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 data in a poster presentation and
discussion at the American Society of Clinical Oncology Annual Meeting
being held May 30 – June 3, 2014, at McCormick Place in Chicago, IL.
Verastem presented interim data from the ongoing Phase 1/1b trial of its
lead product candidate, VS-6063 (defactinib), an oral small molecule
that targets cancer stem cells through the inhibition of focal adhesion
kinase (FAK), in combination with paclitaxel in patients with ovarian
cancer. The study, which has completed enrollment, is evaluating 22
patients at three sites in the US.
“These data continue to show that the combination of cancer stem
cell-targeting agent VS-6063 and paclitaxel is well tolerated, with no
unexpected toxicity or worsening of the well understood side effects of
paclitaxel, and support the further study of this combination in the
clinic,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “In
addition to the demonstration of safety, we continue to see interesting
signs of early clinical activity where 14 of 22 (64%) patients enrolled
in this study achieved a best overall response of at least stable
disease including two partial responses and two complete responses to
date.”
“We believe that to change the way cancer is treated it will be
necessary to kill both the cancer stem cells, and the bulk tumor cells,
in order to improve patient outcomes for many types of cancer,” said
Robert Forrester, Verastem President and Chief Executive Officer. “The
ability to combine VS-6063 with paclitaxel provides an opportunity to
explore additional indications where the tumors are driven by cancer
stem cells and paclitaxel is the standard of care. We are encouraged by
these initial data and will update on the progress of the 9 patients
that remain on study later this year.”
A summary of the data presented by Verastem at the conference is below:
Poster Presentation and Discussion
Date & Time: Monday, June 2, 2014, from 8:00 a.m. to 12:45
p.m. CT
Poster Title: Phase 1/1b Study of the FAK Inhibitor Defactinib
(VS-6063) in Combination with Weekly Paclitaxel for Advanced Ovarian
Cancer
Abstract Number: 5521
Session ID: Poster Highlights Session: Gynecologic Cancer
Location: E354b & E354a
Summary: Defactinib (VS-6063) is an oral small molecule that
targets cancer stem cells through the inhibition of focal adhesion
kinase (FAK). FAK is a protein kinase that is critical for cancer stem
cell survival. Tumor initiation by cancer stem cells requires
attachment, proliferation and survival which are all mediated by FAK.
Standard of care (SOC) agents such as paclitaxel have been shown in
preclinical models to enrich for chemoresistant and tumor initiating
cancer stem cells, while preclinical studies with VS-6063 have shown
that it can selectively target cancer stem cells and attenuates the
enrichment of cancer stem cells typically seen with standard of care
agents. This multicenter study evaluated the safety and efficacy of the
combination of VS-6063 and weekly paclitaxel in advanced ovarian cancer.
The study results demonstrated that combination therapy was generally
well tolerated with no dose limiting toxicities or exacerbation of
paclitaxel related toxicities observed at either dose level (200mg or
400mg BID). The recommended Phase 2 dose was determined to be VS-6063
400mg BID with weekly paclitaxel (80 mg/m2) administered on
Day 1, 8 and 15 of a 28 day cycle. Interesting signs of clinical
activity have been observed in this ongoing trial with nine patients
remaining on study. To date, four patients have exhibited objective
responses: Two complete responses and two partial responses. Treatment
with VS-6063 resulted in substantial decreases in FAK expression in four
of the five paired biopsies. The successful administration of VS-6063
with weekly paclitaxel may enable the utilization of this combination in
other clinical settings where paclitaxel is used.
Other Poster Presentations
In addition, investigators presented posters describing the trial
designs from two of Verastem’s other ongoing clinical studies: one for
the registration-directed COMMAND study of VS-6063 for patients with
malignant pleural mesothelioma, and the other for the Phase 2 study of
VS-6063 for patients with non-small cell lung cancer.
Analyst Event and Webcast
The company hosted an analyst and investor event during ASCO where a
scientific update on the COMMAND study and the rationale for targeting
cancer stem cells in mesothelioma was discussed. Professor Dean Fennell,
Ph.D., FRCP, Chair of Thoracic Oncology at the University of Leicester
and Incoming President of the International Mesothelioma Interest Group
was a guest speaker.
A replay of the event webcast can be accessed here
or by visiting the Verastem website.
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 the FAK
pathway 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 Phase 1/1b study in combination with paclitaxel for patients with
ovarian cancer and a Phase 2 trial in patients with Kras-mutated
non-small cell lung cancer. VS-6063 has been granted orphan drug
designation in the U.S. and E.U. for use in mesothelioma.
About COMMAND
COMMAND is a registration-directed, double-blind, placebo-controlled
trial of VS-6063 with Progression Free Survival (PFS) and Overall
Survival (OS) as the primary endpoints. VS-6063 targets cancer stem
cells. Cancer stem cells are an underlying cause of tumor progression
and recurrence. The design of COMMAND allows the opportunity to enrich
for patients with tumors low in the biomarker, merlin. Preclinical and
early clinical research has demonstrated that low merlin levels may be
predictive of increased effectiveness of FAK inhibitors such as VS-6063.
The COMMAND study stratifies patients to evaluate the effect of VS-6063
in both the overall patient population and the subgroup of patients
whose tumors are low in merlin.
COMMAND is expected to enroll approximately 350-400 patients at clinical
sites in 12 countries, including the US, UK, Japan, Australia, Canada,
South Africa, New Zealand and countries in mainland Europe. Eligible
patients who had a partial response or stable disease following standard
first-line therapy with platinum/pemetrexed will be stratified to merlin
low or high and then randomized to receive either placebo or 400 mg of
defactinib. For more information visit www.COMMANDmeso.com
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 inhibition program, 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, and potential indications for clinical development. 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 ongoing or later clinical trials, including the
ongoing Phase 1/1b ovarian cancer study, 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,
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 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.
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