Company Expects to Report Top-line Data from
Phase 3 DUO™ Study in Late Summer 2017
Verastem, Inc. (NASDAQ: VSTM), focused on discovering and
developing drugs to improve the survival and quality of life of
cancer patients, today reported financial results for the second
quarter ended June 30, 2017 and provided an overview of certain
corporate developments.
“The second quarter of 2017 was marked by several duvelisib data
presentations at top hematology-focused medical meetings,”
said Robert Forrester, President and Chief Executive Officer
of Verastem. “Long-term follow-up data from the Phase 2
DYNAMO™ study, which was presented at the 14th International
Conference on Malignant Lymphoma (ICML), demonstrated a durable 47%
response rate and a well characterized and manageable safety
profile in patients with indolent non-Hodgkin Lymphoma (iNHL) whose
disease is refractory to both rituximab and chemotherapy or
radioimmunotherapy. These overall DYNAMO results were followed by
promising subgroup data for DYNAMO patients with follicular
lymphoma (FL) or small lymphocytic lymphoma (SLL), presented at the
22nd Congress of the European Hematology Association (EHA), which
described high response rates of 43% and 68% in FL and SLL,
respectively. We remain highly encouraged by the data generated to
date from the duvelisib program and we look forward to reporting
top-line data from the Phase 3 DUO™ study in relapsed/refractory
chronic lymphocytic leukemia and small lymphocytic lymphoma
(CLL/SLL) currently expected in the latter part of summer
2017.”
Second Quarter 2017 and Recent Highlights:
Duvelisib
- Ongoing Phase 3 DUO Study in
Relapsed or Refractory CLL/SLL – The efficacy and safety of
duvelisib is currently being evaluated in the randomized Phase 3
DUO study in patients with relapsed or refractory CLL/SLL. In the
DUO study, approximately 300 patients were randomized 1:1 to
receive duvelisib or ofatumumab. The trial was fully enrolled in
November 2015. The primary endpoint of this study is progression
free survival (PFS). Key secondary endpoints include overall
response rate (ORR), overall survival (OS), duration of response
(DOR) and safety. Verastem expects to report top-line data from the
DUO study in the latter part of summer 2017.
- Presented Long-Term Follow Up Data
in Patients with Double-Refractory FL and SLL at EHA
2017 – Long-term follow up data from the subsets of patients
with FL or SLL who were enrolled in the ongoing Phase 2 DYNAMO
study were the subject of presentations at EHA 2017 in Madrid,
Spain. In an oral presentation, titled “DYNAMO: A Phase 2 Study
Demonstrating the Clinical Activity of Duvelisib in Patients with
Double-Refractory Follicular Lymphoma,” Pier Luigi Zinzani, M.D.,
Ph.D., of the University of Bologna Institute of Hematology,
reported that duvelisib monotherapy demonstrated an ORR of 43%, as
determined by an independent review committee, with 83% of patients
experiencing a reduction in the size of target lymph nodes. The
median DOR was 7.9 months, the median PFS was 8.3 months, and the
median overall survival (OS) was 27.8 months. In an e-poster
presentation, titled “DYNAMO: The Clinical Activity of Duvelisib in
Patients with Double-Refractory Small Lymphocytic Lymphoma in a
Phase 2 Study,” Dr. Zinzani reported that duvelisib as a
monotherapy demonstrated an ORR of 68%, as determined by an
independent review committee, with 100% of patients experiencing a
reduction in the size of target lymph nodes. With a median time on
duvelisib of 12 months, median DOR was 10.1 months, median PFS was
11.7 months, and median OS was 28.9 months. The safety profile of
duvelisib monotherapy remained consistent with what has been
previously reported in iNHL and other hematologic malignancies.
Copies of Dr. Zinzani’s oral and e-poster presentations are
available here and here.
- Presented Long-Term Follow Up Data
from the Phase 2 DYNAMO Study at ICML 2017 – Long-term follow
up data from the ongoing Phase 2 DYNAMO study was highlighted in an
oral presentation at ICML 2017 in Lugano, Switzerland. In the
presentation, titled “DYNAMO: A Phase 2 Study Demonstrating the
Clinical Activity of Duvelisib in Patients with Double-Refractory
Indolent Non-Hodgkin Lymphoma,” Dr. Zinzani, reported that
duvelisib as a monotherapy demonstrated an ORR of 47%, as
determined by an independent review committee, with 88% of patients
experiencing a reduction in the size of target lymph nodes.
Overall, the median DOR was 10 months, the median PFS was 9 months,
and the median overall survival was 27.8 months. With additional
follow-up (median 18 months), the safety profile of duvelisib
monotherapy remained consistent with what has been previously
reported in iNHL and other hematologic malignancies. The DYNAMO
study met its primary ORR endpoint (p=0.0001) at the primary
analysis. A PDF of Dr. Zinzani’s oral presentation is available
here.
Defactinib (VS-6063)
- Published Scientific Data
Highlighting Potential Role of Focal Adhesion Kinase (FAK)
Inhibition in Pancreatic and Breast Cancer – In July 2017,
Verastem announced the publication of two papers in the
peer-reviewed journals, PLoS One and Oncotarget. The two published
articles reported scientific findings from studies evaluating FAK
inhibition in preclinical models of pancreatic and breast cancer
and continue to validate the underlying thesis for ongoing clinical
collaborations evaluating Verastem’s lead FAK inhibitor defactinib
in combination with chemotherapeutic and leading immunotherapeutic
agents in several difficult to treat types of cancer. The PLoS One
paper in pancreatic cancer is available here and the Oncotarget
paper in breast cancer is available here.
- Presented Preclinical Data at the
2017 American Association for Cancer Research Annual Meeting –
In an oral presentation titled, “Reprogramming the tumor
microenvironment to improve responses to therapy,” Verastem
scientific collaborator David G. DeNardo, Ph.D., Associate
Professor of Medicine, Division of Oncology, Department of
Immunology, Washington University School of Medicine in St. Louis,
described data demonstrating that FAK inhibition can enable
efficacy of PD-1 inhibition in preclinical models of pancreatic
cancer that, like the clinical disease, are otherwise refractory to
checkpoint inhibition. Verastem’s FAK inhibitor, defactinib, is
currently being evaluated in combination with Merck’s PD-1
inhibitor, pembrolizumab, and gemcitabine in patients with advanced
pancreatic ductal adenocarcinoma (PDAC). Initial analysis of immune
biomarkers from matched pairs of metastatic biopsies, taken either
pre- or post-treatment, from patients with PDAC showed an increase
in activated proliferating cytotoxic T cells together with a
reduction in tumor-associated macrophages (TAMs). A PDF copy of Dr.
DeNardo’s oral presentation is available here.
Corporate and Financial
- Julie B. Feder Appointed Chief
Financial Officer – In July 2017, Verastem announced the
appointment of Ms. Feder as its Chief Financial Officer. Ms. Feder
is an accomplished financial professional with invaluable
leadership experience in the healthcare industry. She joins
Verastem from the Clinton Health Access Initiative, Inc. (CHAI),
where she served as Chief Financial Officer. Prior to joining CHAI,
Ms. Feder held finance roles of increasing responsibility at
Genzyme Corporation including leading the internal audit process.
Ms. Feder began her career at Deloitte & Touche LLP and she
holds a Bachelor of Science in Accounting from Yeshiva University’s
Sy Syms School of Business.
- Eric K. Rowinsky, M.D. Appointed to
the Board of Directors – Verastem announced the appointment of
Eric K. Rowinsky, M.D., to its Board of Directors. Dr. Rowinsky
brings to Verastem nearly 30 years of experience in the development
of cancer treatments, such as cetuximab (Erbitux®) when he was
Chief Medical Officer of ImClone Systems, as well as Cyramza®,
Portrazza®, Taxol®, Taxotere®, Hycamtin®, Tarceva®, Camptosar®,
Tykerb®, and cixutumumab, among others. He is a member of the board
of directors of Biogen, Navidea, and Fortress Biotech, all public
life sciences companies, and has served on the board of directors
of BIND Therapeutics, a life-science company acquired by Pfizer.
Dr. Rowinsky replaced Paul A. Friedman, M.D. who transitioned from
his role as Director to become a member of Verastem’s Clinical and
Scientific Advisory Board.
Second Quarter 2017 Financial Results
Net loss for the three months ended June 30, 2017 (2017 Quarter)
was $13.4 million, or $0.36 per share, as compared to a
net loss of $8.6 million, or $0.23 per share, for the three
months ended June 30, 2016 (2016 Quarter). Net loss includes
non-cash stock-based compensation expense of $1.2
million and $1.7 million for the 2017 Quarter and 2016
Quarter, respectively. Verastem used $14.5
million for operating activities during the 2017 Quarter.
Research and development expense for the 2017 Quarter was
$9.0 million compared to $4.5 million for the 2016
Quarter. The $4.5 million increase from the 2016 Quarter to
the 2017 Quarter was primarily related to an increase of
$3.6 million in contract research organization expense for
outsourced biology, development and clinical services, which
includes our clinical trial costs, an increase of approximately
$894,000 in consulting fees, and an increase in personnel related
costs of approximately $244,000. These increases were offset by a
decrease in stock-based compensation and other expenses of
approximately $176,000.
General and administrative expense for the 2017 Quarter was
$4.4 million compared to $4.2 million for the 2016
Quarter. The increase of approximately $208,000 from the 2016
Quarter to the 2017 Quarter primarily resulted from an increase in
consulting and professional fees of approximately $870,000,
partially offset by decreases in stock-based compensation expense
of approximately $534,000 and personnel costs of approximately
$152,000.
As of June 30, 2017, Verastem had cash, cash
equivalents and investments of $57.9 million compared
to $80.9 million as of December 31, 2016.
The number of outstanding common shares as of June 30,
2017, was 36,992,418.
Financial Guidance
Based on our current operating plans, we expect to have
sufficient cash, cash equivalents and investments to fund our
research and development programs and operations into 2018.
About the Tumor Microenvironment
The tumor microenvironment encompasses various cellular
populations and extracellular matrices within the tumor or cancer
niche that support cancer cell survival. This includes
immunosuppressive cell populations such as regulatory T-cells,
myeloid-derived suppressor cells, M2 TAMS, as well as
tumor-associated fibroblasts and extracellular matrix proteins
which can hamper the entry and therapeutic benefit of cytotoxic
immune cells and anti-cancer drugs. In addition to targeting the
proliferative and survival signaling of cancer cells, Verastem’s
compounds duvelisib and defactinib target the tumor
microenvironment as a mechanism of action to potentially improve a
patient’s response to therapy.
About Duvelisib
Duvelisib is an investigational, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes
that are known to help support the growth and survival of malignant
B-cells and T-cells. PI3K signaling may lead to the proliferation
of malignant B-cells and is thought to play a role in the formation
and maintenance of the supportive tumor microenvironment.1,2,3
Duvelisib is currently being evaluated in late- and mid-stage
clinical trials, including DUO®, a randomized, Phase 3 monotherapy
study in patients with relapsed/refractory CLL/SLL,4 and DYNAMO®, a
single-arm, Phase 2 monotherapy study in patients with refractory
iNHL that achieved its primary endpoint of ORR upon topline
analysis of efficacy data.5 Duvelisib is also being evaluated for
the treatment of hematologic malignancies through
investigator-sponsored studies, including T-cell lymphoma.6
Information about duvelisib clinical trials can be found on
www.clinicaltrials.gov.
About Defactinib
Defactinib (VS-6063) is an investigational inhibitor of FAK, a
non-receptor tyrosine kinase that mediates oncogenic signaling in
response to cellular adhesion and growth factors.7 Based on the
multi-faceted roles of FAK, defactinib is used to treat cancer
through modulation of the tumor microenvironment, enhancement of
anti-tumor immunity, and reduction of cancer stem cells.8,9
Defactinib is currently being evaluated in three separate clinical
collaborations in combination with immunotherapeutic agents for the
treatment of several different cancer types including pancreatic,
ovarian, non-small cell lung cancer, and mesothelioma. These
studies are combination clinical trials with pembrolizumab and
avelumab from Merck & Co. and Pfizer/Merck KGaA,
respectively.10,11,12 Information about these and additional
clinical trials evaluating the safety and efficacy of defactinib
can be found on www.clinicaltrials.gov.
About Verastem, Inc.
Verastem, Inc. (NASDAQ:VSTM) is a biopharmaceutical company
focused on discovering and developing drugs to improve outcomes for
patients with cancer. Verastem is currently developing duvelisib, a
dual inhibitor of PI3K-delta and PI3K-gamma, which has successfully
met its primary endpoint in a Phase 2 study and is currently being
evaluated in a Phase 3 clinical trial in patients with
relapsed/refractory CLL/SLL. In addition, Verastem is
developing the FAK inhibitor, defactinib, which is currently being
evaluated in three separate clinical collaborations in combination
with immunotherapeutic agents for the treatment of several
different cancer types, including pancreatic, ovarian, non-small
cell lung cancer, and mesothelioma. Verastem’s product candidates
seek to treat cancer by modulating the local tumor
microenvironment, enhancing anti-tumor immunity and reducing cancer
stem cells. For more information, please visit
www.verastem.com.
Verastem, Inc. forward-looking statements notice:
This press release includes forward-looking statements about
Verastem's strategy, future plans and prospects, including
statements regarding the development and activity of Verastem's
investigational product candidates, including duvelisib and
defactinib (VS-6063), and Verastem's PI3K and FAK programs
generally, the structure of our planned and pending clinical trials
and the timeline and indications for clinical development,
including reporting top-line data, and regulatory submissions, our
rights to develop or commercialize our product candidates and our
ability to finance contemplated development activities and fund
operations for a specified period. The words "anticipate,"
"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
Verastem'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 expected, including for the Phase 3 DUO™ study; that
enrollment of clinical trials may take longer than expected; that
our product candidates will cause unexpected safety events or
result in an unmanageable safety profile as compared to their level
of efficacy; that duvelisib will be ineffective at treating
patients with lymphoid malignancies; that Verastem will be unable
to successfully initiate or complete the clinical development of
its product candidates; that the development of Verastem's product
candidates will take longer or cost more than planned; that
Verastem may not have sufficient cash to fund its contemplated
operations; that Verastem or Infinity Pharmaceuticals, Inc.
(Infinity) will fail to fully perform under the duvelisib license
agreement; that Verastem may be unable to make additional draws
under its debt facility or obtain adequate financing in the future
through product licensing, co-promotional arrangements, public or
private equity, debt financing or otherwise; that Verastem will not
pursue or submit regulatory filings for its product candidates,
including for duvelisib in patients with CLL or iNHL; and that
Verastem's product candidates will not receive regulatory approval,
become commercially successful products, or result in new treatment
options being offered to patients. Other risks and uncertainties
include those identified under the heading "Risk Factors" in
Verastem's Annual Report on Form 10-K for the year ended December
31, 2016 and in any subsequent filings with the U.S. Securities and
Exchange Commission. The forward-looking statements contained in
this press release reflect Verastem's views as of the date of this
release, and Verastem does not undertake and specifically disclaims
any obligation to update any forward-looking statements.
References
1 Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and
PI3K-gamma inhibition by IPI-145 abrogates immune responses and
suppresses activity in autoimmune and inflammatory disease models.
Chem Biol 2013; 20:1-11.
2 Reif K et al. Cutting Edge: Differential Roles for
Phosphoinositide 3 kinases, p110-gamma and p110-delta, in
lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240.
3 Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs
Unexpectedly activate myeloid cell PI3K, a single convergent point
promoting tumor inflammation and progression. Cancer Cell
2011;19:715-727.
4 www.clinicaltrials.gov, NCT02004522
5 www.clinicaltrials.gov, NCT01882803
6 www.clinicaltrials.gov, NCT02783625, NCT02783625,
NCT02158091
7 Schaller M.D. and Parsons J.T. Focal adhesion kinase: an
integrin-linked protein tyrosine kinase. Trends Cell Biol. 1993 3:
258-62.
8 Jiang H et al. Targeting focal adhesion kinase renders
pancreatic cancers responsive to checkpoint immunotherapy. Nat Med
2016: Aug 22(8) 851-60.
9 Sulzmaier F.J. et al. FAK in cancer: mechanistic findings and
clinical applications. Nature Rev Cancer. 2014 14: 598-610.
10 www.clinicaltrials.gov, NCT02546531
11 www.clinicaltrials.gov, NCT02943317
12 www.clinicaltrials.gov, NCT02758587
Note: Presentations referenced in this press release can be
downloaded at www.verastem.com/research/posters.aspx
Verastem, Inc.
Condensed Consolidated Balance
Sheets
(in thousands)
June 30, December 31,
2017 2016 (unaudited) Cash, cash equivalents
and investments $ 57,918 $ 80,897 Prepaid expenses and other
current assets 1,835 398 Property and equipment, net 1,127 1,417
Other assets 959 917
Total assets $
61,839 $ 83,629 Accounts payable and
accrued expenses $ 11,066 $ 10,991 Long-term debt 2,293 — Other
liabilities 248 341 Stockholders’ equity 48,232
72,297
Total liabilities and stockholders’ equity $
61,839 $ 83,629
Verastem, Inc.
Unaudited Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
Three months ended June 30,
Six months ended June 30, 2017 2016
2017 2016 Operating expenses:
Research and development $ 9,042 $ 4,492 $ 17,427 $ 8,671 General
and administrative 4,425 4,217
9,188 8,472 Total operating expenses
13,467 8,709 26,615
17,143 Loss from operations (13,467 ) (8,709 ) (26,615 )
(17,143 ) Interest income 140 140 295 280 Interest expense
(109 ) — (121 ) —
Net
loss $ (13,436 ) $ (8,569
) $ (26,441 ) $ (16,863
) Net loss per share—basic and diluted $
(0.36 ) $ (0.23 ) $
(0.71 ) $ (0.46 )
Weighted-average number of
common
shares used in net loss per share-basic
and diluted
36,992 36,992
36,992 36,983
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version on businesswire.com: http://www.businesswire.com/news/home/20170808006089/en/
Verastem, Inc.Brian Sullivan,
781-292-4214bsullivan@verastem.com
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