Press Release

March 10, 2016
Verastem to Present Scientific Data Supporting FAK and PI3K/mTOR Inhibition to Target Cancer Stem Cells at the Keystone Symposium on Stem Cells and Cancer

BOSTON--(BUSINESS WIRE)--Mar. 10, 2016-- Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer, today announced the presentation of preclinical data and participation in an expert panel at the Keystone Symposium on Stem Cells and Cancer being held March 6 – 10, 2016 in Breckenridge, CO.

“The scientific consensus on the importance of targeting cancer stem cells to enable a durable clinical response continues to build,” said Dr. Jonathan Pachter, Verastem Head of Research. “Our presentation at the Keystone Symposium describes our current understanding of the role of PI3K/mTOR and FAK in the survival and tumor-initiating capability of cancer stem cells.”

“Of particular interest, we have also found that FAK inhibition increases influx of cytotoxic T cells into tumors while reducing immuno-suppressive and stromal density barriers to anti-tumor immune attack,” continued Dr. Pachter. “New data from preclinical models presented today demonstrate that FAK inhibition enhances the anti-tumor effect of adoptive T cell transfer. These data suggest that the benefit of FAK inhibitor combination is likely to extend to various approaches that enhance cytotoxic T cell function, including combination with antibodies against PD-1 and PD-L1. We are now clinically testing this with the combination of VS-6063 and pembrolizumab at Washington University in Saint Louis in patients with advanced pancreatic cancer. We also recently announced a clinical collaboration with Pfizer and Merck KGaA to combine VS-6063 and avelumab for the treatment of patients with ovarian cancer.”

Details for the presentation at the Keystone Symposium on Stem Cells and Cancer are as follows:

Oral Presentation and Panel

Title: Targeting Cancer Stem Cells with Selective Inhibitors of FAK and PI3K/mTOR

Session: Targeting Cancer Stem Cells: Trials and Translation

Date and time: Thursday, March 10, 2016, 8:00 – 11:00 am PT

A copy of the oral presentation will be available following the presentation at http://bit.ly/R3M6wc.

About Focal Adhesion Kinase

Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for patients with cancer.

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 dose escalation trial of VS-5584 in patients with advanced solid tumors.

About Verastem, Inc.

Verastem, Inc. (NASDAQ:VSTM) is a biopharmaceutical company focused on discovering and developing drugs to improve outcomes for patients with cancer. Our product candidates utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. Our most advanced clinical product candidates are the Focal Adhesion Kinase inhibitors, VS-6063 and VS-4718, and the dual PI3K/mTOR inhibitor, VS-5584. For more information, please visit www.verastem.com.

Verastem forward-looking statements notice:

This press release includes forward-looking statements about Verastem’s strategy, future plans and prospects, including statements regarding the development and activity of Verastem’s product candidates, VS-6063, VS-4718 and VS-5584, and Verastem’s FAK, PI3K/mTOR and diagnostics programs generally, the utility of FAK inhibitors for the treatment of cancer, the timeline for clinical development and regulatory approval of our product candidates, the structure of our planned or pending clinical trials, our rights to develop or commercialize our product candidates and our ability to finance contemplated development activities and fund operations for a specified period. 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 Verastem’s product candidates and preliminary or interim 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 our product candidates will cause unexpected safety events, that Verastem will be unable to successfully initiate or complete the clinical development of its product candidates, that the development of Verastem’s product candidates will take longer or cost more than planned, and that Verastem’s product candidates will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading “Risk Factors” in Verastem’s Annual Report on Form 10-K for the year ended December 31, 2015 and in any subsequent SEC filings. The forward-looking statements contained in this press release reflect Verastem’s current views with respect to future events, and Verastem does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Source: Verastem, Inc.

Verastem, Inc.
Brian Sullivan, 781-292-4214
bsullivan@verastem.com