the myelodysplastic syndromes market - but there's room for other players, too.
"This is an unknown market, to a large degree, by physicians and Wall Street, both," said Christopher Raymond, of Robert W. Baird & Co. in Chicago, who issued a research note Monday featuring results from the survey by CancerConsultants Inc.
The poll found 76 percent of surveyed physicians already have used the subcutaneously delivered Vidaza. At the same time, only 11 percent of MDS patients actually have received the drug from Vidaza prescribers, which means the likes of upcoming products Dacogen and Revlimid could make inroads if approved.
Dacogen (decitabine), from SuperGen Inc., of Dublin, Calif., and Minneapolis-based MGI Pharma Inc. is expected to get an FDA decision by Sept. 1. Revlimid (lenalidomide), from Celgene Corp., of Summit, N.J., is the subject of a new drug application planned this quarter.
Celgene in early February published in the New England Journal of Medicine positive data from a Phase I/II trial showing 24 of the 36 evaluable MDS patients achieved an erythroid response to the drug, defined by at least a 50 percent decrease in transfusions or an increase in hemoglobin of 1 g/dl.
Another potential drug for MDS, a pre-leukemic disorder, that's on the horizon is Johnson & Johnson's oral farnesyl transferase inhibitor Zarnestra (tipifarnib), on which the FDA is expected to act in the second half of this year.
Vidaza is "top of mind" in the survey conducted by CancerConsultants, with 49 percent of physicians naming Vidaza as their preferred front-line therapy, Raymond said. Twelve percent liked Revlimid and 5 percent Dacogen. During its first six months of sales, Vidaza sold $47 million.
Still strong in the MDS market is Celgene's Thalomid, preferred by 16 percent of surveyed physicians in the front-line setting - and a leading 30 percent in second-line treatment.
"That was the big surprise to me, how much use is happening in Thalomid already," Raymond told BioWorld Today, noting that Revlimid has a "very similar mechanism of action" and, if approved, could cannibalize Thalomid, which is approved for leprosy but used off label in MDS.
Lack of an FDA-approved label for MDS hasn't hurt Thalomid, Raymond said.
"The fact that it's not approved in multiple myeloma, yet is standard of care and is even reimbursed by Medicare [for that indication], is pretty telling," he said.
In MM, too, Thalomid is holding its ground against Velcade (bortezomib) from Cambridge, Mass.-based Millennium Pharmaceuticals Inc., he said.
"The trend we thought we started to see with Velcade overtaking Thalomid in front-line MM hasn't happened," Raymond said. "It's starting to reverse actually, which is shocking, since we saw a lot of good front-line data" at the American Society of Hematology meeting in San Diego in December.
"I would speculate that you've got a reimbursement algorithm this year that doctors are still feeling their way around, trying to figure it out," Raymond said, noting that other injectable oncology drugs are getting the same scrutiny.
"Or maybe Millennium has taken more time than we thought to get the word out on the ASH data," he said.
In the MDS indication, Boulder, Colo.-based Pharmion said during its fourth-quarter conference call in February that 70 percent of the usage of Vidaza (which is labeled for all MDS classes) has been in high-risk patients. By the end of 2004, a total of 2,500 had been treated.
Raymond said the survey is "the first of many we'll be doing" on MDS, as the market evolves. The next step is to ask physicians their plans for prescribing against high-risk vs. low-risk MDS.
Meanwhile, "you've got very differing mechanisms and routes of administration" among the prospective treatments, he said.
"Combination use will ultimately pop into the heads of some investigators who will do studies," Raymond said, and the growing market has room for more therapies. "It seems to be getting larger in terms of the overall opportunity," he said.