Verastem Initiates Phase 1/1b Clinical Trial of VS-6063 Plus Paclitaxel for Patients with Ovarian Cancer

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, announced the initiation of a Phase 1/1b trial of VS-6063 in combination with paclitaxel for the treatment of advanced ovarian cancer.

VS-6063 is a potent inhibitor of focal adhesion kinase (FAK) and has demonstrated signs of clinical activity in ovarian cancer in a single agent, Phase 1 clinical trial.

“In the Phase 1 study we demonstrated that VS-6063 as a single agent was generally well tolerated, giving us optimism that this novel agent can be combined with the widely used drug paclitaxel," said Principal Investigator Howard “Skip” Burris, III, M.D., Chief Medical Officer and Executive Director, Drug Development Program, Sarah Cannon Research Institute, Nashville, TN. "Moreover, clinically meaningful disease stabilization for about 6 months was observed in 3 of 4 patients with ovarian cancer treated with a dose of VS-6063 in the range of expected activity. All of these patients had received multiple lines of prior chemotherapy, including platinum-based treatment which has been shown to preferentially select chemo-resistant cancer stem cells."

Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board, has demonstrated that the FAK pathway is a critical component for the growth and survival of cancer stem cells, which are an underlying cause of tumor metastasis and recurrence.

“We believe that cancer stem cells are ultimately responsible for disease progression with ovarian and other cancers,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Through FAK inhibition, we have the potential to provide more durable clinical responses for these diseases. Verastem has advanced the science of targeting cancer stem cells so that we can now clinically evaluate the therapeutic benefit of this approach in the treatment of cancer.”

The Phase 1b trial is an open-label, multicenter, dose-escalation trial of VS-6063 in combination with paclitaxel. The endpoints of the study are safety, tolerability and efficacy as determined by Response Evaluation Criteria in Solid Tumors (RECIST) and proprietary cancer stem cell biomarkers. The study will enroll up to 30 patients with ovarian cancer at three U.S. locations.

“Our unique understanding of cancer stem cell biology, coupled with our focus on execution in research and development, has allowed us to build the leading portfolio of FAK inhibitors,” said Christoph Westphal, M.D., Ph.D., Verastem Chairman and Chief Executive Officer. “The VS-6063 combination study marks the first of three trials we have planned for the FAK inhibitor program in 2013, including a potentially pivotal study in mesothelioma expected to begin mid-year.”

For more information on the trial, please visit: http://clinicaltrials.gov/ct2/show/NCT01778803.

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 www.verastem.com.

About VS-6063

VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK) which is a critical pathway for cancer stem cells. VS-6063 was well-tolerated and demonstrated signs of clinical activity in a Phase 1 study in advanced solid tumors. VS-6063 is currently in a Phase 1/1b study in patients with advanced ovarian cancer, where VS-6063 is being tested in combination with paclitaxel. VS-6063 is anticipated to enter a potentially pivotal study in mesothelioma midyear 2013.

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 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,” “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 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, 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.