Does anyone even say, “Hot off the presses!” anymore? My contact at Celgene just forwarded me this news about pomalidomide five minutes ago:
POMALIDOMIDE DEMONSTRATED SIGNIFICANT PROGRESSION-FREE SURVIVAL AND OVERALL SURVIVAL ADVANTAGES IN PHASE III STUDY OF HEAVILY PRE-TREATED MYELOMA PATIENTS
BOUDRY, Switzerland – (Oct. 23, 2013) Celgene International Sàrl, a subsidiary of Celgene Corporation (NASDAQ: CELG) today announced that its phase III, multi-center, randomized, open-label study (MM-003) of pomalidomide plus low-dose dexamethasone compared to high-dose dexamethasone in patients with relapsed and/or refractory multiple myeloma was reviewed by a data safety monitoring board (DSMB). The DSMB determined MM-003 met the primary endpoint of improvement in progression-free survival (PFS) at the PFS final analysis. Additionally, at the OS interim analysis, the study crossed the superiority boundary for overall survival (OS), a key secondary endpoint that the study was also powered to evaluate. Improvements in PFS and OS were both highly statistically significant and clinically meaningful. As a result, the DSMB recommended that patients who had not yet progressed in the high-dose dexamethasone arm should be crossed-over to the pomalidomide plus low-dose dexamethasone arm.
Safety results observed in MM-003 were consistent with previous studies of pomalidomide in relapsed/refractory multiple myeloma patients. Full data from the study are being prepared for submission to a future medical meeting for presentation.
“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, Head of the Department of Hematology at the University of Salamanca, 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.”
The MM-003 study compared pomalidomide plus low-dose dexamethasone to high-dose dexamethasone in patients who were relapsed on or refractory to at least two prior therapies that must have included both lenalidomide and bortezomib.
Patients in the pomalidomide arm received 4 mg of oral pomalidomide on days 1-21 of each 28-day cycle, with patients 75 years or younger also receiving 40 mg of oral low-dose dexamethasone and patients older than 75 years receiving 20 mg oral low-dose dexamethasone on days 1, 8, 15 and 22 of each 28-day cycle, until disease progression.
Patients in the comparator arm 75 years and younger received 40 mg oral high-dose dexamethasone on days 1-4, 9-12 and 17-20 of each 28-day cycle, and patients older than 75 years received 20 mg oral high-dose dexamethasone on days 1-4, 9-12 and 17-20 of a 28-day cycle, until disease progression.
The primary endpoint of the study was PFS, with the key secondary endpoint being OS (with alpha control), and other secondary endpoints including safety, response rates and time to progression.
A Marketing Authorisation Application (MAA) for pomalidomide in combination with dexamethasone was submitted to the EMA in May 2012 and Celgene anticipates a decision by European regulatory authorities in the second half of 2013.
Additionally, a new drug application (NDA) has been accepted for review by the U.S. Food and Drug Administration, with a Prescription Drug User Fee Act (PDUFA) date of Feb. 10, 2013.
These results are from an investigational study. Pomalidomide is not approved for the treatment of any indication.
Pomalidomide is an IMiDs® compound. Pomalidomide and other IMiDs compounds continue to be evaluated in over 100 clinical trials. The IMiDs compound pipeline is covered by a comprehensive intellectual property estate of issued and pending patent applications in the US, EU and other regions.
I wrote in my new book, New Multiple Myeloma Therapies from a Patient’s Perspective, that “Pomalidomide is a game changer.”
Keep in mind that European regulators are a lot tougher and more conservative than the FDA. It may seem strange, but they still use a lot of “dex only” and Thalomid therapies in Europe. If pom can meet stringent safety guidelines and PFS/OS criteria in Europe, that bodes well for FDA approval over here.
It isn’t a cure, but pomalidomide should join Revlimid and Velcade as cornerstones of myeloma therapy.
Feel good and keep smiling! Pat