CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 24, 2013--
Verastem, Inc., (NASDAQ: VSTM) focused on discovering and developing
drugs to treat cancer by the targeted killing of cancer stem cells,
announced that lead cancer stem cell inhibitor, VS-6063 (defactinib),
has received orphan drug designation from the U.S. Food and Drug
Administration (FDA) for use in the treatment of mesothelioma, a rare
form of lung cancer. The designation is designed to encourage the
development of drugs which may provide significant benefit to patients
suffering from rare diseases.
“Mesothelioma is among the most aggressive and lethal cancers but has
limited treatment options,” said Robert Forrester, Verastem President
and Chief Executive Officer. “We are pleased that the FDA recognizes the
significant unmet medical need in mesothelioma. We previously received
orphan medicinal product status for defactinib in Europe and these two
designations are an important component of our development strategy.”
Verastem recently outlined details of the registration-directed clinical
study of defactinib in patients with malignant pleural mesothelioma.
This study is designed as a double-blind, placebo-controlled trial with
an expected enrollment of approximately 350-400 patients at clinical
sites in 11 countries.
“We are in discussions with the regulatory agencies and clinical
investigators worldwide,” said Dr. Joanna Horobin, Verastem Chief
Medical Officer. “We recently held our investigator meetings for the
physicians conducting the trial in the US and Australia and we are on
track to begin enrolling patients in the third quarter. We plan to open
sites worldwide on a rolling basis as we clear regulatory and clinical
review in each country.”
Orphan drug designation is granted by the FDA Office of Orphan Drug
Products to novel drugs or biologics that treat a rare disease or
condition affecting fewer than 200,000 patients in the U.S. The
designation provides eligibility for a seven-year period of market
exclusivity in the U.S. after product approval, FDA assistance in
clinical trial design, and an exemption from FDA user fees.
“Cancer stem cells play a central role in treatment resistance in many
types of cancers,” said Christoph Westphal, M.D., Ph.D., Verastem
Executive Chairman. “We believe new treatment options targeting cancer
stem cells will be critical to achieve a durable clinical benefit for
patients. This designation will provide us with many benefits as we
pursue the development of defactinib for the treatment of mesothelioma.”
In addition to mesothelioma, Verastem recently announced the completion
of the Phase 1 stage and initial data from an ongoing Phase 1/1b study
of defactinib in combination with weekly paclitaxel for patients with
ovarian cancer. Verastem also expects to initiate a Phase 1 study in
Japan, and a Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer,
for defactinib during the third quarter of 2013.
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,
and the Company’s FAK and diagnostic programs generally, the timeline
for clinical development and regulatory approval of the Company’s
compounds, the structure of the Company’s planned clinical trials and
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 the Company will be unable to successfully
complete the clinical development of its compounds, including VS-6063,
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