Pomalidomide Meets Primary PFS Endpoint

Jesus San Miguel, MD, PhD

A data safety monitoring board (DSMB) has found that a phase III study of pomalidomide, an oral immunomodulatory agent, met its primary endpoint by demonstrating a significant improvement in progression-free survival for patients with multiple myeloma (MM).

According to the drug’s developer, Celgene International Sàrl, the DSMB determined that study MM-003 demonstrated statistically significantly improved progression- free survival (PFS) in patients with relapsed and/or refractory MM who took pomalidomide with low-dose dexamethasone, in comparison with those who took high-dose dexamethasone alone. Pomalidomide, which also inhibits angiogenesis, is a derivative of thalidomide.

In addition, at the overall survival (OS) interim analysis, the DSMB found that the multicenter, randomized, openlabel study crossed the superiority boundary for OS, a key secondary endpoint the trial was also powered to evaluate.

Based on those results, the DSMB recommended that patients in the dexamethasone arm who had not yet progressed should cross over to pomalidomide.

Celgene called the data “highly statistically significant and clinically meaningful.”

“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.”,” said principal investigator Jesus San Miguel, MD, PhD, head of the Department of Hematology at the University of Salamanca, Spain.

The FDA has accepted Celgene’s New Drug Application for pomalidomide, with a decision expected by February 10, 2013. The company also anticipates a European Medicines Agency decision in the second half of 2013.