Updated Phase 1/2 Data in Recurrent Low-Grade
Serous Ovarian Cancer (LGSOC) Continues to Demonstrate Activity,
Durability and a Favorable Tolerability Profile for VS-6766 +
Defactinib Combination
Two Registration-Directed Phase 2 Trials
Underway in LGSOC and KRAS G12V Non-Small Cell Lung Cancer with
Multiple Key Catalysts Expected in 1H 2022
Verastem, Inc. (Nasdaq: VSTM) (also known as Verastem Oncology),
a biopharmaceutical company committed to advancing new medicines
for patients battling cancer, today reported financial results for
the three months ended March 31, 2021, highlighted recent progress
and outlined key corporate objectives.
“During the first quarter of 2021, we expanded the selection
portion of the Phase 2 RAMP 201 study to include all recurrent
low-grade serous ovarian cancer types. This decision was based on
the positive updated data from the LGSOC cohort of the Phase 1/2
FRAME which continues to show strong response rates across both
KRAS mutant and wild-type recurrent LGSOC, along with robust
durability and a favorable tolerability profile,” said Brian
Stuglik, Chief Executive Officer of Verastem Oncology. “We closed
the first quarter with just over $127 million in cash, cash
equivalent and investments, leaving us well positioned to execute
on our two ongoing Phase 2 studies evaluating VS-6766 and
defactinib in LGSOC and KRAS G12V non-small cell lung cancer
(NSCLC), as well as our other key corporate objectives.”
Recent Corporate Highlights
LGSOC
- Reported updated data from the LGSOC cohort of the ongoing,
investigator-sponsored Phase 1/2 FRAME study evaluating VS-6766 in
combination with defactinib in patients with recurrent LGSOC.
Combination continues to demonstrate activity, durability and a
favorable tolerability profile, including in patients who have
progressed following treatment with a MEK inhibitor.
- Overall response rate (ORR) across all patients was 52% (11 of
21 patients).
- ORR for patients with KRAS mutant LGSOC was 70% (7 of 10
patients).
- ORR for patients with wild type LGSOC was 44% (4 of 9
patients).
- The most common side effects were Grade 1/2 rash, creatine
kinase elevation, nausea, hyperbilirubinemia and diarrhea, which
were reversible.
- Company-sponsored, registration-directed Phase 2 study (RAMP
201) underway investigating VS-6766 alone and in combination with
defactinib for the treatment of recurrent LGSOC. Study recently
expanded to include both KRAS mutant and KRAS wild-type patients in
the selection phase to determine the optimal go-forward regimen for
both types of LGSOC.
KRAS G12V Mutant NSCLC
- Company-sponsored, registration-directed Phase 2 study (RAMP
202) underway investigating VS-6766 alone and in combination with
defactinib for the treatment of patients with KRAS G12V mutant
NSCLC.
Upcoming Milestones and Key Priorities for 2021-2022
LGSOC
- Updated data from Phase 1/2 FRAME study to be submitted for
presentation at a major medical meeting during the second half of
2021.
- Complete selection portion of RAMP 201 during first half of
2022; commence expansion portion.
G12V NSCLC
- Complete selection portion of RAMP 202 during first half of
2022; commence expansion portion.
First Quarter 2021 Financial Results
Verastem Oncology ended the first quarter 2021 with cash, cash
equivalents and investments of $127.1 million.
Total revenue for the three months ended March 31, 2021 (2021
Quarter) was $1.0 million, compared to $5.1 million for the three
months ended March 31, 2020 (2020 Quarter).
Total operating expenses for the 2021 Quarter were $15.1
million, compared to $31.4 million for the 2020 Quarter.
Selling, general and administrative expenses for the 2021
Quarter were $6.2 million, compared to $19.6 million for the 2020
Quarter. The decrease of $13.4 million, or 68.4%, primarily
resulted from the Company’s shift in strategic direction and
COPIKTRA sale to Secura Bio, Inc., which led to lower employee
related expenses and consulting and professional fees.
Research and development expense for the 2021 Quarter was $8.9
million, compared to $10.9 million for the 2020 Quarter. The
decrease of $2.0 million, or 18.3%, was primarily related to the
upfront non-refundable payment of $3.0 million to Chugai
Pharmaceutical Co., Ltd for the VS-6766 license in the 2020 Quarter
and decreased contract research organization costs, partially
offset by increased drug substance and drug product costs and
increased investigator sponsored trial expenses.
Net loss for the 2021 Quarter was $15.0 million, or $0.09 per
share (basic and diluted), compared to $38.0 million, or $0.35 per
share (basic and diluted), for the 2020 Quarter.
For the 2021 Quarter, non-GAAP adjusted net loss was $12.4
million, or $0.07 per share (diluted), compared to non-GAAP
adjusted net loss of $21.3 million, or $0.20 per share (diluted),
for the 2020 Quarter. Please refer to the GAAP to Non-GAAP
Reconciliation attached to this press release.
Financial Guidance and Outlook
With the proceeds from the sale of COPIKTRA, Verastem Oncology
expects that it will have 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 KRAS mutant NSCLC. Verastem Oncology expects its 2021
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, 2021 and 2020 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 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.1,2
About the VS-6766/Defactinib Combination
RAS mutant tumors are present in ~30% of all human cancers, 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 patients with KRAS mt tumors. In an ongoing
investigator-initiated Phase 1/2 FRAME study, the combination of
VS-6766 and defactinib is being evaluated in patients with LGSOC,
KRAS mt NSCLC and colorectal cancer (CRC). The FRAME study was
expanded to include new cohorts in pancreatic cancer, KRASmt
endometrioid cancer and KRAS-G12V NSCLC. Verastem Oncology is also
supporting an investigator-initiated Phase 2 trial evaluating
VS-6766 with defactinib in patients with metastatic uveal
melanoma.
Verastem Oncology has initiated Phase 2 registration-directed
trials of VS-6766 with defactinib in patients with recurrent LGSOC
and in patients with recurrent KRAS-G12V NSCLC as part of its RAMP
(Raf And Mek Program).
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a development-stage
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
RAF/MEK inhibition and focal adhesion kinase (FAK) inhibition. For
more information, please visit www.verastem.com.
Forward-Looking Statements Notice
This press release includes forward-looking statements about
Verastem Oncology’s strategy and future plans and prospects,
including statements related to the expected use of the Company’s
cash and cash equivalents, the potential clinical value of the
RAF/MEK/FAK combination and the timing of commencing and completing
registration-directed trials for 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 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 or Chugai Pharmaceutical Co., Ltd. will fail to
fully perform under the VS-6766 license agreement; that we may not
have sufficient cash to fund our contemplated operations; that we
may be unable to 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, 2020 as filed with the Securities
and Exchange Commission (SEC) on March 18, 2021 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.
References
1 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.
2 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.
Verastem, Inc.
Condensed Consolidated Balance
Sheets
(in thousands)
(unaudited)
March 31,
December 31,
2021
2020
Cash, cash equivalents, &
investments
$
127,095
$
147,221
Accounts receivable, net
1,125
239
Prepaid expenses and other current
assets
5,456
3,473
Property and equipment, net
335
416
Right-of-use asset, net
2,628
2,726
Restricted cash and other assets
262
274
Total assets
$
136,901
$
154,349
Current Liabilities
$
11,844
$
17,093
Convertible senior notes
19,672
19,051
Lease Liability, long-term
2,776
2,931
Stockholders’ equity
102,609
115,274
Total liabilities and stockholders’
equity
$
136,901
$
154,349
Verastem, Inc.
Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
(unaudited)
Three months ended March
31,
2021
2020
Revenue:
Product revenue, net
$
—
$
5,034
License and collaboration revenue
—
22
Sale of COPIKTRA license and related
assets revenue
850
—
Transition services revenue
156
—
Total revenue
1,006
5,056
Operating expenses:
Cost of sales - product
—
495
Cost of sales - intangible
amortization
—
392
Research and development
8,896
10,924
Selling, general and administrative
6,218
19,604
Total operating expenses
15,114
31,415
Loss from operations
(14,108
)
(26,359
)
Other expense
—
(1,313
)
Interest income
52
356
Interest expense
(975
)
(10,674
)
Net loss
(15,031
)
(37,990
)
Net loss per share—basic and diluted
$
(0.09
)
$
(0.35
)
Weighted average common shares outstanding
used in computing:
Net loss per share – basic and diluted
171,586
108,153
Verastem, Inc.
Reconciliation of GAAP to
Non-GAAP Financial Information
(in thousands, except per share
amounts)
(unaudited)
Three months ended March 31,
2021
2020
Net loss reconciliation
Net loss (GAAP basis)
$
(15,031
)
$
(37,990
)
Adjust:
Amortization of acquired intangible
asset
—
392
Stock-based compensation expense
1,980
1,370
Non-cash interest, net
636
8,779
Severance and Other
—
1,788
Change in fair value of derivative
—
1,313
Chugai license payment
—
3,000
Adjusted net loss (non-GAAP
basis)
$
(12,415
)
$
(21,348
)
Reconciliation of Net loss Per
Share
Net loss per share – diluted (GAAP
Basis)
$
(0.09
)
$
(0.35
)
Adjust per diluted share
Amortization of acquired intangible
asset
—
—
Stock-based compensation expense
0.01
0.01
Non-cash interest, net
0.01
0.08
Severance and Other
—
0.02
Change in fair value of derivative
—
0.01
Chugai license payment
—
0.03
Adjusted net loss per share –
diluted
(non-GAAP Basis)
$
(0.07
)
$
(0.20
)
Weighted average common shares outstanding
used in computing net loss per share—diluted
171,586
108,153
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210511006144/en/
Investors: Ajay Munshi Vice President, Corporate Development +1
781-469-1579 amunshi@verastem.com
Sherri Spear Argot Partners +1 212 600 1902
sherri@argotpartners.com
Media: Lisa Buffington Corporate Communications +1 781-292-4205
lbuffington@verastem.com
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