Two Phase 2, Registration-Directed Trials
Underway in High Unmet Need Indications with Multiple Key Catalysts
Expected in 1H 2022
RAMP 201 Low-Grade Serous Ovarian Cancer Trial
Expanded to Now Include Patients with KRAS Wild-Type Tumors in
Selection Phase Based on New Clinical Data
Strong Balance Sheet with Cash, Cash
Equivalents and Investments Totaling $147.2 Million, Expected to
Have Cash Runway Until at Least 2024
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 and full year ending December 31, 2020, and
highlighted its key corporate objectives for 2021 and early
2022.
“We are encouraged as we continue to gain greater understanding
of the profile of our RAF/MEK inhibitor VS-6766 and its role in
blocking multiple nodes in the RAS pathway without the resistance
and tolerability issues that have historically been challenges.
Based on its unique profile, we believe that VS-6766 could play a
significant role in combination therapy across multiple tumor types
and where patients need better options,” said Brian Stuglik, Chief
Executive Officer of Verastem Oncology. “We have a strong balance
sheet to execute on our corporate objectives and report across
multiple key catalysts. Our annual spend is projected to be
approximately $50 million for 2021, and we still anticipate being
comfortably funded until at least 2024.”
In Q4 2020, the Company advanced VS-6766 into two Phase 2
registration-directed clinical studies: RAMP 201 in patients with
recurrent low-grade serous ovarian cancer (LGSOC) and RAMP 202 in
patients with KRAS-G12V mutant non-small cell lung cancer (NSCLC).
Both are evaluating VS-6766 with the Company’s FAK inhibitor,
defactinib. The combination of VS-6766 and defactinib had been
found to be clinically active in patients with KRAS mutant tumors
through an ongoing investigator-initiated Phase 1/2 FRAME study.
The FRAME study is evaluating the combination in patients with
recurrent LGSOC, KRAS mutant NSCLC, KRAS-G12V mutant NSCLC,
pancreatic cancer and KRAS mutant endometrioid cancer.
“In the FRAME study, additional patients with recurrent LGSOC
have now responded to treatment with clinically meaningful results
for patients with KRAS wild-type tumors, which represents
approximately 70% of all LGSOC patients. The results add to the
robust and durable responses that have been demonstrated for
patients with KRAS mutations,” said Jonathan Pachter, Chief
Scientific Officer of Verastem Oncology. “The majority of LGSOC
cases are RAS pathway-driven, and these new data support the
premise that VS-6766 could be a treatment for all LGSOC patients.
Therefore, we are expanding the selection phase of our
company-sponsored RAMP 201 study to now include both patient
populations (KRAS mutant and KRAS wild-type) to determine the
optimal go-forward regimen for both.”
In an updated December 2020 read-out of the FRAME study LGSOC
cohort (n=24), the overall response rate (ORR) is 52% (11 of 21
response evaluable patients), with KRAS mutant ORR at 70% (7 of 10
response evaluable patients), KRAS wild-type ORR at 44% (4 of 9
response evaluable patients) and KRAS status undetermined ORR at 0%
(0 of 2 response evaluable patients). As reported previously, the
most common side effects seen in the study were rash, creatine
kinase elevation, nausea, hyperbilirubinemia and diarrhea, most
being NCI CTC Grade 1/2 and all were reversible. The data from the
LGSOC cohort are anticipated to be presented at a major medical
meeting during the second half of 2021.
Recent Corporate Highlights
- Phase 2 registration-directed (RAMP 201) study underway
investigating VS-6766 alone and in combination with defactinib for
the treatment of patients with recurrent LGSOC.
- Supportive updated data from LGSOC cohort of the
investigator-sponsored Phase 1/2 FRAME study continues to show
encouraging clinical activity, durability and a favorable safety
profile in both the overall patient population and in the subgroup
of patients with KRAS mutant LGSOC, including patients who had
previously progressed following treatment with a MEK
inhibitor.
- Ongoing data from the FRAME study are demonstrating clinically
meaningful results in patients with KRAS wild-type LGSOC in
addition to those with KRAS mutation as more patients have had an
opportunity to respond to treatment.
- Phase 2 registration-directed (RAMP 202) study underway
investigating VS-6766 alone and in combination with defactinib for
the treatment of patients with recurrent KRAS-G12V mutant
NSCLC.
- Closed COPIKTRA sale to Secura Bio, Inc. (Secura) in a deal
valued at up to $311 million, plus royalties.
- Ended 2020 with $147.2 million in cash, cash equivalents and
investments.
Upcoming Milestones and Key Priorities for 2021-2022
LGSOC
- Amend RAMP 201 protocol to add enrollment of patients with KRAS
wild-type LGSOC in selection phase.
- Report top-line results from the selection phase of RAMP 201
and commence expansion phase during the first half of 2022.
- Report updated data from Phase 1/2 FRAME study LGSOC cohort,
including the KRAS wild-type population, during the second half of
2021.
NSCLC
- Report top-line results from the selection phase of RAMP 202
and commence expansion phase during the first half of 2022.
- Updated data from the Phase 1/2 FRAME study NSCLC cohort will
be presented by the investigator at the 2021 American Association
for Cancer Research (AACR) Annual Meeting. The presentation will
include an update on all variants of mutant KRAS, including further
updates on the KRAS G12V NSCLC patients who had been previously
highlighted. As no new KRAS G12V patients were enrolled in the
original FRAME study, a KRAS G12V-specific NSCLC cohort of FRAME
has been opened in addition to the Company-sponsored RAMP 202
registration-directed trial.
Corporate and Financial
- Expect 2021 annual operating expenses of approximately $50
million.
Fourth Quarter 2020 Financial Results
Total revenue for the three months ended December 31, 2020 (2020
Quarter) was $0.5 million, compared to $3.6 million for the three
months ended December 31, 2019 (2019 Quarter).
Total research and development (R&D) and selling, general
and administrative (SG&A) expenses for the 2020 Quarter were
$17.2 million, compared to $36.2 million for the 2019 Quarter.
R&D expense for the 2020 Quarter was $10.1 million, compared
to $12.5 million for the 2019 Quarter. The decrease of $2.4
million, or 18.5%, was primarily related to a reduction in contract
research organization costs partially offset by an increase in drug
substance and drug product manufacturing costs.
SG&A expense for the 2020 Quarter was $7.1 million, compared
to $23.7 million for the 2019 Quarter. The decrease of $16.6
million, or 70.1%, primarily resulted from the Company’s shift in
strategic direction and COPIKTRA sale to Secura which led to lower
employee related expenses and consulting and professional fees.
Net loss for the 2020 Quarter was $(19.9) million, or $(0.12)
per share (basic and diluted), compared to $(38.8) million, or
$(0.51) per share (basic and diluted), for the 2019 Quarter.
For the 2020 Quarter, non-GAAP adjusted net loss was $(14.8)
million, or $(0.09) per share (diluted), compared to non-GAAP
adjusted net loss of $(30.3) million, or $(0.40) per share
(diluted), for the 2019 Quarter. Please refer to the GAAP to
Non-GAAP Reconciliation attached to this press release.
Full-Year 2020 Financial Results
Verastem Oncology ended 2020 with cash, cash equivalents and
investments of $147.2 million.
During the year ended December 31, 2020 (2020 Period), Verastem
Oncology repaid in full all principal, accrued and unpaid interest,
fees, and expenses under the Amended Loan Agreement with Hercules
in an aggregate amount of $37.4 million and the Amended Loan
Agreement was terminated along with Hercules’ commitment to provide
funding under any future term loans. All liens on substantially all
of the Company’s assets to secure the loans under the Amended Loan
Agreement have been terminated and released.
Total revenue for the 2020 Period was $88.5 million, compared to
$17.5 million for the year ended December 31, 2019 (2019
Period).
Sale of COPIKTRA license and related assets revenue for the 2020
Period was $70.0 million, compared to $0.0 million for the 2019
Period. The 2020 Period was comprised of a $70.0 million upfront
payment received as part of the COPIKTRA sale to Secura.
Net product revenue for the 2020 Period was $15.2 million,
compared to $12.3 million for the 2019 Period. License and
collaboration revenue for the 2020 Period was $2.9 million,
compared to $5.1 million for the 2019 Period.
Total R&D and SG&A expenses for the 2020 Period were
$104.1 million, compared to $147.0 million for the 2019 Period.
R&D expense for the 2020 Period was $41.4 million, compared
to $45.8 million for the 2019 Period. The decrease of $4.4 million,
or 9.6%, was primarily related to a reduction in contract research
organization costs and employee related expense, partially offset
by the $3.0 million up-front non-refundable payment made to Chugai
Pharmaceuticals, Co. Ltd. for the VS-6766 license in the first
quarter of 2020.
SG&A expense for the 2020 Period was $62.7 million, compared
to $101.2 million for the 2019 Period. The decrease of $38.5
million, or 38.0%, was primarily due to the Company’s shift in
strategic direction and sale of COPIKTRA business which led to
lower employee related expense and consulting and professional
fees.
Net loss for the 2020 Period was $(67.7) million, or $(0.44) per
share (basic and diluted), compared to $(149.2) million, or $(2.00)
per share (basic and diluted), for the 2019 Period.
For the 2020 Period, non-GAAP adjusted net loss was $(37.8)
million, or $(0.25) per share (diluted), compared to non-GAAP
adjusted net loss of $(126.0) million, or $(1.69) per share
(diluted), for the 2019 Period. 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 and
year ended December 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 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, future plans and prospects, including
statements related to the potential clinical value of the
RAF/MEK/FAK combination and the timing of commencing
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 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, 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)
December 31,
December 31,
2020
2019
Cash, cash equivalents, &
investments
$
147,221
$
75,506
Accounts receivable, net
239
2,524
Inventory
—
3,096
Restricted cash, prepaid expenses and
other current assets
3,473
3,835
Property and equipment, net
416
947
Intangible assets, net
—
20,008
Right-of-use asset, net
2,726
3,077
Restricted cash and other assets
274
36,053
Total assets
$
154,349
$
145,046
Current Liabilities
$
17,093
$
29,890
Long-term debt
—
35,067
Convertible senior notes
19,051
68,556
Lease Liability, long-term
2,931
3,489
Other liabilities
—
870
Stockholders’ equity
115,274
7,174
Total liabilities and stockholders’
equity
$
154,349
$
145,046
Verastem, Inc.
Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
(unaudited)
Three months ended December
31,
Year ended December
31,
2020
2019
2020
2019
Revenue:
Product revenue, net
$
134
$
3,617
$
15,232
$
12,339
License and collaboration revenue
—
—
2,912
5,117
Sale of Copiktra license and related
assets revenue
—
—
70,000
—
Transition services revenue
372
—
372
—
Total revenue
506
3,617
88,516
17,456
Operating expenses:
Cost of sales - product
12
332
1,765
1,238
Cost of sales - intangible
amortization
—
393
793
1,569
Cost of sales – sale of Copiktra license
and related assets
—
—
31,187
—
Research and development
10,153
12,455
41,376
45,778
Selling, general and administrative
7,095
23,728
62,755
101,212
Total operating expenses
17,260
36,908
137,876
149,797
Loss from operations
(16,754
)
(33,291
)
(49,360
)
(132,341
)
Other expense
—
(641
)
(1,313
)
(641
)
Interest income
18
611
515
4,381
Interest expense
(1,354
)
(5,453
)
(15,794
)
(20,608
)
Loss on debt extinguishment
(1,580
)
—
(1,580
)
—
Net loss before income taxes
$
(19,670
)
$
(38,774
)
$
(67,532
)
$
(149,209
)
Income tax expense
194
—
194
—
Net loss
(19,864
)
(38,774
)
(67,726
)
(149,209
)
Net loss per share—basic and diluted
$
(0.12
)
$
(0.51
)
$
(0.44
)
$
(2.00
)
Weighted average common shares outstanding
used in computing:
Net loss per share – basic and diluted
169,902
76,331
153,330
74,578
Verastem, Inc.
Reconciliation of GAAP to
Non-GAAP Financial Information
(in thousands, except per share
amounts)
(unaudited)
Three months ended December
31,
Year ended December
31,
2020
2019
2020
2019
Net loss reconciliation
Net loss (GAAP basis)
$
(19,864
)
$
(38,774
)
$
(67,726
)
$
(149,209
)
Adjust:
Amortization of acquired intangible
asset
—
393
793
1,569
Stock-based compensation expense
2,933
1,311
8,118
8,539
Non-cash interest, net
554
2,705
10,319
7,131
Severance and Other
(160
)
1,232
4,621
3,200
Change in fair value of derivative
—
641
1,313
641
Chugai license payment
—
—
3,000
—
Loss on debt extinguishment
1,580
—
1,580
Notes third party exchange costs
171
2,168
171
2,168
Adjusted net loss (non-GAAP
basis)
$
(14,786
)
$
(30,324
)
$
(37,811
)
$
(125,961
)
Reconciliation of Net loss Per
Share
Net loss per share – diluted (GAAP
Basis)
$
(0.12
)
$
(0.51
)
$
(0.44
)
$
(2.00
)
Adjust per diluted share
Amortization of acquired intangible
asset
—
—
—
0.02
Stock-based compensation expense
0.02
0.02
0.05
0.11
Non-cash interest, net
—
0.03
0.07
0.10
Severance and Other
—
0.02
0.03
0.04
Change in fair value of derivative
—
0.01
0.01
0.01
Chugai license payment
—
—
0.02
—
Loss on debt extinguishment
0.01
—
0.01
—
Notes third party exchange costs
—
0.03
—
0.03
Adjusted net loss per share –
diluted
(non-GAAP Basis)
$
(0.09
)
$
(0.40
)
$
(0.25
)
$
(1.69
)
Weighted average common shares outstanding
used in computing net loss per share—diluted
169,902
76,331
153,330
74,578
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210318005924/en/
Verastem Oncology Contacts:
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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