Pomalyst pomalidomide: Updated Phase III data

Updated data from the open-label, international Phase III MM-003 trial in 455 patients with relapsed and/or refractory MM showed that oral Pomalyst plus low-dose dexamethasone led to a median PFS, the primary endpoint, of 4 months vs. 1.9 months for high-dose dexamethasone at a median follow-up of 10 months (p<0.0001). Additionally, Pomalyst plus low-dose dexamethasone led to a median OS, a secondary endpoint, of 12.7 months vs. 8.1 months for high-dose dexamethasone (p=0.028). The trial enrolled patients who relapsed on or were refractory to >=2 prior therapies that must have included Revlimid lenalidomide and Velcade bortezomib. Patients received once-daily 4 mg Pomalyst on days 1-21 every 28 days plus 20 (>75 years old) or 40 mg (<=75 years old) dexamethasone on days 1, 8, 15 and 22. Patients in the control arm received 20 (>75 years old) or 40 mg (<=75 years old) dexamethasone on days 1-4, 9-12 and 17-20 every 28 days. Data were presented at the American Society of Clinical Oncology meeting in Chicago.

Last October, an independent DSMB said Pomalyst plus low-dose dexamethasone met the primary endpoint of PFS vs. high-dose dexamethasone at a median follow-up of 4 months (3.6 vs. 1.8 months, p<0.001). Previously reported data also showed that median OS had not been reached in patients receiving pomalidomide plus low-dose dexamethasone vs. 7.8 months for high-dose dexamethasone (p<0.001) (see BioCentury, Oct. 29, 2012 & Dec. 17, 2012). Last February, FDA granted accelerated approval for Pomalyst to treat patients with MM who have received >=2 prior therapies including Revlimid and Velcade, and who exhibit disease progression on or within 60 days of completion of the last therapy (see BioCentury, Feb. 11). Last month, EMA's CHMP issued a positive opinion recommending approval of an MAA for Pomalyst in the indication (see BioCentury, June 3). The compound is in Phase III testing for myelofibrosis and systemic sclerosis, for which it has Orphan Drug designation in the EU. ...