Verastem Initiates Clinical Trial of Dual mTOR/PI3K Inhibitor

Verastem Initiates Clinical Trial of Dual mTOR/PI3K Inhibitor
Phase 1 dose escalation and schedule finding trial in solid tumors and lymphomas 

CAMBRIDGE, MA - Dec. 10, 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 initiation of a Phase 1 trial of VS-5584, a dual mTORC1/2 and PI3K inhibitor, in patients with advanced solid tumors or lymphoma.

"Initiation of the clinical development of VS-5584 is an important milestone for Verastem," said Dr. Joanna Horobin, Verastem Chief Medical Officer. "We are conducting this study with clinical investigators who have extensive experience with PI3K and mTOR inhibitors and applying our expertise in cancer stem cell biology to progress the development of the compound."

Verastem presented preclinical research at the American Association of Cancer Research and the AACR-NCI-EORTC Molecular Targets Meetings in 2013 on the ability of VS-5584 to preferentially target cancer stem cells. Study data demonstrated that VS-5584 has equipotency against all four human Class I PI3K isoforms and both the mTORC1 and mTORC2 complexes of the mTOR kinase. VS-5584 decreased cancer stem cells across multiple in vitro and in vivo cancer models including triple negative breast cancer, small cell lung cancer and ovarian cancer. This is in contrast to the effect of standard of care treatments like chemotherapy which increase the proportion of cancer stem cells in these studies.

"Recent research by my laboratory and others has demonstrated that the dual inhibition of both PI3K and mTOR may be necessary to reduce the observed resistance to PI3K-selective inhibitors in solid tumors," said José Baselga, M.D., Ph.D., Verastem Senior Medical Advisor and Physician-in-Chief at Memorial Sloan-Kettering Cancer Center. "The initiations of clinical studies like this one are important next steps in the effort to identify potential new ways to address this resistance."

The Phase 1 open-label, dose escalation and schedule finding study is designed to assess the safety, pharmacokinetics, pharmacodynamics and initial clinical activity of single agent VS-5584. VS-5584 is an orally-available compound. The study will enroll up to 62 patients.

"2013 has been an important year of clinical execution for Verastem," said Robert Forrester, Verastem President and Chief Executive Officer. "Initiation of this Phase 1 trial for VS-5584 marks the third compound into the clinic and sixth clinical trial that we have started this year. We have made significant progress in the development of novel drugs targeting cancer stem cells and we continue to work diligently towards our goal of bringing new treatment options for patients. We look forward to 2014 when we expect additional clinical data from these trials will become available."

About VS-5584
VS-5584 is an oral compound that potently and selectively inhibits the class 1 PI3K isoforms, mTORC1 and mTORC2. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in multiple tumor models. VS-5584 is currently being evaluated in a Phase 1 dose escalation and schedule finding trial in patients with advanced solid tumors or 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-5584 and the Company's mTOR/PI3K program, 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, including the Phase 1 trial of VS-5584. 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 the Company will be unable to successfully complete the clinical development of its compounds, including 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, 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
[email protected]