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 first
quarter ended March 31, 2017 and provided an overview of certain
corporate developments.
“Following the presentation of positive data from the DYNAMO™
study of duvelisib in indolent non-Hodgkins Lymphoma (iNHL) at the
American Society of Hematology conference in December 2016, we are
focused on executing against the important milestones that lie
ahead, beginning with reporting top-line duvelisib data from the
Phase 3 DUO™ study in chronic lymphocytic leukemia (CLL), which is
expected mid-year 2017,” said Robert Forrester, President and
Chief Executive Officer of Verastem. “We continue to believe
duvelisib has significant potential as a convenient, oral
monotherapy for patients with relapsed CLL and possibly other
lymphomas, where there remains an unmet medical need.”
Mr. Forrester continued, “For defactinib, the program continues
to advance across three ongoing clinical collaborations evaluating
focal adhesion kinase (FAK) inhibition in combination with
immuno-oncology agents.”
First Quarter 2017 and Recent Highlights:
Duvelisib
- Long Term Follow Up Data from the
DYNAMO Study Selected for Oral Presentation at the 14th
International Conference on Malignant Lymphoma (ICML) – In
early May, Verastem announced that an abstract highlighting long
term follow up data from the ongoing Phase 2 DYNAMO study was
selected for oral presentation at ICML 2017 in Lugano, Switzerland.
The presentation, titled “DYNAMO: A Phase 2 Study Demonstrating the
Clinical Activity of Duvelisib in Patients with Double-Refractory
Indolent Non-Hodgkin Lymphoma,” will be presented by Pier Luigi
Zinzani, M.D., Ph.D., of the University of Bologna Institute of
Hematology, on Thursday, June 15, 2017 at 15:40 CET in Room A,
Cinema Corso and Aula Magna (Lugano University).
- Ongoing Phase 3 DUO Study in
Relapsed or Refractory CLL – The efficacy and safety of
duvelisib is currently being evaluated in the randomized Phase 3
DUO study in patients with relapsed or refractory CLL. In the DUO
study, approximately 300 patients were randomized 1:1 to receive
duvelisib (25mg BID) or ofatumumab (8 weekly infusions, starting
with an initial intravenous dose of 300mg on day 1 followed by 7
weekly doses of 2,000mg, then 2,000mg monthly for 4 cycles). 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,
duration of response (DOR) and safety. Verastem expects to report
top-line data from the DUO study in mid-year 2017.
- Published Scientific Research
Demonstrating the Potential of Duvelisib in Combination with
Venetoclax – A recent publication1 in Leukemia by Patel and
colleagues provides scientific rationale for the combination of
duvelisib with the BCL2 inhibitor venetoclax for the treatment of
CLL. Using samples from duvelisib-treated CLL patients, this group
at the University of Texas MD Anderson Cancer Center found that
duvelisib-treatment increased expression of several pro-apoptotic
proteins such that the CLL cells were poised for apoptosis. They
went on to show that CLL cells from patients after duvelisib
treatment were killed more effectively by venetoclax than CLL cells
taken from the same patients before duvelisib treatment.
Defactinib (VS-6063)
- Presented Defactinib 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., Assistant
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).
- Dosed the First Patient in
Combination Trial of Defactinib and Avelumab in Patients with
Ovarian Cancer – As announced in January 2017, the first
patient was dosed in a new clinical trial evaluating avelumab, an
investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in
combination with defactinib in patients with advanced ovarian
cancer. This multicenter, open-label, dose-escalation and
dose-expansion Phase 1/2 clinical trial is designed to assess the
safety, pharmacokinetics, pharmacodynamics, and initial
observations of clinical activity of the avelumab/defactinib
combination in patients with recurrent or refractory stage III-IV
ovarian cancer. The study is being conducted in collaboration with
the alliance between Merck KGaA, Darmstadt, Germany, which in the
U.S. and Canada operates as EMD Serono, and Pfizer, and is expected
to enroll approximately 100 patients at up to 15 sites across the
U.S.
- Updated Data from the Window of
Opportunity Study in Mesothelioma Selected for Poster Presentation
at the American Society of Clinical Oncology (ASCO) 2017 Annual
Meeting – An abstract highlighting updated data from the
ongoing Phase 2 Window of Opportunity study was selected for a
poster presentation at ASCO 2017 in Chicago. The presentation,
titled “Effect of FAK inhibitor defactinib on tumor immune changes
and tumor reductions in a phase II window of opportunity study in
malignant pleural mesothelioma (MPM),” will be presented by Raphael
Bueno, M.D., of the Brigham and Women's Hospital and Harvard
Medical School, on Saturday, June 3, 2017 from 8:00-11:30am CT in
Hall A at McCormick Place.
Corporate and Financial
- Eric K. Rowinsky 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. Dr. Rowinsky 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 is replacing Paul A. Friedman, M.D. who is
transitioning from his role as Director to become a member of
Verastem’s Clinical and Scientific Advisory Board.
- Hagop Youssoufian, MSc, M.D., Named
Head of Hematology and Oncology Development – In January 2017,
Dr. Youssoufian assumed this leadership role at Verastem to oversee
the clinical and regulatory development of Verastem’s pipeline,
including duvelisib, and provide overall strategic and tactical
leadership to its hematology-oncology clinical programs. Dr.
Youssoufian brings over 25 years of product development and
commercialization experience to Verastem, having served as Chief
Medical Officer at BIND Therapeutics, Ziopharm Oncology and Imclone
Systems, and other senior roles at Progenics, Sanofi Aventis and
Bristol-Myers Squibb where he was involved in the development of
Sprycel®, Taxotere® Erbitux®, Cyramza®, Portrazza® and
Lartruvo®.
- Additional Key Personnel
Appointments – Michael Ferraresso joined Verastem as Vice
President, Commercial Operations, and Verastem also appointed
several highly experienced individuals to its Clinical and
Scientific Advisory Board, including Lori Kunkel, M.D., former
Chief Medical Officer at Pharmacyclics, Edmund J. Pezalla, M.D.,
MPH, Former Vice President, Pharmaceutical Policy and Strategy at
Aetna, Greg Berk, M.D., former Chief Medical Officer at Verastem,
Inc., Cheryl Cohen, former Chief Commercial Officer at Medivation,
Inc., and Brian Stuglik, R.Ph, former Vice President and Chief
Marketing Officer, Oncology Global Marketing at Eli Lilly.
- Secured $25 Million Loan
Facility – In March 2017, Verastem entered into a Loan and
Security Agreement with Hercules Capital, Inc. for up to $25.0
million in financing. Verastem received the first $2.5 million of
financing under the Loan and Security Agreement when the
transaction closed. The proceeds will be used for Verastem’s
ongoing research and development programs and for general corporate
purposes. Additional tranches of up to $22.5 million in aggregate
will be available subject to certain conditions, including positive
data from the Phase 3 DUO clinical trial evaluating duvelisib in
patients with relapsed or refractory CLL.
First Quarter 2017 Financial Results
Net loss for the three months ended March 31, 2017 (2017
Quarter) was $13.0 million, or $0.35 per share, as
compared to a net loss of $8.3 million, or $0.22 per share,
for the three months ended March 31, 2016 (2016 Quarter). Net loss
includes non-cash stock-based compensation expense of $1.2
million and $1.6 million for the 2017 Quarter and 2016
Quarter, respectively.
Research and development expense for the 2017 Quarter was
$8.4 million compared to $4.2 million for the 2016
Quarter. The $4.2 million increase from the 2016 Quarter to
the 2017 Quarter was primarily related to an increase of
$2.8 million in contract research organization expense for
outsourced biology, chemistry, development and clinical services,
which includes clinical trial costs, an increase in personnel
related costs of approximately $965,000, and an increase of
approximately $554,000 in consulting fees. These increases were
offset by a decrease in stock-based compensation and other expenses
of approximately $86,000.
General and administrative expense for the 2017 Quarter was
$4.8 million compared to $4.3 million for the 2016
Quarter. The increase of approximately $508,000 from the 2016
Quarter to the 2017 Quarter primarily resulted from an increase in
consulting and professional fees of approximately $922,000,
partially offset by a decrease in stock-based compensation expense
of approximately $397,000.
As of March 31, 2017, Verastem had cash, cash
equivalents and investments of $72.6 million compared
to $80.9 million as of December 31, 2016.
Verastem used $10.7 million for operating activities
during the 2017 Quarter.
The number of outstanding common shares as of March 31,
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 tumor-associated macrophages,
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 product candidates, including duvelisib and defactinib,
also 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.2,3,4
Duvelisib is currently being evaluated in late- and mid-stage
clinical trials, including DUO™, a randomized, Phase 3 monotherapy
study in patients with relapsed or refractory CLL5, and DYNAMO™, a
single-arm, Phase 2 monotherapy study in patients with refractory
iNHL that achieved its primary endpoint of ORR upon top-line
analysis of efficacy data6. Duvelisib is also being evaluated for
the treatment of hematologic malignancies through
investigator-sponsored studies, including T-cell lymphoma.7
Information about duvelisib clinical trials can be found on
www.clinicaltrials.gov.
About Defactinib
Defactinib is an investigational inhibitor of FAK, a
non-receptor tyrosine kinase encoded by the PTK-2 gene that
mediates oncogenic signaling in response to cellular adhesion and
growth factors.8 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.9,10 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 cancer, ovarian cancer, 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.11,12,13 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 in iNHL and is
currently being evaluated in a Phase 3 clinical trial in patients
with CLL. 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 cancer, ovarian cancer, 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.
References1 Patel V.M., et al. Duvelisib treatment is
associated with altered expression of apoptotic regulators that
helps in sensitization of chronic lymphocytic leukemia cells to
venetoclax (ABT-199). Leukemia. 2017 Feb 3. doi:
10.1038/leu.2016.382.2 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; 20:1-11.3 Reif K et al. Cutting
Edge: Differential Roles for Phosphoinositide 3 kinases, p110-gamma
and p110-delta, in lymphocyte chemotaxis and homing. J Immunol
2004:173:2236-2240.4 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;19:715-727.5 www.clinicaltrials.gov, NCT020045226
www.clinicaltrials.gov, NCT018828037 www.clinicaltrials.gov,
NCT02783625, NCT02783625, NCT021580918 Schaller M.D. and Parsons
J.T. Focal adhesion kinase: an integrin-linked protein tyrosine
kinase. Trends Cell Biol. 1993 3: 258-62.9 Jiang H et al. Targeting
focal adhesion kinase renders pancreatic cancers responsive to
checkpoint immunotherapy. Nat Med 2016: Aug 22(8) 851-60.10
Sulzmaier F.J. et al. FAK in cancer: mechanistic findings and
clinical applications. Nature Rev Cancer. 2014 14: 598-610.11
www.clinicaltrials.gov, NCT0254653112 www.clinicaltrials.gov,
NCT0294331713 www.clinicaltrials.gov, NCT02758587
Verastem, Inc.
Unaudited Condensed Consolidated Balance Sheets
(in thousands)
March 31, December 31, 2017 2016
Cash, cash equivalents and investments $ 72,571 $ 80,897 Prepaid
expenses and other current assets 1,434 398 Property and equipment,
net 1,271 1,417 Other assets 973
917
Total assets $
76,249 $ 83,629 Accounts payable and
accrued expenses $ 13,233 $ 10,991 Long-term debt 2,249 — Other
liabilities 295 341 Stockholders’ equity
60,472 72,297
Total liabilities and
stockholders’ equity $
76,249 $ 83,629
Verastem, Inc. Unaudited Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
Three months ended March 31, 2017 2016
Operating expenses: Research and development $ 8,385 $ 4,179
General and administrative 4,763 4,255
Total operating expenses 13,148 8,434
Loss from operations (13,148 ) (8,434 ) Interest income 155 140
Interest expense (12 ) —
Net loss
$ (13,005 ) $ (8,294 )
Net loss per share—basic and diluted $ (0.35
) $ (0.22 ) Weighted-average number
of common shares used in net loss per share-basic and diluted
36,992 36,975
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Verastem, Inc.Brian Sullivan,
781-292-4214bsullivan@verastem.com
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