Verastem, Inc., (NASDAQ: VSTM) a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, reported new data from its focal adhesion kinase (FAK) inhibition program and commented on encouraging clinical data on FAK inhibition presented at the 2012 EORTC Symposium on Molecular Targets and Cancer Therapeutics being held in Dublin, Ireland.
Data presented by Verastem in breast cancer models lacking the tumor suppressor Merlin demonstrate that FAK inhibition effectively reduces cancer stem cells in vitro and in vivo. Oral administration of a FAK inhibitor as a single agent was shown to induce tumor regression in a Merlin-negative breast cancer mouse model. These data extend previous research from Verastem, which demonstrated similar inhibition of cancer stem cells and strong single agent efficacy in Merlin-negative mesothelioma models.
Several groups presented data at the conference on the use of FAK inhibitors for treatment of cancer. In one clinical study by GlaxoSmithKline, researchers demonstrated that Merlin loss may identify a subset of patients with improved progression free survival in response to FAK inhibition. “These results provide important clinical validation of our internal research demonstrating enhanced sensitivity of Merlin-negative mesothelioma to FAK inhibitors,” said Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research.
Verastem anticipates initiating a potentially pivotal study in mesothelioma midyear 2013.“The sum of the data on development of FAK inhibitors presented at this conference is very promising,” said Professor Dean Fennell, Chair of Thoracic Medical Oncology, University of Leicester. “In particular, GSK reported in a Phase 1 trial novel activity of a FAK inhibitor in mesothelioma, where treatment in the second and third-line setting resulted in a median progression free survival of 17.7 weeks.” The largest clinical trial to date in malignant pleural mesothelioma was a 2011 Phase 3 study of Zolinza ® as a second- and third-line treatment conducted by Merck in 660 patients. In this study, a median progression free survival of only 6 weeks was observed. “Interestingly, loss of the tumor suppressor Merlin correlated with increased clinical sensitivity to FAK inhibition,” Prof. Fennell continued. “A subset of patients with Merlin negative mesothelioma had more than double the median progression free survival of 24.1 weeks versus 11.4 weeks for the Merlin positive group. These early results suggest that a targeted therapy, particularly when used in combination with a specific biomarker, has the potential to significantly improve treatment of this aggressive and deadly disease.”