Verastem Reports Data on Focal Adhesion Kinase Program at the 2012 EORTC Symposium on Molecular Targets and Cancer Therapeutics

  Verastem Reports Data on Focal Adhesion Kinase Program at the 2012 EORTC
  Symposium on Molecular Targets and Cancer Therapeutics

Clinical Data Reported by GSK Demonstrates Improved Progression Free Survival
in Response to FAK Inhibition in Mesothelioma Patients Lacking the Tumor
Suppressor Merlin

Business Wire

CAMBRIDGE, Mass. -- November 09, 2012

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, reported new data from its focal adhesion kinase
(FAK) inhibition program and commented on encouraging clinical data on FAK
inhibition presented at the 2012 EORTC Symposium on Molecular Targets and
Cancer Therapeutics being held in Dublin, Ireland.

Data presented by Verastem in breast cancer models lacking the tumor
suppressor Merlin demonstrate that FAK inhibition effectively reduces cancer
stem cells in vitro and in vivo. Oral administration of a FAK inhibitor as a
single agent was shown to induce tumor regression in a Merlin-negative breast
cancer mouse model. These data extend previous research from Verastem, which
demonstrated similar inhibition of cancer stem cells and strong single agent
efficacy in Merlin-negative mesothelioma models.

Several groups presented data at the conference on the use of FAK inhibitors
for treatment of cancer. In one clinical study by GlaxoSmithKline, researchers
demonstrated that Merlin loss may identify a subset of patients with improved
progression free survival in response to FAK inhibition. “These results
provide important clinical validation of our internal research demonstrating
enhanced sensitivity of Merlin-negative mesothelioma to FAK inhibitors,” said
Jonathan Pachter, Ph.D., Verastem Vice President and Head of Research.

Verastem anticipates initiating a potentially pivotal study in mesothelioma
midyear 2013.

“The sum of the data on development of FAK inhibitors presented at this
conference is very promising,” said Professor Dean Fennell, Chair of Thoracic
Medical Oncology, University of Leicester. “In particular, GSK reported in a
Phase 1 trial novel activity of a FAK inhibitor in mesothelioma, where
treatment in the second and third-line setting resulted in a median
progression free survival of 17.7 weeks.”

The largest clinical trial to date in malignant pleural mesothelioma was a
2011 Phase 3 study of Zolinza^® as a second- and third-line treatment
conducted by Merck in 660 patients. In this study, a median progression free
survival of only 6 weeks was observed.

“Interestingly, loss of the tumor suppressor Merlin correlated with increased
clinical sensitivity to FAK inhibition,” Prof. Fennell continued. “A subset of
patients with Merlin negative mesothelioma had more than double the median
progression free survival of 24.1 weeks versus 11.4 weeks for the Merlin
positive group. These early results suggest that a targeted therapy,
particularly when used in combination with a specific biomarker, has the
potential to significantly improve treatment of this aggressive and deadly

“We are encouraged by the initial clinical data presented today by GSK,” said
Dr. Joanna Horobin, Verastem Chief Medical Officer. “Merlin loss occurs in
approximately 50% of mesothelioma patients and we believe that VS-6063 has
potential as a targeted therapy in this disease. We are moving rapidly to
initiate a potentially pivotal, double blind controlled study next year.”

FAK is a cytoplasmic tyrosine kinase that mediates signal transduction by
integrins and growth factor receptors. FAK has been implicated in different
steps of tumor development including tumor initiation, growth, angiogenesis
and metastasis. Verastem has pioneered research on FAK as a critical regulator
of cancer stem cells using technology developed by cofounder and chair of the
Scientific Advisory Board, Robert Weinberg, Ph.D.

The poster presentation by Verastem is titled: “FAK Inhibitor VS-4718
Attenuates Breast Cancer Stem Cell Function and Inhibits Tumor Growth in

The poster presentation by GlaxoSmithKline is titled: “Loss of the Tumor
Suppressor Merlin as a Potential Predictive Biomarker of Clinical Activity for
the Oral, Focal Adhesion Kinase (FAK) Inhibitor GSK2256098 in Pts with
Recurrent Mesothelioma.”

About VS-6063

VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK).
FAK is critical for the survival of cancer stem cells, which are an underlying
cause of tumor recurrence and metastasis. VS-6063 was well-tolerated and
demonstrated signs of clinical activity in a Phase 1 study in advanced solid
tumors. Verastem has demonstrated that loss of the tumor suppressor Merlin
confers increased susceptibility to FAK inhibition. Verastem is planning to
initiate multiple clinical trials in 2013 with VS-6063, including a potential
registration study in mesothelioma, which will evaluate patient response based
on Merlin status.

AboutVerastem, 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

Zolinza^® is a registered trademark of Merck, Inc.

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 VS-4718, and the
Company’s FAK 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,”
“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 and VS-4718, 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.


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