Verastem, Inc. (NASDAQ:VSTM), focused on developing and
commercializing drugs to improve the survival and quality of life
of cancer patients, today reported financial results for the year
ended December 31, 2017 and provided an overview of certain
corporate developments and plans.
“The last year has been marked by significant achievement for
Verastem with the reporting of positive data from the pivotal Phase
3 DUO™ study and culminating in the recent submission of a New Drug
Application (NDA) to the U.S. Food and Drug Administration (FDA)
seeking full approval for duvelisib for the treatment of relapsed
or refractory chronic lymphocytic leukemia/small lymphocytic
lymphoma (CLL/SLL) and accelerated approval for the treatment of
relapsed or refractory follicular lymphoma (FL),” said Robert
Forrester, President and Chief Executive Officer of Verastem. “As
we await the potential acceptance and approval of the duvelisib
NDA, we are diligently working to build our commercial
infrastructure and preparing for our first potential product
launch. I am delighted that Joe Lobacki has joined the team. Joe’s
formidable expertise in commercialising oncology drugs at
Medivation, Micromet and Genzyme positions Verastem to successfully
execute on our launch plan for duvelisib in the US.”
Fourth Quarter 2017 and Recent Highlights:
Duvelisib
- Duvelisib NDA submitted to FDA –
In early February 2018, Verastem submitted an NDA to the FDA
seeking full approval for its lead product candidate duvelisib, a
first-in-class oral dual inhibitor of phosphoinositide-3-kinase
(PI3K)-delta and PI3K-gamma, for the treatment of relapsed or
refractory CLL/SLL and accelerated approval for the treatment of
relapsed or refractory FL. The NDA is supported by clinical data
from the randomized Phase 3 DUO™ study, which met its primary
endpoint by demonstrating statistically significant efficacy, along
with a consistent and manageable safety profile, for duvelisib
monotherapy in patients with relapsed or refractory CLL/SLL. The
NDA is also supported by results from the Phase 2 DYNAMO™ study,
which also met its primary endpoint by demonstrating a
statistically significant improvement in overall response rate
(ORR) compared to an historical control in patients with indolent
non-Hodgkin’s lymphoma that are double-refractory to both rituximab
and chemotherapy or radioimmunotherapy.
- Clinical Data from Pivotal Phase 3
DUO Study Highlighted in an Oral Presentation at ASH 2017 –
Verastem presented results from the Phase 3 DUO study at the
American Society of Hematology 2017 Annual Meeting (ASH 2017). The
presentation, titled “Results from the Phase 3 DUO Trial: A
Randomized Comparison of Duvelisib vs Ofatumumab in Patients with
Relapsed/Refractory Chronic Lymphocytic Leukemia or Small
Lymphocytic Lymphoma,” was presented by principal investigator Ian
Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program
at Sarah Cannon Research Institute. The DUO study met its primary
endpoint with oral duvelisib monotherapy achieving a statistically
significant improvement in progression free survival (PFS) compared
to ofatumumab in patients with relapsed or refractory CLL/ SLL per
a blinded independent review committee (IRC) using modified
international workshop on CLL (iwCLL) and revised International
Working Group (IWG) Response Criteria (median PFS=13.3 months
versus 9.9 months, respectively; HR=0.52, p<0.0001),
representing a 48% reduction in the risk of progression or death.
Oral duvelisib monotherapy also achieved a statistically
significant improvement in ORR compared to ofatumumab (74% vs 45%,
respectively; p<0.0001), and reduced lymph node burden of less
than 50% in most patients compared to ofatumumab (85% vs 16%,
respectively). Duvelisib monotherapy demonstrated a manageable
safety profile, with results from this study consistent with the
well-characterized safety profile of duvelisib monotherapy in
patients with advanced hematologic malignancies in previous
studies. For duvelisib-treated patients, the median time on
treatment was 50.3 weeks (range, 0.9 - 160.0) compared to 23.1
weeks (range, 0.1 - 26.1) for ofatumumab.
- Additional Duvelisib Abstracts
Presented at ASH 2017 – Along with the Phase 3 DUO results, two
additional duvelisib abstracts were presented at ASH 2017. The
abstract, titled “In Vitro, In Vivo, and Parallel Phase I Evidence
Support the Safety and Activity of Duvelisib, a PI3K-δ,γ Inhibitor,
in Combination with Romidepsin or Bortezomib in Relapsed/Refractory
T-Cell Lymphoma,” was given as an oral presentation by Alison
Moskovitz, M.D., Memorial Sloan Kettering Cancer Center.
- Preclinical Data Highlighting the
Synergistic Effects in Combination with Immunotherapy Presented at
the American Society of Clinical Oncology Clinical Immuno-Oncology
Symposium (ASCO-SITC) – In January 2018, Jonathan Pachter,
Ph.D., Chief Scientific Officer of Verastem, presented preclinical
data highlighting the potential synergistic effects of duvelisib in
combination with immune checkpoint or co-stimulatory antibodies in
B-cell lymphoma. This data, outlined in a poster titled “The Dual
PI3K-δ,γ Inhibitor Duvelisib Stimulates Anti-Tumor Immunity and
Enhances Efficacy of Immune Checkpoint and Co-Stimulatory
Antibodies in a B-Cell Lymphoma Model,” supports the further
exploration of duvelisib in combination with anti-PD-1/PD-L1 or
co-stimulatory antibodies in patients with B-cell
malignancies.
Defactinib
- Defactinib Preclinical Abstract
Presented at ASH 2017 – A poster describing preclinical data in
combination with B-cell lymphoma 2 (BCL-2) was presented at ASH
2017. The abstract, titled “Combinatorial Inhibition of Focal
Adhesion Kinase and BCL-2 in AML,” was presented by Xiangmeng Wang,
Ph.D., MD Anderson Cancer Center.
Corporate and Financial
- Joseph Lobacki Appointed Chief
Commercial Officer – In January 2018, Verastem announced the
appointment of Joseph Lobacki as Executive Vice President and Chief
Commercial Officer. Mr. Lobacki, formerly Chief Commercial Officer
and Executive Council Member at Medivation, is responsible for
overseeing the commercial strategy and execution for Verastem’s
lead product candidate, duvelisib. Mr. Lobacki is a skilled leader
in commercializing oncology drugs and his strong experience in
hematologic oncology commercialization and marketing make him an
invaluable addition to the Verastem team.
- Additional Financing Through
Increasing Debt Facility to up to $50.0 Million and a $25.0 Million
Public Offering – In January 2018, Verastem amended its
loan and security agreement with Hercules Capital, Inc. (Hercules),
increasing its existing borrowing limit under the loan facility
from up to $25.0 million to up to $50.0 million in financing,
subject to certain conditions of funding. In December 2017, the
Company successfully completed an underwritten public offering of
shares of common stock with gross proceeds totaling
approximately $25.0 million.
- NgocDiep Le, MD, PhD, Appointed
Chief Medical Officer – In October 2017, Verastem announced the
appointment of Dr. Le as its Chief Medical Officer. A trained
medical oncologist, Dr. Le is board certified in internal medicine
and has 15 years of drug development experience across all phases
in both solid and liquid tumors, with specialized expertise in
clinical development. Dr. Le joins Verastem from MedImmune (a
wholly owned subsidiary of AstraZeneca) where she served as Vice
President, Immuno-Oncology Innovative Medicines and led the product
development teams for multiple high-priority immuno-oncology
assets. Dr. Le oversees the development strategy and activities for
Verastem’s core assets, duvelisib and defactinib.
- Paid First Development Milestone to
Infinity Pharmaceuticals – In October 2017, Verastem paid to
Infinity Pharmaceuticals, Inc. (Infinity) a $6.0 million milestone
payment, representing the first milestone under the duvelisib
license agreement. This milestone is based on the achievement of
positive top-line results from the Phase 3 DUO study evaluating the
efficacy and safety of duvelisib in patients with relapsed or
refractory CLL/SLL. The milestone was paid using funds drawn from
Verastem’s existing loan and security agreement with Hercules.
Full Year 2017 Financial Results
Net loss for the year ended December 31, 2017 (2017 Period)
was $67.8 million, or $1.76 per share, as compared to a
net loss of $36.4 million, or $0.99 per share, for the year
ended December 31, 2016 (2016 Period). Net loss includes non-cash
stock-based compensation expense of $5.0 million and $6.2
million for the 2017 Period and 2016 Period, respectively.
Verastem used $57.3 million of cash for operating
activities during the 2017 Period.
Research and development expense for the 2017 Period was
$46.4 million compared to $19.8 million for the 2016
Period. The $26.6 million increase from the 2016 Period to the 2017
Period was primarily related to an increase of $13.4 million in
external clinical research organization expense for outsourced
biology, chemistry, development and clinical services, which
includes our clinical trial costs, the achievement of a $6.0
million milestone pursuant to our license agreement with Infinity,
an increase of $5.1 million in consulting fees, and an increase in
personnel related costs of $1.9 million.
General and administrative expense for the 2017 Period was
$21.4 million compared to $17.2 million for the 2016
Period. The increase of $4.2 million from the 2016 Period to the
2017 Period primarily resulted from increases in consulting and
professional fees of $4.4 million, including $2.5 million related
to commercial launch preparation, and an increase in personnel
costs of $1.0 million. These increases were partially offset by a
decrease in stock-based compensation expense of $1.5 million.
As of December 31, 2017, Verastem had cash, cash
equivalents and investments of $86.7 million compared
to $80.9 million as of December 31, 2016.
The number of outstanding common shares as of December 31,
2017, was 50,800,908.
Financial Guidance
Based on our current operating plans, we expect to have
sufficient cash, cash equivalents and investments to fund our
current operating plan and capital expenditure requirements into
the second half of 2018.
About Duvelisib
Duvelisib is a first-in-class investigational, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes
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- and T-cells and is thought to play a role in the
formation and maintenance of the supportive tumor
microenvironment.1,2,3 Duvelisib was evaluated in late- and
mid-stage extension trials, including DUO™, a randomized, Phase 3
monotherapy study in patients with relapsed or refractory chronic
lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL),4 and
DYNAMO™, a single-arm, Phase 2 monotherapy study in patients with
refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and
DYNAMO achieved their primary endpoints and Verastem has submitted
a new drug application (NDA) requesting the full approval of
duvelisib for the treatment of patients with relapsed or refractory
CLL/SLL, and accelerated approval for the treatment of patients
with relapsed or refractory follicular lymphoma (FL). Duvelisib is
also being developed by Verastem for the treatment of peripheral
T-cell lymphoma (PTCL), and is being investigated in combination
with other agents through investigator-sponsored studies.6
Information about duvelisib clinical trials can be found on
www.clinicaltrials.gov.
About Defactinib
Defactinib is an investigational inhibitor of focal adhesion
kinase (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 and enhancement of anti-tumor immunity.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 (NSCLC), 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 developing and commercializing drugs to improve the
survival and quality of life of cancer patients. 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 indolent Non-Hodgkin Lymphoma (iNHL) and a Phase 3
clinical trial in patients with chronic lymphocytic leukemia/small
lymphocytic lymphoma (CLL/SLL). Verastem has submitted a New
Drug Application (NDA) requesting the full approval of duvelisib
for the treatment of patients with relapsed or refractory CLL/SLL,
and accelerated approval for the treatment of patients with
relapsed or refractory follicular lymphoma (FL). 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 (NSCLC), and
mesothelioma. Verastem’s product candidates seek to treat cancer by
modulating the local tumor microenvironment and enhancing
anti-tumor immunity. 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, and Verastem's PI3K and FAK programs generally, the
structure of our planned and pending clinical trials, Verastem’s
financial guidance and the timeline and indications for clinical
development and regulatory submissions. 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 acceptance or approval of the
NDA will not occur on the expected timeframes or at all; that even
if data from clinical trials is positive, regulatory authorities
may require additional studies for approval and the product may not
prove to be safe and effective; 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 the full data from the DUO
study will not be consistent with the previously presented results
of the study; that data may not be available when expected,
including for the Phase 3 DUO™ study; that the degree of market
acceptance of product candidates, if approved, may be lower than
expected; that the timing, scope and rate of reimbursement for our
product candidates is uncertain; that there may be competitive
developments affecting our product candidates; that data may not be
available when expected; 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/SLL 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, 2017 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 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 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 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, NCT021580917 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, NCT0254653111 www.clinicaltrials.gov,
NCT0294331712 www.clinicaltrials.gov, NCT02758587
Verastem, Inc.
Selected Consolidated Balance
Sheets
(in thousands)
December 31, December 31, 2017
2016 Cash, cash equivalents and investments $ 86,672
$ 80,897 Prepaid expenses and other current assets 1,115 398
Property and equipment, net 861 1,417 Other assets 1,143
917
Total assets $ 89,791 $
83,629 Accounts payable and accrued expenses $ 17,128
$ 10,991 Long-term debt 14,828 — Other liabilities 151 341
Stockholders’ equity 57,684 72,297
Total
liabilities and stockholders’ equity $ 89,791
$ 83,629
Verastem, Inc.
Consolidated Statements of
Operations
(in thousands, except per share
amounts)
Year ended December 31, 2017
2016 Operating expenses: Research and development $
46,423 $ 19,779 General and administrative 21,381
17,223 Total operating expenses 67,804
37,002 Loss from operations (67,804 ) (37,002 )
Interest income 561 562 Interest expense (559 ) —
Net loss $ (67,802 ) $
(36,440 ) Net loss per share—basic and diluted
$ (1.76 ) $ (0.99 )
Weighted-average number of common
shares used in net loss per share-basic and diluted
38,422 36,988
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Verastem, Inc.Marianne M. LambertsonVice President,
Corporate CommunicationsInvestor Relations/Public Relations+1
781-292-4273mlambertson@verastem.com
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