News Release

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 12, 2012-- Verastem, Inc., (NASDAQ: VSTM) a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells, hosted its annual Research and Development Day where Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board, gave a seminar on cancer stem cells. Members of the Company’s management team provided updates to the product portfolio and advancements in the research and development pipeline.

“In order to generate a durable, clinical response in cancer we must design a treatment strategy that attacks the cancer stem cells, in addition to the other cancer cells, that comprise a tumor,” said Dr. Weinberg. “We founded Verastem in order to create therapies designed to specifically target cancer stem cells as these cells are resistant to standard cancer treatments. Verastem has made significant strides in the identification and development of small molecules that target this cancer stem cell population.”

Yesterday, Verastem announced the acquisition of an exclusive license to VS-6063 (formerly PF-04554878) from Pfizer. VS-6063 is a focal adhesion kinase (FAK) inhibitor that has completed a Phase 1 clinical study in advanced solid tumors. VS-6063 was well tolerated and showed signs of clinical activity in the study.

“A key component of Verastem’s IPO was to raise sufficient capital to rapidly progress the development of novel compounds targeting cancer stem cells,” said Christoph Westphal, M.D., Ph.D., Verastem Chairman and Chief Executive Officer. “With the in-license of VS-6063 from Pfizer, we have accelerated our clinical development plans for the FAK program by 12-18 months.”

“Our research has identified and prioritized key pathways necessary for the survival of cancer stem cells and yielded specific inhibitors to block these critical targets,” said Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research. “We are now poised to translate that research into multiple clinical trials over the next year with our cancer stem cell-targeted agents.”

Research and Development Day Program Highlights:

Focal Adhesion Kinase (FAK) Inhibition

FAK is amplified in human cancers and mediates survival of cancer stem cells in response to growth factors and integrin stimulation

• Accelerated the FAK program by 12-18 months with the in-license of VS-6063 from Pfizer
  • VS-6063 has completed a Phase 1 study in advanced solid tumors, where it was well tolerated and demonstrated signs of clinical activity
  • VS-6063 is expected to enter into a Phase 2 mesothelioma study in mid-2013. A positive Phase 2 in mesothelioma could present an opportunity for an accelerated approval
• Loss of the protein Merlin (resulting from mutation or deletion of the NF2 gene) confers susceptibility to targeted FAK inhibition
  • Approximately 40% of mesothelioma tumors lack Merlin
  • Verastem’s FAK inhibitors have enhanced activity in cell lines and tumor models that are Merlin negative
• Cancer stem cells have been identified in 90% of human mesothelioma patient samples
• Nominated VS-4718 as a development candidate
  • IND-enabling toxicology studies initiated in June, 2012
  • Accelerated VS-4718 clinical development plan with a Phase 1 healthy volunteer study anticipated to initiate in Q1 2013

Dual PI3K/mTOR Inhibition

PI3K and mTOR are key enzymes in the AKT signaling pathway that promote cancer stem cell proliferation and survival

• PI3K/mTOR dual inhibitor (VS-5584) was in-licensed from S*Bio in Q2 2012

• VS-5584 demonstrated potent inhibition across all PI3K isoforms and mTORC1 and mTORC2, and selectivity for cancer stem cells
• Clinical development is planned to initiate mid-2013 in advanced solid tumors with a high percentage of cancer stem cells

Wnt Inhibition

The Wnt pathway is critical for the self-renewal and proliferation of cancer stem cells

• Deferred advancing VS-507 into clinical trials as a result of a portfolio review
• Established a research collaboration with Eisai to discover next-generation small molecule inhibitors of Wnt signaling

Companion Diagnostics

Verastem is using both drug specific and cancer stem cell-specific biomarkers to identify patients most likely to benefit from our drugs and monitor their response to treatment

• Successful assay development of Pharmacodynamic tests for FAK in tumor samples and platelets
• Pharmacodynamic and candidate cancer stem cell markers have been identified to progress into clinical testing with therapeutic product candidates

Conference Call and Webcast Information

A live webcast of the event can be accessed by visiting the investors section of the Company’s website at www.verastem.com. A replay will be available for two weeks from the date of the event.

A live, listen-only conference call of the event can be accessed by dialing 1-866-700-7173 five minutes prior to the start of the event and providing the passcode 73322380.

The details for the annual Research and Development Day are as follows:
Location: 215 First Street, Cambridge, MA, 02142
Date: July 12, 2012
Time: 9:00am – 12:00pm (ET)
RSVP: bsullivan@verastem.com
Conference Call Dial-in: (866)700-7173
Conference Call Passcode: 73322380
Webcast Link: http://www.media-server.com/m/p/ijf69diw

About Verastem, Inc.

Verastem, Inc. (NASDAQ: VSTM) is a biopharmaceutical company focused on discovering and developing drugs to treat breast and other cancers by targeting cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. For more information please visit www.verastem.com.

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, VS-4718, VS-5584 and VS-507, and the Company’s FAK, PI3K/mTOR, Wnt and diagnostics programs generally, the timeline for clinical development and regulatory approval of the Company’s compounds, the structure of the Company’s planned clinical trials, the Company’s rights to develop or commercialize its compounds, the Company’s obligations to make milestone payments and royalties and the ability of the Company to finance contemplated development activities. The words “anticipate,” “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, VS-4718, and 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.

Source: Verastem, Inc.

Verastem, Inc.
Investors:
Brian Sullivan, 617-252-9314
bsullivan@verastem.com
or
Media:
Kari Watson, 781-235-3060
kwatson@macbiocom.com