Press Release

October 8, 2014
Verastem to Present New Data at the 12th International Mesothelioma Interest Group Conference

NEEDHAM, Mass.--(BUSINESS WIRE)--Oct. 8, 2014-- Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, today announced that it will present new data at the 12th International Mesothelioma Interest Group (iMig) Conference being held October 22-24, 2014, in Cape Town, South Africa.

Investigators and members of the Verastem leadership team will give oral presentations discussing the Company’s development programs targeting cancer stem cells (CSC’s) with a focus on lead inhibitor VS-6063 (defactinib). VS-6063 targets CSC’s, a subpopulation of cancer cells that have tumor-initiating capability, are particularly resistant to chemotherapy, and can mediate tumor recurrence both locally and at metastatic sites. VS-6063 targets CSC’s through the potent inhibition of focal adhesion kinase (FAK). Research on the FAK signaling pathway has revealed its critical role in CSC survival and disease progression.

Professor Raphael Bueno, M.D., Chief, Division of Thoracic Surgery, Brigham & Women’s Hospital and principal investigator of Verastem’s ongoing “Window of Opportunity” study evaluating the effect of single agent VS-6063 on specific biomarkers in patients with mesothelioma undergoing surgery, will present data from the ongoing study.

“We are honored to have been selected for several presentations at this year’s iMig Conference and to participate with other industry and medical leaders in the fight against mesothelioma,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “These presentations, along with our ongoing development efforts with our lead candidate VS-6063, which is currently being studied in the global, registration-directed COMMAND study for patients with mesothelioma, demonstrate our commitment to growing the body of knowledge surrounding the treatment of this highly aggressive form of cancer.”

The schedule for the presentations is as follows:

iMig Special Keynote Lecture: “Cancer Stem Cells as Target Pathways”
Presenter: Robert Weinberg, Ph.D., Whitehead Institute and Verastem scientific cofounder and chair of the Scientific Advisory Board

Presentation Title: “Determination of Biomarker response in a Phase II Window of Opportunity Study of Defactinib (VS-6063), a Focal Adhesion Kinase (FAK) Inhibitor, in Subjects with Resectable MPM”
Presenter: Raphael Bueno, M.D., Chief, Division of Thoracic Surgery, Brigham & Women’s Hospital, Boston, MA

Presentation Title: “FAK Inhibitor VS-6063 (Defactinib) Targets Mesothelioma Cancer Stem Cells which are Enriched by Standard of Care Chemotherapy”
Presenter: Paul Baas, M.D., Ph. D., Department of Thoracic Oncology,The Netherlands Cancer Institute

Presentation Title: “The Cancer Stem Cell Inhibitors VS-6063 (Defactinib) and VS-5584 Exhibit Synergistic Anticancer Activity in Preclinical Models of Mesothelioma”
Presenter: Mitchell Keegan, Ph.D., Vice President, Development, Verastem

About Malignant Pleural Mesothelioma

Malignant pleural mesothelioma is an aggressive form of cancer that occurs in the mesothelium, the thin layer of tissue that covers the lungs. Mesothelioma is associated with exposure to asbestos in most cases. According to the World Health Organization, there are a total of 59,000 cases of mesothelioma worldwide each year. Most mesotheliomas begin as one or more nodules that progressively grow to form a solid coating of tumor surrounding the lung leading to eventual suffocation and death. A high percentage of mesotheliomas contain cancer stem cells which are generally resistant to the currently available treatment options for mesothelioma.

About VS-6063

VS-6063 (Defactinib) is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research by Robert Weinberg, Ph.D., scientific cofounder and chair of Verastem’s Scientific Advisory Board, and Verastem has demonstrated that FAK activity is critical for the growth and survival of cancer stem cells. VS-6063 is currently being studied in the registration-directed COMMAND trial in mesothelioma (, a “Window of Opportunity” study in patients with mesothelioma prior to surgery, a Phase 1/1b study in combination with paclitaxel in patients with ovarian cancer, and a trial in patients with Kras-mutated non-small cell lung cancer. VS-6063 has been granted orphan drug designation in the U.S. and EU for use in mesothelioma.

About VS-5584

VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in chemotherapy-resistant models. Verastem is currently conducting a Phase 1 dose escalation trial of VS-5584 in patients with advanced solid tumors and lymphomas.

About Verastem, Inc.

Verastem, Inc. (NASDAQ:VSTM) is discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation: FAK, PI3K/mTOR and Wnt. For more information, please visit

Forward-looking statements:

This press release includes forward-looking statements about the Company’s strategy, future plans and prospects, including statements regarding the development of the Company’s compounds, including VS-6063, or defactinib and VS-5584 and the Company’s FAK inhibition and PI3K/mTOR programs generally, the timeline for clinical development and regulatory approval of the Company’s compounds, the expected timing for the reporting of data from ongoing trials, and the structure of the Company’s planned or pending clinical trials. The words “anticipate,” “appear,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “will,” “would,” “could,” “should,” “continue,” and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the preclinical testing of the Company’s compounds and preliminary data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that data may not be available when we expect it to be, that enrollment of clinical trials may take longer than expected, that the Company will be unable to successfully complete the clinical development of its compounds, including VS-6063 and VS-5584, that the development of the Company’s compounds will take longer or cost more than planned, and that the Company’s compounds will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2013 and in any subsequent SEC filings. The forward-looking statements contained in this press release reflect the Company’s current views with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Source: Verastem, Inc.

Verastem, Inc.
Brian Sullivan, 617-252-9314