Celgene ($CELG) took a big step toward a possible approval of its multiple myeloma drug pomalidomide today, saying the drug hit its primary endpoint for progression-free survival as well as a key secondary endpoint on overall survival. The data were "highly statistically significant and clinically meaningful," noted Celgene, which is pushing for an approval to use the drug among treatment-resistant patients. The safety review board recommended that patients in the control arm whose myeloma hadn't progressed should be provided the experimental drug combo.
The Phase III success greatly improves the odds for pomalidomide, a new formulation of thalidomide now in front of regulators in the U.S. as a last-ditch regimen for patients who had already failed at least two other treatments. An approval would set up pomalidomide as a likely follow-up therapy for patients who no longer responded to its blockbuster drug Revlimid as well as Velcade. The FDA has until February 10 to make its marketing decision on the drug, which was tested in combination with a low-dose of dexamethasone compared to a high-dose dexamethasone arm.
Piper Jaffray gives the drug good odds at hitting the billion-dollar blockbuster mark, especially if it is positioned well against Onyx's ($ONXX) newly-approved Kyprolis, now approved for the same refractory market that Celgene has its eye on.
"The survival results in this study build on earlier observations of high response rates for pomalidomide and dexamethasone in multiple myeloma patients who had been exposed to multiple therapies, including immunomodulatory agents and proteasome inhibitors," said Dr. Jesus San Miguel, director of the Biomedical Research Institute of Salamanca and principal investigator in the study. "The continued progress of new agents in this area of disease, particularly in later-stage patients is critical as we look to extend remissions and survival for these individuals."
Late last year Celgene reported that pomalidomide combined with the corticosteroid triggered a 34% partial response among patients in Phase II, compared to 13% on pomalidomide alone. The FDA had initially planned a Nov. 8 panel review for the experimental treatment but Celgene noted that the agency recently canceled that session.
- here's the press release
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