Press Release

March 25, 2014
Verastem to Present Data on Product Candidates Targeting Cancer Stem Cells at the American Association of Cancer Research Meeting

-New research to be presented in an oral session and three poster presentations-

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 25, 2014-- Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that the Company will present data at the upcoming American Association of Cancer Research (AACR) meeting to be held April 5-9, 2014 at the San Diego Convention Center in San Diego, CA.

“The four presentations at the upcoming AACR meeting demonstrate our commitment to furthering the body of knowledge surrounding the role of cancer stem cells as the driving factor in tumor progression,” said Jonathan Pachter, Ph.D., Verastem Head of Research. “The data in these presentations support our growing pipeline of clinical product candidates and provide insight into our clinical development in a number of different cancers. We will present data generated in experiments with our cancer stem cell targeting agents that act through inhibition of the FAK and PI3K/mTOR pathways, including our lead candidate VS-6063, currently in the registration-directed COMMAND study for the treatment of mesothelioma.”

The details of the presentations by Verastem scientists are below:

Oral Presentation
Title: VS-6063 (defactinib) targets cancer stem cells directly and through inhibition of tumor-associated macrophages and cytokine production
Date and time: Tuesday, April 8, 2014, 4:20 pm4:35 pm PT
Location: Room 6B
Abstract Number: 4797
Session: Minisymposium “Elucidation and Niche Targeting of Cancer Stem Cell Epigenetic and Metabolic Alterations”

Poster Presentations
Title: Focal adhesion kinase (FAK) inhibitor VS-6063 (defactinib) preferentially targets cancer stem cells in triple negative breast cancer
Date and time: Tuesday, April 8, 2014, 1:00 pm5:00 pm PT
Location: Hall A-E
Abstract Number: 213
Poster Section 2: Cancer Stem Cell Phenotype, Function, and Targeting

Title: VS-5584 a dual mTORC1/2 and PI3K inhibitor has anti-tumor activity in multiple in vivo xenograft tumor models and enhanced efficacy in combination with cisplatin or docetaxel
Date and time: Sunday, April 6, 2014, 1:00 pm5:00 pm PT
Location: Hall A-E
Abstract Number: 3906
Poster Section 8: Stem Cell Expansion and Cancer Stem Cell Targeting

Title: Combined inhibition of PI3K isoforms and mTOR kinase is critical for cancer stem cell inhibition by VS-5584
Date and time: Tuesday, April 8, 2014, 1:00 pm5:00 pm PT
Location: Hall A-E
Abstract Number: 3908
Poster Section 2: Cancer Stem Cell Phenotype, Function, and Targeting

About VS-6063

VS-6063 is an orally available compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research by Robert Weinberg, Ph.D., scientific cofounder and chair of Verastem’s Scientific Advisory Board, and Verastem has demonstrated that the FAK pathway is critical for the growth and survival of cancer stem cells. VS-6063 is currently being studied in the registration-directed COMMAND trial in mesothelioma (www.COMMANDmeso.com), a Phase 1/1b study in combination with paclitaxel for patients with ovarian cancer, a Phase 1 study in Japan in patients with advanced solid tumors and a Phase 2 trial in patients with Kras-mutated non-small cell lung cancer. VS-6063 has been granted orphan drug designation in the U.S. and E.U. for use in mesothelioma.

About VS-5584

VS-5584 is an orally available compound that has demonstrated potent and highly selective activity against class 1 PI3K enzymes and dual inhibitory actions against mTORC1 and mTORC2 pathways. In preclinical studies, VS-5584 has been shown to reduce the percentage of cancer stem cells and induce tumor regression in taxane-resistant models. Verastem is currently conducting a Phase 1 dose escalation trial of VS-5584 in patients with advanced solid tumors and 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 www.verastem.com.

Forward-looking statements:

Any statements in this press release about future expectations, plans and prospects for the Company constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.

Source: Verastem, Inc.

Verastem, Inc.
Brian Sullivan, 617-252-9314
bsullivan@verastem.com