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Promising Clinical Data on VS-6063 for the Treatment of Mesothelioma Presented at the 2014 iMig Conference

11-03-2014

On October 24, 2014, at the iMig ( International Mesothelioma Interest Group) Conference, Professor Raphael Bueno, M.D., Chief of Thoracic Surgery, Brigham and Women’s Hospital (BWH), presented preliminary data on Verastem, Inc.’s clinical ‘Window of Opportunity’ study evaluating the biomarker response to VS-6063 (defactinib), an oral small molecule that targets cancer stem cells through the inhibition of focal adhesion kinase (FAK) in pleural mesothelioma patients (mpm) eligible for resection surgery.

The drug was administered to 10 patients twice a day, for 12 days, with biopsies performed before and after the administration of the drug. Tumor size was measured using CT/ PET scans before and after the 12-day administration of VS-6063.

VS-6063 reduced FAK activity by an average of 70%, and in 5 of the 7 patients reduced the presence of cancer stem cell markers in the post-treatment biopsies. None of the 10 patients saw any tumor growth in the 12 days, and 2 of the 10 patients experienced partial tumor shrinkage of -30%, and -49%.

“To follow up on these encouraging results, the protocol is being amended to explore more extended dosing in 10-15 additional patients with mesothelioma prior to surgery,” said Dr. Bueno. “The window of opportunity before elective surgery provides a novel platform for the clinical evaluation of promising new agents for the treatment of this devastating disease.”

In addition to the ‘Window of Opportunity’ study, Verastem is conducting a registration-directed COMMAND (Control Of Mesothelioma with MAinteNance Defactinib) study as a disease maintenance program following successful treatment with Alimta and cisplatin in patients with mpm.

Verastem also presented preclinical data at the iMig Conference which demonstrated the ability of VS-6063 and the combination of VS-6063 and VS-5584 to target and kill cancer stem cells in models of mesothelioma.

The administration of drugs prior to surgery to suppress tumor growth is not new. Many patients undergo chemotherapy before surgery in an effort to reduce tumor size and minimize complications. The possibility of inhibiting the disease’s production of a particular biomarker linked directly to tumor growth is very promising.

Verastem’s approach to developing targeted therapies for mesothelioma has the potential to not only improve treatment of this aggressive and deadly disease, but to offer new hope to patients struggling with their disease.

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