Study Represents First-in-Asia Use of Lead FAK Inhibitor to Target
Cancer Stem Cells
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 24, 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 defactinib
(VS-6063), a potent inhibitor of focal adhesion kinase (FAK), in
Japanese patients with advanced solid tumors. FAK is a pathway critical
for the growth and survival of cancer stem cells and the study marks the
launch of Verastem’s development efforts in Japan.
The Phase 1 open-label, dose-escalation study, which will be conducted
at Kinki University in Osaka, is designed to assess the safety,
pharmacokinetics and pharmacodynamics of single agent defactinib. The
study will enroll 12-18 patients. Pending a successful outcome to the
Phase 1 study, Verastem may add additional clinical sites in Japan to
the recently initiated COMMAND trial, a global registration-directed
study of defactinib in patients with malignant pleural mesothelioma, and
may pursue additional development efforts in Japan.
“Initiation of this trial is another important clinical milestone for
Verastem and for defactinib, a drug candidate which is now being studied
at sites in 12 countries over five continents,” said Dr. Joanna Horobin,
Verastem Chief Medical Officer. “In the US Phase 1 study, defactinib
demonstrated good tolerability, as well as early signs of activity. With
this study we hope to take the first steps toward a path to registration
in Japan in parallel to our efforts in Europe and the US.”
“The simultaneous Japanese development of defactinib with the ongoing
efforts in the United States and Europe is highly exciting,” said
Professor Kazuhiko Nakagawa, M.D., Ph.D., Director, Department of
Medical Oncology, Kinki University Faculty of Medicine and Coordinating
Investigator for defactinib development in Japan. “At Kinki University,
we are dedicated to accelerating the development of novel compounds such
as defactinib that have the potential to make a significant difference
in the treatment of cancer for our patients in Japan.”
Historically, the initiation of development in Japan for most drugs has
been delayed in comparison to efforts in the US and EU. Verastem is
pursuing a rapid expansion of the geographical development of defactinib
in an attempt to rapidly make defactinib available for patients
worldwide.
“We are pleased to take part in this novel Phase 1 trial of defactinib
that targets cancer stem cells,” said Toshio Shimizu, M.D., Principal
Investigator of the Phase 1 study. “Led by scientific breakthroughs from
Professor Robert Weinberg on the role of focal adhesion kinase in cancer
stem cells, Verastem is taking a new approach to the treatment of cancer
in an attempt to address the underlying cause of disease progression and
tumor recurrence.”
“With a dramatic rise in the incidence of mesothelioma in Japan, it is
imperative that new treatments with the potential to address this deadly
disease are developed in parallel with other major markets,” said
Christoph Westphal, M.D., Ph.D., Verastem Executive Chairman.
“Initiation of this study in Japan serves to underscore our passion for
carrying out Verastem’s mission, which is to deliver new medicines that
will make a meaningful difference in the lives of cancer patients around
the world.”
About Defactinib
Defactinib (VS-6063) is an oral 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.
Defactinib is currently being studied in the registration-directed
COMMAND trial in mesothelioma, a Phase 1/1b study in ovarian cancer and
a Phase 1 study in Japan. A Phase 2 trial in KRAS-mutated Non-Small Cell
Lung Cancer is expected to begin in the third quarter of 2013.
Defactinib has been granted orphan drug designation in the U.S. and E.U.
for use in mesothelioma.
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:
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,
or defactinib, and the Company’s FAK and diagnostic programs generally,
the timeline for clinical development and regulatory approval of the
Company’s compounds, the expected timing for the reporting of data from
ongoing trials, the structure of the Company’s planned or pending
clinical trials and estimates of the Company’s ability to fund
operations. 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 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 defactinib, 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.
Source: Verastem, Inc.
Verastem, Inc.
Brian Sullivan, 617-252-9314
bsullivan@verastem.com