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Scientific Data on PI3K/mTOR Inhibitor VS-5584 Published in Molecular Cancer Therapeutics

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Feb 6, 2013 - 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 publication of data for VS-5584 in Molecular Cancer Therapeutics.

VS-5584 is a potent and selective inhibitor of the PI3K/mTOR pathway. Previous research has described a central role of this pathway in cancer stem cells. The current study details the biochemical properties and preclinical effects of this compound in multiple human tumor models.

“The PI3K/mTOR signaling pathway appears critical for the survival and self-renewal of cancer stem cells,” said Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research. “We have established a broad platform to assess cancer stem cell inhibition in multiple types of human cancer. VS-5584 has been one of the most impressive cancer stem cell inhibitors we have seen to date across these assays.”

VS-5584 demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual-inhibitory actions against mTORC1 and mTORC2. The compound was profiled across >400 kinases covering all major families of the human kinome. In addition to the biochemical effects, VS-5584 exhibited broad anti-tumor efficacy, including tumor regression, across multiple human tumor models in the published study.

“PI3K and mTOR inhibitors continue to show promise for the treatment of cancer,” said Dr. Jose Baselga, member of the Verastem Scientific Advisory Board. “We have gained clinical experience with PI3K and mTOR inhibitors in recent years, and it appears that the dual blockade of PI3K and mTOR may be synergistic for maximum clinical benefit.”

VS-5584 is an orally available compound with favorable pharmacokinetics that induces long-lasting and dose-dependent inhibition of PI3K/mTOR signaling in tumor tissue.

“We are currently evaluating VS-5584 in IND-enabling toxicity studies prior to entry into a Phase 1 trial,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “We anticipate initiating clinical testing of VS-5584 during the second half of 2013 in patients with advanced cancers. In addition to safety, the study will evaluate initial signs of clinical activity and cancer stem cell inhibition.”

The publication is titled “VS-5584, a Novel and Highly Selective PI3K/mTOR Kinase Inhibitor for the Treatment of Cancer” and can be accessed at:

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

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 PI3k/mTOR 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-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, 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.

Contact: Verastem, Inc.
Investor contact:
Brian Sullivan, 617-252-9314
Media contact:
Kari Watson, 781-235-3060


Posted: February 2013