Verastem, Inc. (NASDAQ:VSTM), focused on discovering and developing
drugs to treat cancer by the targeted killing of cancer stem cells,
today announced that VS-5584 has received orphan drug designation from
the U.S. Food and Drug Administration for use in the treatment of
mesothelioma. The designation was created to encourage the development
of drugs that may provide significant benefit to patients suffering from
rare diseases.
“This is an important regulatory milestone for Verastem and, together
with our European orphan medicinal product designation, will facilitate
our global development of VS-5584 to help improve the available
treatment options for patients suffering from this highly aggressive
cancer,” said Robert Forrester, Verastem President and Chief Executive
Officer. "We look forward to taking full advantage of the opportunities
that orphan designation allows in order to bring this potential new
treatment option to patients as rapidly as possible."
Verastem recently initiated a Phase 1 clinical study evaluating the
combination of VS-5584 and VS-6063 in patients with relapsed or
progressive malignant pleural mesothelioma. The combination clinical
trial is supported by preclinical work demonstrating the synergistic
activity of VS‐6063 and VS‐5584 in mesothelioma models in vitro and in
vivo. VS-5584 is also being evaluated in a Phase 1 study in advanced
solid tumors where the compound has been generally well tolerated and
preliminary activity has been observed, including in mesothelioma. Some
patients have been on study for over 6 months and the maximum tolerated
dose of VS-5584 has not been reached.
In the U.S., under the Orphan Drug Act, the FDA's Office of Orphan
Products Development (OOPD) grants orphan drug status to a drug intended
to treat a rare disease or condition, which is generally a disease that
affects fewer than 200,000 individuals in the country. Upon approval, if
received, the designation provides VS-5584 with certain benefits,
including seven years of U.S. market exclusivity in the specified
indications if the sponsor complies with certain FDA requirements.
Additional incentives for the sponsor include tax credits related to
qualified clinical trial expenses and a possible exemption from FDA
application fees.
About Mesothelioma
Mesothelioma is an aggressive form of cancer that occurs in the
mesothelium, the thin layer of tissue that covers the lungs and other
organs. Mesothelioma is associated with exposure to asbestos in most
cases. According to the World Health Organization, there are a total of
59,000 cases of mesothelioma worldwide each year. Most mesotheliomas
begin as one or more nodules that progressively grow to form a solid
coating of tumor surrounding the lung leading to eventual suffocation
and death. A high percentage of mesotheliomas contain cancer stem cells
which are generally resistant to the currently available treatment
options for mesothelioma. Current treatment in the front line setting
consists of 4-6 cycles of Alimta (pemetrexed) in combination with
platinum-based therapy. Alimta is the only approved treatment for
mesothelioma and there are no approved therapies for relapsed
mesothelioma. Relapsed mesothelioma is highly aggressive with a median
time to disease progression of only 6 weeks.
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. In preclinical studies,
VS-5584 has been shown to reduce the percentage of cancer stem cells and
induce tumor regression in chemotherapy-resistant models. Verastem is
currently conducting a Phase 1 dose escalation trial of VS-5584 in
patients with advanced solid tumors as a single agent and a combination
trial of VS-5584 and VS-6063 in patients with relapsed mesothelioma.
VS-5584 has been granted orphan drug designation in the EU for use in
mesothelioma.
About VS-6063
VS-6063 (defactinib) 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 FAK
activity 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 “Window of Opportunity” study in patients with mesothelioma prior to
surgery, a Phase 1/1b study in combination with paclitaxel in patients
with ovarian cancer, and a trial in patients with Kras-mutated non-small
cell lung cancer and a trial evaluating the combination of VS-6063 and
VS-5584 in patients with relapsed mesothelioma. VS-6063 has been granted
orphan drug designation in the U.S. and EU 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
including FAK and PI3K/mTOR. 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 product candidates, including
VS-5584 and VS-6063, the timeline for clinical development and
regulatory approval of the Company’s product candidates, including the
impact of and any potential benefits from FDA’s orphan drug designation,
and the structure of the Company’s pending clinical trials. 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 product
candidates and preliminary or interim data from clinical trials may not
be predictive of the results or success of ongoing or later clinical
trials, that data may not be available when we expect it to be, that
enrollment of clinical trials may take longer than expected, that the
Company will be unable to successfully complete the clinical development
of its product candidates, including VS-5584 and VS-6063, that the
development of the Company’s product candidates will take longer or cost
more than planned, and that the Company’s product candidates 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, 2013 and in any subsequent SEC filings. The
forward-looking statements contained in this press release 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.
Copyright Business Wire 2015