Defactinib in Combination with Weekly Paclitaxel Achieves a Complete Response in Platinum-Resistant Ovarian Cancer

CAMBRIDGE, MA - July 23, 2013 - Verastem, Inc., (NASDAQ: VSTM) focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, today announced the confirmation of a complete response in an ongoing trial of VS-6063 (defactinib) in combination with weekly paclitaxel.

At Verastem's Research and Development Day on July 11th , Dr. Horobin, Chief Medical Officer, presented preliminary results describing a normalization of CA-125, a biomarker of ovarian cancer progression, and an initial radiologic complete response for one of the patients in the trial. Response Evaluation Criteria in Solid Tumors, or RECIST, is the standard by which investigational drugs are evaluated for activity in oncology studies.

"We have now confirmed the previously reported complete response by RECIST criteria with a second radiographic scan at the end of cycle four," said Dr. Joanna Horobin. "The CA-125 level in this subject with platinum-resistant ovarian cancer normalized after the first cycle of treatment and has remained at normal levels since. We are encouraged by this early result and will continue to follow all patients in this study closely. We plan to report an update on the results of this study at scientific conferences later this year."

The Phase 1 dose escalation portion of the study in six patients treated at the Sarah Cannon Research Institute was well tolerated at all dose levels with no exacerbation of the well-known adverse effects of paclitaxel.

In a randomized, placebo controlled study presented by McNeish et. al. at the 2013 ASCO annual conference, only 1 complete response was reported in a total of 107 patients with platinum-resistant ovarian cancer treated with weekly paclitaxel alone or weekly paclitaxel plus study drug.

Verastem recently initiated the expansion Phase 1b stage of the study and expects to enroll an additional 15 patients with ovarian cancer at three U.S. locations. The Phase 1b trial is an open-label, multicenter, trial of defactinib 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.

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, and the Company's FAK and diagnostic programs generally, the timeline for clinical development and regulatory approval of the Company's compounds, expectations on patient enrollment in clinical studies, 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 and preliminary data from clinical trials may not be predictive of the results or 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, 2012 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. 

Contact Verastem, Inc.

Brian Sullivan, 617-252-9314

[email protected]

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