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Verastem Initiates Phase 2 Trial of Defactinib in Lung Cancer
Study Focuses on Patients with KRAS-mutated Non-Small Cell Lung Cancer
“We currently do not have any effective targeted treatment options for
patients with KRAS-mutated non-small cell lung cancer (NSCLC) which
represents about 30% of the NSCLC population,” said Dr.
Dr. Scaglioni’s group at UTSW published a paper in 2013 in Cancer Discovery titled “RHOA-FAK is a Required Signaling Axis for the Maintenance of KRAS-Driven Lung Adenocarcinoma” that describes the critical role of FAK signaling in NSCLC that has both a KRAS-mutation and accompanying secondary mutation in the p16/ARF/INK4a locus (p16) or p53. As reported in the paper, genetic silencing or deletion of FAK replicated the effects of treatment with Verastem’s first generation small molecule inhibitor of FAK, VS-6062, in both in vitro and xenograft models of the disease.
"The reported rates vary, but we believe approximately 10-15% of NSCLC
tumors have both the KRAS mutation and an accompanying secondary
mutation in p16 or p53 leading to silencing of those tumor suppressor
genes, which appears to be a prerequisite for the activity of FAK
inhibitors in KRAS-mutated NSCLC models,” said Dr.
The Phase 2 study is designed to assess the effect of defactinib on Progression Free Survival (PFS), Overall Response Rate (ORR) and Overall Survival (OS). There will be 4 study arms, including patients with a KRAS-mutation, or a KRAS-mutation with accompanying secondary mutations in p16, p53 or both p16 and p53. The trial is being conducted at 8-10 sites in the US and is expected to enroll up to 44 patients (11 per arm) in the first stage of the Simon two-stage trial design. A Simon two-stage trial design incorporates a single, pre-planned interim analysis to determine which enrollment arms are exhibiting a favorable response to treatment and allows for enrollment of an additional 12 patients with the same tumor mutation profile per arm.
“With the initiation of this study we now have a total of five clinical
studies underway,” said
The paper “RHOA-FAK is a Required Signaling Axis for the Maintenance of KRAS-Driven Lung Adenocarcinoma” by Konstantinidou et. al., Cancer Discovery 2013, can be accessed at http://bit.ly/1bd1Zta
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
About Verastem, Inc.
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
Source:
Verastem, Inc.
Brian Sullivan, 617-252-9314
bsullivan@verastem.com