Announced Path Forward for VS-6766 and
Defactinib Combination Following Meeting with FDA
Phase 2 Registration-Directed Trials Expected
to Commence by Year End 2020 in Both Low-Grade Serous Ovarian
Cancer and KRAS Mutant Non-Small Cell Lung Cancer
Company Monetizes COPIKTRA® (duvelisib)
Providing Cash Runway Until at Least 2024
Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology),
a biopharmaceutical company committed to developing and
commercializing new medicines for patients battling cancer, today
reported financial results for the three months ending June 30,
2020, and provided an overview of recent corporate highlights.
“The first half of 2020 has been a time of transformational
change at Verastem Oncology. We recently announced our newest
strategic transaction, the sale of COPIKTRA to Secura Bio, which
allows us to monetize this asset while focusing our resources and
efforts on advancing the VS-6766 and defactinib combination program
in KRAS mutant solid tumors,” commented Brian Stuglik, Chief
Executive Officer of Verastem Oncology. “We are now looking forward
to a catalyst-driven second half of 2020, including reporting
updated data from the LGSOC arm of the investigator-initiated Phase
1/2 FRAME study in September and commencing registration-directed
clinical trials in low-grade serous ovarian cancer (LGSOC) and KRAS
mutant non-small cell lung cancer (NSCLC) by the end of this
year.”
Second Quarter 2020 and Recent Highlights
- Announced Path Forward for VS-6766/Defactinib Combination in
LGSOC Following Meeting with U.S. FDA. Verastem announced today
that the company met with the FDA in July 2020 to discuss the
registration-directed study design for the VS-6766/defactinib
combination in patients with LGSOC. The FDA was supportive of the
Company’s development strategy and adaptive design for LGSOC.
Verastem’s NSCLC study will also be an adaptive design with a focus
on patients with KRAS-G12V mutant tumors. Verastem intends to seek
input from the FDA after completing the initial cohort of the lung
cancer study. Verastem expects to commence registration-directed
clinical trials for potential accelerated approval in LGSOC and
KRAS mutant NSCLC by the end of 2020.
- Selling COPIKTRA Franchise to Secura Bio in a Deal Totaling
$311 Million, Plus Royalties. Verastem recently announced its
entry into a definitive agreement to sell its global commercial and
development rights to COPIKTRA in all oncology indications to
Secura Bio, Inc. The transaction, which carries a total deal value
of up to $311 million, plus royalties, will provide Verastem’s
current programs with a cash runway until at least 2024 and will
allow the Company to focus its resources and efforts on the
clinical development of VS-6766, its RAF/MEK inhibitor, and
defactinib, its FAK inhibitor, in KRAS mutant solid tumors.
Verastem is pursuing development of this combination in LGSOC and
KRAS mutant NSCLC.
- Presented Preliminary Results from Investigator-initiated
Phase 1 FRAME Study Evaluating the Combination of VS-6766 and
Defactinib in KRAS Mutant Solid Tumors at AACR 2020 Virtual Meeting
I. In a virtual poster presentation, Udai Banerji, MBBS, MD,
DNB, PhD, FRCP, NIHR, Professor of Molecular Cancer Pharmacology at
The Institute of Cancer Research and Honorary Consultant in Medical
Oncology at The Royal Marsden NHS Foundation Trust, highlighted
data from this ongoing, open-label, dose-escalation and expansion
study in patients with KRAS mutant advanced solid tumors, including
LGSOC and NSCLC. Preliminary data demonstrated a 67% overall
response rate and long duration of therapy among patients with
LGSOC. Based on higher response rates seen in NSCLC patients with
KRAS-G12V mutations, Verastem will also be further exploring the
role of the VS-6766/defactinib combination in KRAS-G12V NSCLC.
Expansion cohorts remain ongoing in LGSOC and NSCLC and the study
will be expanding to include new cohorts in pancreatic, KRAS mutant
endometrial and KRAS-G12V NSCLC.
- Presented New Preclinical VS-6766/Defactinib Combination
Data in Uveal Melanoma at AACR 2020 Virtual Meeting II. In this
study, researchers identified and reinforced that FAK inhibition is
a viable pathway to inhibit downstream from the GNAQ pathway, which
is constitutively active in uveal melanoma. It was observed that
co-targeting FAK and RAF/MEK signaling led to tumor collapse in
uveal melanoma xenograft and liver metastasis models in vivo. Based
on these encouraging results, Verastem plans to support an
investigator-sponsored, Phase 2 clinical testing of the
VS-6766/defactinib combination in uveal melanoma, which is expected
to commence by the end of 2020.
- Appointed John H. Johnson to the Board of Directors. In
April, Verastem Oncology announced the appointment of John H.
Johnson to its Board of Directors. Mr. Johnson’s career spans
multiple executive management roles at leading global corporations
where he was responsible for overseeing oncology and immunology
drug development initiatives and commercialization. Mr. Johnson
will serve on the Compensation and Nominating and Governance
Committees.
Upcoming Milestones
- Close transaction with Secura Bio during the third quarter of
2020.
- Present updated data from the LGSOC cohort of the
investigator-initiated Phase 1/2 FRAME study evaluating VS-6766 and
defactinib in KRAS mutant solid tumors in September, including at
the 2nd Annual RAS-Targeted Drug Development Conference on
September 16, 2020.
- Present new preclinical data from studies investigating VS-6766
and defactinib in combination with KRAS-G12C inhibitors in
September, including at the 2nd Annual RAS-Targeted Drug
Development Conference on September 16, 2020.
- Commence registration-directed clinical trials in LGSOC and
KRAS mutant NSCLC by the end of 2020.
- Submit updated data from the NSCLC cohort of the
investigator-initiated Phase 1/2 FRAME study to the International
Association for the Study of Lung Cancer (IASLC) World Lung Cancer
Conference, taking place in January 2021.
Second Quarter 2020 Financial Results
Net product revenue for the three months ending June 30, 2020
(2020 Quarter) was $4.2 million, compared to $3.0 million for the
three months ending June 30, 2019 (2019 Quarter). License and
collaboration revenue for both the 2020 Quarter and 2019 Quarter
was $0.1 million.
Total operating expenses for the 2020 Quarter were $25.6
million, compared to $41.4 million for the 2019 Quarter.
Research and development (R&D) expense for the 2020 Quarter
was $9.3 million, compared to $11.3 million for the 2019 Quarter.
The decrease of $2.0 million, or 18%, was primarily related to a
decrease in contract research organization (CRO) costs and lower
employee related expense.
Selling, general and administrative (SG&A) expense for the
2020 Quarter was $15.4 million, compared to $29.3 million for the
2019 Quarter. The decrease of $13.9 million, or 47%, primarily
resulted from the company’s shift in strategic direction which led
to lower commercial program and employee related expense.
Net loss for the 2020 Quarter was $23.0 million, or $0.14 per
share (basic and diluted), compared to $42.2 million, or $0.57 per
share (basic and diluted), for the 2019 Quarter.
For the 2020 Quarter, non-GAAP adjusted net loss was $20.5
million, or $0.12 per share (diluted), compared to non-GAAP
adjusted net loss of $35.6 million, or $0.48 per share (diluted),
for the 2019 Quarter. Please refer to the GAAP to Non-GAAP
Reconciliation attached to this press release.
Verastem Oncology ended the second quarter of 2020 with cash,
cash equivalents and short-term investments of $160.8 million.
Financial Guidance and Outlook
With the proceeds from the sale of COPIKTRA, Verastem has a cash
runway until at least 2024 to deliver on the current programs for
VS-6766 and defactinib, including clinical and regulatory
milestones and development in LGSOC and KRASmt NSCLC. Verastem
expects its 2020 operating expenses to be approximately 40% lower
than its 2019 operating expenses. As a result of its new strategic
direction and operating plans, along with the expected sale of the
COPIKTRA franchise during the third quarter and associated
transition activities, the Company expects total operating expenses
for the full year 2020 to be in the range of $80 million to $90
million. Beginning in 2021 Verastem expects its annual operating
expenses to be approximately $50 million.
Use of Non-GAAP Financial Measures
To supplement Verastem Oncology’s condensed consolidated
financial statements, which are prepared and presented in
accordance with generally accepted accounting principles in the
United States (GAAP), the Company uses the following non-GAAP
financial measures in this press release: non-GAAP adjusted net
loss and non-GAAP net loss per share. These non-GAAP financial
measures exclude certain amounts or expenses from the corresponding
financial measures determined in accordance with GAAP. Management
believes this non-GAAP information is useful for investors, taken
in conjunction with the Company’s GAAP financial statements,
because it provides greater transparency and period-over-period
comparability with respect to the Company’s operating performance
and can enhance investors’ ability to identify operating trends in
the Company’s business. Management uses these measures, among other
factors, to assess and analyze operational results and trends and
to make financial and operational decisions. Non-GAAP information
is not prepared under a comprehensive set of accounting rules and
should only be used to supplement an understanding of the Company’s
operating results as reported under GAAP, not in isolation or as a
substitute for, or superior to, financial information prepared and
presented in accordance with GAAP. In addition, these non-GAAP
financial measures are unlikely to be comparable with non-GAAP
information provided by other companies. The determination of the
amounts that are excluded from non-GAAP financial measures is a
matter of management judgment and depends upon, among other
factors, the nature of the underlying expense or income amounts.
Reconciliations between these non-GAAP financial measures and the
most comparable GAAP financial measures for the three months ended
March 31, 2020 and 2019 are included in the tables accompanying
this press release after the unaudited condensed consolidated
financial statements.
About VS-6766
VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a
unique inhibitor of the RAF/MEK signaling pathway. In contrast to
other MEK inhibitors in development, VS-6766 blocks both MEK kinase
activity and the ability of RAF to phosphorylate MEK. This unique
mechanism allows VS-6766 to block MEK signaling without the
compensatory activation of MEK that appears to limit the efficacy
of other inhibitors.
About Defactinib
Defactinib (VS-6063) is an oral small molecule inhibitor of FAK
and PYK2 that is currently being evaluated as a potential
combination therapy for various solid tumors. The Company has
received Orphan Drug designation for defactinib in ovarian cancer
and mesothelioma in the US, EU and Australia. Preclinical research
by Verastem Oncology scientists and collaborators at world-renowned
research institutions has described the effect of FAK inhibition to
enhance immune response by decreasing immuno-suppressive cells,
increasing cytotoxic T cells, and reducing stromal density, which
allows tumor-killing immune cells to enter the tumor.i,ii
About the VS-6766/Defactinib Combination
RAS mutant tumors are present in 30% of all human cancers and
have historically presented a difficult treatment challenge and are
often associated with significantly worse prognosis. Challenges
associated with identifying new treatment options for these types
of cancers include resistance to single agents, identifying
tolerable combination regimens with MEK inhibitors and new RAS
inhibitors in development addressing only a minority of all RAS
mutated cancers.
The combination of VS-6766 and defactinib has been found to be
clinically active in KRAS mutant tumors. In an ongoing
investigator-initiated Phase I/2 FRAME study, the combination of
VS-6766 and defactinib is being evaluated in patients with LGSOC,
KRAS mutant NSCLC and colorectal cancer (CRC). Preliminary data
from this study presented at the American Association for Cancer
Research (AACR) 2020 Virtual Annual Meeting I demonstrated a 67%
overall response rate and long duration of therapy among patients
with KRASmt LGSOC. Based on an observation of higher response rates
seen in patients with KRAS-G12V mutations in the study, Verastem
will also be further exploring the role of VS-6766 and defactinib
in KRAS-G12V NSCLC. The FRAME study is expanding in August 2020 to
include new cohorts in pancreatic, KRAS mutant endometrial and
KRAS-G12V NSCLC.
About COPIKTRA® (duvelisib)
COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase
(PI3K), and the first approved dual inhibitor of PI3K-delta and
PI3K-gamma, two enzymes known to help support the growth and
survival of malignant B-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.iii,iv,v COPIKTRA is indicated for the treatment
of adult patients with relapsed or refractory chronic lymphocytic
leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two
prior therapies and relapsed or refractory follicular lymphoma (FL)
after at least two prior systemic therapies. COPIKTRA is also being
developed by Verastem Oncology for the treatment of peripheral
T-cell lymphoma (PTCL), for which it has received Fast Track status
and Orphan Drug Designation, and is being investigated in
combination with other agents through investigator-sponsored
studies.vi For more information on COPIKTRA, please visit
www.COPIKTRA.com. Information about duvelisib clinical trials can
be found on www.clinicaltrials.gov.
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a commercial
biopharmaceutical company committed to the development and
commercialization of new medicines to improve the lives of patients
diagnosed with cancer. Our pipeline is focused on novel small
molecule drugs that inhibit critical signaling pathways in cancer
that promote cancer cell survival and tumor growth, including
phosphoinositide 3-kinase (PI3K), focal adhesion kinase (FAK) and
RAF/MEK inhibition.
Our first FDA approved product is available for the treatment of
patients with certain types of indolent non-Hodgkin’s lymphoma
(iNHL).
For more information, please visit www.verastem.com.
Forward-Looking Statements Notice
This press release includes forward-looking statements about
Verastem Oncology’s strategy, future plans and prospects, including
statements related to the expected sale of COPIKTRA, the Company’s
future funding requirements and the potential clinical value of the
RAF/MEK/FAK combination. The words "anticipate," "believe,"
"estimate," "expect," "intend," "may," "plan," "predict,"
"project," "target," "potential," "will," "would," "could,"
"should," "continue," “can,” “promising” 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 and
uncertainties, among other things, regarding: the satisfaction of
closing conditions with respect to the sale of the COPIKTRA assets
to Secura Bio; the ability of Secura Bio to achieve the clinical
and sales milestones necessary to result in additional
consideration payable to Verastem; the inherent uncertainty in
forecasting expected funding needs of the Company in advancing its
product candidates; the success in the development and potential
commercialization of our product candidates, including defactinib
in combination with VS-6766; the occurrence of adverse safety
events and/or unexpected concerns that may arise from additional
data or analysis or result in unmanageable safety profiles as
compared to their levels of efficacy; our ability to obtain,
maintain and enforce patent and other intellectual property
protection for our product candidates; the scope, timing, and
outcome of any legal proceedings; decisions by regulatory
authorities regarding labeling and other matters that could affect
the availability or commercial potential of our product candidates;
whether preclinical testing of our product candidates and
preliminary or interim data from clinical trials will be predictive
of the results or success of ongoing or later clinical trials; that
the timing, scope and rate of reimbursement for our product
candidates is uncertain; that third-party payors (including
government agencies) may not reimburse; 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 experience manufacturing or supply interruptions or
failures; that we will be unable to successfully initiate or
complete the clinical development and eventual commercialization of
our product candidates; that the development and commercialization
of our product candidates will take longer or cost more than
planned; that we may not have sufficient cash to fund our
contemplated operations; that we may be unable to make additional
draws under our debt facility or obtain adequate financing in the
future through product licensing, co-promotional arrangements,
public or private equity, debt financing or otherwise; that we will
be unable to execute on our partnering strategies for defactinib in
combination with VS-6766; that we will not pursue or submit
regulatory filings for our product candidates, and that our 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 the Company’s Annual Report on Form 10-K
for the year ended December 31, 2019 as filed with the Securities
and Exchange Commission (SEC) on March 11, 2020 and in any
subsequent filings with the SEC. The forward-looking statements
contained in this press release reflect Verastem Oncology’s views
as of the date hereof, and the Company does not assume and
specifically disclaims any obligation to update any forward-looking
statements whether as a result of new information, future events or
otherwise, except as required by law.
Verastem, Inc.
Condensed Consolidated Balance
Sheets
(in thousands)
(unaudited)
June 30,
December 31,
2020
2019
Cash, cash equivalents, &
investments
$
125,328
$
75,506
Accounts receivable, net
1,500
2,524
Inventory
6,316
3,096
Restricted cash, Prepaid expenses and
other current assets
11,448
3,835
Property and equipment, net
791
947
Intangible assets, net
19,223
20,008
Right-of-use asset, net
2,909
3,077
Restricted cash and other assets
31,017
36,053
Total assets
$
198,532
$
145,046
Current Liabilities
$
28,784
$
29,890
Long-term debt
30,899
35,067
Convertible senior notes
20,381
68,556
Lease Liability, long-term
3,225
3,489
Other liabilities
870
870
Stockholders’ equity
114,373
7,174
Total liabilities and stockholders’
equity
$
198,532
$
145,046
Verastem, Inc.
Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
(unaudited)
Three months ended June
30,
Six months ended June
30,
2020
2019
2020
2019
Revenue:
Product revenue, net
$
4,235
$
3,019
$
9,269
$
4,690
License and collaboration revenue
72
117
94
117
Total revenue
4,307
3,136
9,363
4,807
Operating expenses:
Cost of sales - product
392
377
887
534
Cost of sales - intangible
amortization
393
392
785
785
Research and development
9,344
11,346
20,268
21,103
Selling, general and administrative
15,442
29,298
35,046
55,331
Total operating expenses
25,571
41,413
56,986
77,753
Loss from operations
(21,264)
(38,277)
(47,623)
(72,946)
Other expense
—
—
(1,313)
—
Interest income
122
1,268
478
2,765
Interest expense
(1,868)
(5,185)
(12,542)
(10,115)
Net Loss
$
(23,010)
$
(42,194)
$
(61,000)
$
(80,296)
Net loss per share—basic and diluted
$
(0.14)
$
(0.57)
$
(0.45)
$
(1.09)
Weighted average common shares outstanding
used in computing net loss per share—basic and diluted
165,395
73,877
136,775
73,865
Verastem, Inc.
Reconciliation of GAAP to
Non-GAAP Financial Information
(in thousands, except per share
amounts)
(unaudited)
Three months ended June
30,
Six months ended June
30,
2020
2019
2020
2019
Net Loss Reconciliation
Net Loss (GAAP basis)
$
(23,010)
$
(42,194)
$
(61,000)
$
(80,296)
Adjust:
Amortization of acquired intangible
asset
393
392
785
785
Stock-based compensation expense
1,659
3,065
3,029
5,313
Non-cash interest, net
480
1,207
9,259
2,815
Severance and Other
11
1,957
1,798
1,994
Change in fair value of derivative
—
—
1,313
—
Chugai license payment
—
—
3,000
—
Adjusted Net Loss (non-GAAP
basis)
$
(20,467)
$
(35,573)
$
(41,816)
$
(69,389)
Reconciliation of Net Loss Per
Share
Net Loss per share – diluted
(GAAP Basis)
(0.14)
(0.57)
(0.45)
(1.09)
Adjust per diluted share
Amortization of acquired intangible
asset
—
—
0.01
0.01
Stock-based compensation expense
0.01
0.04
0.02
0.07
Non-cash interest, net
0.01
0.02
0.07
0.04
Severance and Other
—
0.03
0.01
0.03
Change in fair value of derivative
—
—
0.01
—
Chugai license payment
—
—
0.02
—
Adjusted Net Loss per share –
diluted
(non-GAAP Basis)
$
(0.12)
$
(0.48)
$
(0.31)
$
(0.94)
Weighted average common shares outstanding
used in computing net loss per share—diluted
165,395
73,877
136,775
73,865
i Gerber D. et al. Phase 2 study of the focal adhesion kinase
inhibitor defactinib (VS-6063) in previously treated advanced KRAS
mutant non-small cell lung cancer. Lung Cancer 2020: 139:60-67.
ii Chénard-Poirier, M. et al. Results from the biomarker-driven
basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor,
in RAS- or RAF-mutated malignancies including multiple myeloma.
Journal of Clinical Oncology 2017: 35.
10.1200/JCO.2017.35.15_suppl.2506.
iii 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.
iv 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.
v 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.
vi www.clinicaltrials.gov, NCT03372057.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200810005166/en/
Verastem Oncology Contacts:
Investors: John Doyle Vice President, Investor Relations &
Finance +1 781-469-1546 jdoyle@verastem.com
Joseph Rayne Argot Partners +1 212 600 1902
joseph@argotpartners.com
Media: Lisa Buffington Corporate Communications +1 781-292-4205
lbuffington@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