Verastem, Inc. (NASDAQ: VSTM), focused on discovering and
developing drugs to treat cancer, today reported financial results
for the year ended December 31, 2016, and also provided an overview
of certain corporate developments.
“2016 was a year of significant achievement for Verastem with
the in-licensing of duvelisib, a late-stage, clinical product
candidate with broad potential across B-cell and T-cell lymphoid
malignancies, and the advancement of defactinib into clinical
development in combination with immuno-oncology agents,”
said Robert Forrester, President and Chief Executive Officer
of Verastem. “As we enter 2017, we are laser-focused on
several important milestones, beginning with reporting top-line
duvelisib data from the Phase 3 DUO™ study in chronic lymphocytic
leukemia (CLL) expected mid-year 2017. There remains an unmet
medical need for patients with relapsed CLL. We believe duvelisib
has potential as a convenient, oral monotherapy with an expected
and manageable safety profile for patients with relapsed CLL. For
defactinib, we look forward to advancing our ongoing combination
trials into important expansion cohorts across several high unmet
need indications.”
Mr. Forrester continued, “On the financial front, we ended 2016
with $80.9 million in cash, cash equivalents and investments, which
we believe is sufficient to support our research and development
programs and operations into 2018. In March 2017, we entered into a
loan facility with Hercules Capital, Inc. for up to $25.0 million,
subject to certain conditions including positive DUO data, which
would provide us with additional financial flexibility to advance
duvelisib.”
Fourth Quarter 2016 and Recent Highlights:
Duvelisib
- In-licensed Late-stage,
Complementary Oncology Product Candidate Duvelisib – Verastem
and Infinity Pharmaceuticals, Inc. (Infinity) announced the signing
of an agreement under which Verastem licensed exclusive worldwide
rights to develop and commercialize duvelisib, an investigational
product candidate currently in development for hematologic
malignancies. Duvelisib is well aligned with Verastem's strategic
focus of developing novel anti-cancer therapeutics that modulate
the tumor microenvironment. The transaction provides a new oncology
product candidate with demonstrated activity in lymphoid
malignancies.
- Ongoing Phase 3 DUO Study in
Relapsed or Refractory CLL – The safety and efficacy 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
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.
- Positive Phase 2 DYNAMO™ Data
Reported at ASH 2016 – Positive Phase 2 clinical data from the
DYNAMO study demonstrating the clinical activity of duvelisib in
patients with relapsed refractory indolent non-Hodgkin lymphoma
(iNHL) were presented at the 58th American Society of Hematology
(ASH) Annual Meeting in December 2016. In an oral presentation,
titled “A phase 2 study demonstrating the clinical activity of
duvelisib in patients with relapsed refractory indolent non-Hodgkin
lymphoma,” (Publication ID: 1218) Ian Flinn, M.D., Ph.D. (Director,
Hematologic Malignancies Program, Sarah Cannon Research Institute),
described results from 129 patients with double refractory iNHL
(median 3 prior anti-cancer regimens, range 1-18). The study met
its primary endpoint, achieving an ORR of 46% as determined by an
independent review committee (IRC) (p=0.0001; 95% CI 0.37-0.55).
Among disease subgroups, the ORR was 41% in follicular lymphoma
(n=83), 68% in small lymphocytic lymphoma (n=28), and 33% in
marginal zone lymphoma (n=18). The median DOR among all patients
was 9.9 months. Notably, 83% of patients had reductions in the size
of their target lymph nodes per the IRC. Duvelisib was generally
well tolerated, with an expected and manageable safety profile with
appropriate risk mitigation. The DYNAMO study showed that duvelisib
monotherapy has a favorable benefit-risk profile in refractory iNHL
patients and may represent an important treatment option in this
population.
Defactinib (VS-6063)
- 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 Verastem’s defactinib, an investigational focal
adhesion kinase (FAK) inhibitor, 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.
Corporate and Financial
- 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 our hematology-oncology clinical programs. Dr.
Youssoufian brings over 25 years of product development and
commercialization experience to Verastem, having served in senior
leadership roles at several oncology-focused companies, including
BIND Therapeutics, Progenics Pharmaceuticals, Ziopharm Oncology,
Imclone Systems, Sanofi Aventis and Bristol-Myers Squibb where he
was involved in the development of Sprycel®, Taxotere® and
Erbitux®.
- Additional Key Personnel
Appointments – Recently, Michael Ferraresso joined Verastem as
Vice President, Commercial Operations, and Verastem also appointed
several highly experienced individuals to the Clinical and
Scientific Advisory Board including:
- Lori Kunkel, M.D., Former Chief Medical
Officer, Pharmacyclics
- Edmund J. Pezalla, M.D., MPH, Former
VP, Pharmaceutical Policy and Strategy at Aetna
- Greg Berk, M.D., Former Chief Medical
Officer, Verastem
- Cheryl Cohen, Former Chief Commercial
Officer, Medivation
- Brian Stuglik, PharmD., Former VP and
Chief Marketing Officer, Oncology Global Marketing, 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.
Full Year 2016 Financial Results
Net loss for the year ended December 31, 2016 (2016 Period)
was $36.4 million, or $0.99 per share, as compared to a
net loss of $57.9 million, or $1.61 per share, for the year
ended December 31, 2015 (2015 Period). Net loss includes non-cash
stock-based compensation expense of $6.2 million and $9.7
million for the 2016 Period and 2015 Period, respectively.
Research and development expense for the 2016 Period was $19.8
million compared to $40.6 million for the 2015 Period. The $20.8
million decrease from the 2015 Period to the 2016 Period was
primarily related to a decrease of $15.6 million in external
contract research organization expense for outsourced biology,
chemistry, development and clinical services, which includes our
clinical trial costs, a $3.4 million decrease in personnel related
costs, primarily due to the reduction in workforce in October 2015,
a decrease of $1.3 million in stock-based compensation expense and
a decrease of $1.5 million in lab supplies, travel and other
research and development expense. These decreases were partially
offset by an increase of approximately $947,000 in consulting and
professional fees.
General and administrative expense for the 2016 Period was $17.2
million compared to $17.6 million for the 2015 Period. The
approximate $411,000 decrease from the 2015 Period to the 2016
Period primarily resulted from a decrease of $2.1 million in
stock-based compensation expense. This decrease was partially
offset by increases of $1.1 million in consulting and professional
fees, approximately $280,000 in personnel costs, and a net increase
of approximately $306,000 of other general and administrative
costs.
As of December 31, 2016, Verastem had cash, cash
equivalents and investments of $80.9 million compared
to $110.3 million as of December 31, 2015.
Verastem used $29.5 million for operating activities
during the 2016 Period.
The number of outstanding common shares as of December 31,
2016, 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.
Conference Call Information
The Verastem management team will host a conference call today,
Thursday, March 23, 2017, at 4:30 PM (ET). The call can be accessed
by dialing 1-877-341-5660 or 1-315-625-3226 five minutes prior to
the start of the call and providing the passcode 89196444.
The live, listen-only webcast of the conference call can be
accessed by visiting the investors section of the Company’s website
at www.verastem.com. A replay of the webcast will be archived on
the Company’s website for 90 days following the call.
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.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 or refractory CLL4, 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 data5. 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 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.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 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.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 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 and 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 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 will fail to fully perform
under the duvelisib license agreement; that the transition of the
duvelisib program from Infinity will not be completed; 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, NCT020045225
www.clinicaltrials.gov, NCT018828036 www.clinicaltrials.gov,
NCT02783625, NCT02783625, NCT021580917Schaller MD and Parsons JT.
Focal adhesion kinase: an integrin-linked protein tyrosine kinase.
Trends Cell Biol. 1993 3: 258-62.8Jiang H et al. Targeting focal
adhesion kinase renders pancreatic cancers responsive to checkpoint
immunotherapy. Nat Med 2016: Aug 22(8) 851-60.9Sulzmaier FJ et al.
FAK in cancer: mechanistic findings and clinical applications.
Nature Rev Cancer. 2014 14: 598-610.10 www.clinicaltrials.gov,
NCT0254653111 www.clinicaltrials.gov, NCT0294331712
www.clinicaltrials.gov, NCT02758587
Verastem, Inc.
Unaudited Selected Consolidated Balance
Sheets
(in thousands)
December 31,
December 31, 2016 2015 Cash, cash equivalents
and investments $ 80,897 $ 110,258 Prepaid expenses and other
current assets 398 585 Property and equipment, net 1,417 2,048
Other assets 917 203
Total assets $
83,629 $ 113,094 Accounts payable and
accrued expenses $ 10,991 $ 10,040 Other liabilities 341 585
Stockholders’ equity 72,297 102,469
Total
liabilities and stockholders’ equity $ 83,629
$ 113,094
Verastem, Inc.
Unaudited Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
Year ended December 31,
2016 2015 Operating expenses: Research
and development $ 19,779 $ 40,565 General and administrative
17,223 17,634 Total operating expenses 37,002
58,199 Loss from operations (37,002) (58,199) Interest income
562 334
Net loss $ (36,440)
$ (57,865) Net loss per share—basic and
diluted $ (0.99) $ (1.61)
Weighted-average number of common
shares used in net loss per share-basic and diluted
36,988 35,932
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170323006244/en/
Verastem, Inc.Brian Sullivan,
781-292-4214bsullivan@verastem.com
Verastem (NASDAQ:VSTM)
Historical Stock Chart
From Aug 2024 to Sep 2024
Verastem (NASDAQ:VSTM)
Historical Stock Chart
From Sep 2023 to Sep 2024