-Results from a Window of Opportunity study in
surgically-eligible patients demonstrate a reduction in pFAK,
cancer stem cell markers and tumor size following 12 days of
treatment with VS-6063-
Verastem, Inc. (NASDAQ:VSTM), focused on discovering and
developing drugs to treat cancer by the targeted killing of cancer
stem cells, announced the presentation of clinical and preclinical
data in oral presentation and discussion sessions at the 12th
International Mesothelioma Interest Group (iMig) Conference being
held October 22-24, 2014, at the Cape Town International Conference
Centre in Cape Town, South Africa.
Professor Raphael Bueno, M.D., Chief of Thoracic Surgery,
Brigham and Women’s Hospital (BWH), Boston and Principal
Investigator of Verastem’s ongoing Window of Opportunity study
presented preliminary clinical results in an oral presentation. The
study is evaluating the biomarker response to VS-6063 (defactinib),
an oral small molecule that targets cancer stem cells through the
inhibition of focal adhesion kinase (FAK) in patients with
resectable pleural mesothelioma. Biopsies of the tumor are taken
before and after oral administration of VS-6063 (400mg BID) for 12
days. The study also measures tumor size using CT/PET scans before
and after the 12-day administration of VS-6063. All study patients
were enrolled at the BWH clinical site.
An analysis of the preliminary data (n=10) showed that VS-6063
reduced FAK activity (pFAK-Y397) by an average of 70% in the
patients evaluated to date, and reduced the presence of cancer stem
cell markers in the post-treatment biopsies in 5 out of the 7
patients with evaluable paired biopsies. Measurement of tumor size
by CT/PET using RECIST modified for mesothelioma confirmed that
there was no progression of disease while on the 12 day treatment
with single agent VS-6063 in any of the 10 patients. Moreover, in 2
patients, tumor shrinkage consistent with a partial response (-30%,
-49%) was noted. VS-6063 was well tolerated with no apparent
negative impact on surgical outcome.
“These initial biopsy data show signs of activity against
biomarkers of cancer stem cells as well as intriguing reductions in
tumor size, after short-term exposure to single agent VS-6063,”
said Dr. Bueno. “To follow up on these encouraging results, the
protocol is being amended to explore more extended dosing in 10-15
additional patients with mesothelioma prior to surgery. The window
of opportunity before elective surgery provides a novel platform
for the clinical evaluation of promising new agents for the
treatment of this devastating disease.”
“This study was designed to evaluate potential biomarkers of
response to VS-6063 treatment in patients with pleural
mesothelioma,” said Dr. Joanna Horobin, Verastem Chief Medical
Officer. “We are encouraged by the biomarker response, and
intrigued by the tumor shrinkage observed after 12 days of VS-6063
administration. As we expand our clinical program with VS-6063 in
mesothelioma into other patient settings, we are encouraged by
these interesting signs of clinical activity.”
In addition to the Window of Opportunity study, Verastem is
conducting the registration-directed COMMAND study as a maintenance
treatment immediately following successful front-line therapy with
Alimta and cisplatin in patients with malignant pleural
mesothelioma. Verastem anticipates starting a study of VS-6063 and
VS-5584 in combination for patients with relapsed or progressive
malignant pleural mesothelioma in early 2015.
Verastem also presented preclinical data at the conference
demonstrating the ability of VS-6063 and the combination of VS-6063
and VS-5584 to target and kill cancer stem cells in models of
mesothelioma. In addition, Professor Robert Weinberg, Ph.D.,
Whitehead Institute/Massachusetts Institute of Technology, Verastem
co-founder and Chair of the Scientific Advisory Board, gave a
keynote address to open the symposium entitled: “Cancer Stem Cells
as Target Pathways.” These presentations support the ongoing and
planned clinical trials that Verastem is conducting in patients
with mesothelioma.
A summary of the data presented by Verastem at the conference is
below:
Oral Presentations and Discussions
iMig Special Keynote Lecture: “Cancer Stem Cells as
Target Pathways”Presenter: Robert Weinberg, Ph.D., Whitehead
Institute/Massachusetts Institute of Technology, Verastem
scientific cofounder and chair of the Scientific Advisory
BoardDate and time: Wednesday, October 22, 2014, 8:50 AM
(local time)Link to presentation: http://bit.ly/12otlcV
Presentation Title: “FAK Inhibitor VS-6063 (defactinib)
Targets Mesothelioma Cancer Stem Cells which are Enriched by
Standard of Care Chemotherapy”Presenter: Paul
Baas, M.D., Ph.D., Department of Thoracic Oncology, The
Netherlands Cancer InstituteDate and time: Thursday, October
23, 2014, 3:30 PM (local time)Link to presentation:
http://bit.ly/12otlcV
Presentation Title: “Determination of Biomarker Response
in a Phase II Window of Opportunity Study of Defactinib (VS-6063),
a Focal Adhesion Kinase (FAK) Inhibitor, in Subjects with
Resectable MPM”Presenter: Raphael Bueno, M.D., Chief,
Division of Thoracic Surgery, Brigham & Women’s HospitalDate
and time: Thursday, October 23, 2014, 3:45 PM (local
time)Link to presentation: http://bit.ly/12otlcV
Presentation Title: “The Cancer Stem Cell Inhibitors
VS-6063 (defactinib) and VS-5584 Exhibit Synergistic Anticancer
Activity in Preclinical Models of Mesothelioma”Presenter:
Mitchell Keegan, Ph.D., Vice President, Development,
VerastemDate and time: Friday, October 24, 2014, 10:30 AM
(local time)Link to presentation: http://bit.ly/12otlcV
Poster Presentation
Presentation Title: “COMMAND: A Phase 2
Randomized, Double-Blind, Placebo-Controlled, Multicenter Study of
Defactinib as Maintenance Therapy in Subjects with Malignant
Pleural Mesothelioma which has not Progressed on at Least 4 cycles
of Pemetrexed/Platinum Therapy”Presenter: Mitchell Keegan,
Ph.D., Vice President, Development, VerastemDate and time:
Thursday, October 23, 2014, 10:30AM (local time)Link to
poster: http://bit.ly/12otlcV
Several of the above-mentioned studies were selected to be
featured in the “Best of iMig” series, which highlighted the most
relevant studies from the conference. Video of the presentations
can be found at http://bit.ly/12d3OU1
About Malignant Pleural Mesothelioma
Malignant pleural mesothelioma is an aggressive form of cancer
that occurs in the mesothelium, the thin layer of tissue that
covers the lungs. 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.
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.
VS-6063 has been granted orphan drug designation in the U.S. and EU
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. 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 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:
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, andVS-5584 and the Company’s FAK
inhibition program and PI3K/mTOR program 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, and the structure of the Company’s planned or
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 compounds and preliminary or interim data from clinical
trials, including the Window of Opportunity study, 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 compounds, including VS-6063 and
VS-5584, 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, 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.
Verastem, Inc.Brian Sullivan,
781-292-4214bsullivan@verastem.com
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