Verastem Reports Data On Focal Adhesion Kinase Program At The 2012 EORTC Symposium On Molecular Targets And Cancer Therapeutics

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.”

“We are encouraged by the initial clinical data presented today by GSK,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Merlin loss occurs in approximately 50% of mesothelioma patients and we believe that VS-6063 has potential as a targeted therapy in this disease. We are moving rapidly to initiate a potentially pivotal, double blind controlled study next year.”

FAK is a cytoplasmic tyrosine kinase that mediates signal transduction by integrins and growth factor receptors. FAK has been implicated in different steps of tumor development including tumor initiation, growth, angiogenesis and metastasis. Verastem has pioneered research on FAK as a critical regulator of cancer stem cells using technology developed by cofounder and chair of the Scientific Advisory Board, Robert Weinberg, Ph.D.

The poster presentation by Verastem is titled: “FAK Inhibitor VS-4718 Attenuates Breast Cancer Stem Cell Function and Inhibits Tumor Growth in Vivo.”

The poster presentation by GlaxoSmithKline is titled: “Loss of the Tumor Suppressor Merlin as a Potential Predictive Biomarker of Clinical Activity for the Oral, Focal Adhesion Kinase (FAK) Inhibitor GSK2256098 in Pts with Recurrent Mesothelioma.”

About VS-6063

VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK). FAK is critical for the survival of cancer stem cells, which are an underlying cause of tumor recurrence and metastasis. VS-6063 was well-tolerated and demonstrated signs of clinical activity in a Phase 1 study in advanced solid tumors. Verastem has demonstrated that loss of the tumor suppressor Merlin confers increased susceptibility to FAK inhibition. Verastem is planning to initiate multiple clinical trials in 2013 with VS-6063, including a potential registration study in mesothelioma, which will evaluate patient response based on Merlin status.

About Verastem, Inc.

Verastem, Inc. (NASDAQ: VSTM) is a clinical-stage biopharmaceutical company focused on 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

Zolinza ® is a registered trademark of Merck, Inc.

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 and VS-4718, and the Company’s FAK and diagnostic programs generally, the timeline for clinical development and regulatory approval of the Company’s compounds and the structure of the Company’s planned clinical trials. The words “anticipate,” “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 may not be predictive of the success of later clinical trials, that the Company will be unable to successfully complete the clinical development of its compounds, including VS-6063 and VS-4718, 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, 2011 and in any subsequent SEC filings. The forward-looking statements contained in this presentation 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.

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