Presented Positive Results from Part A of RAMP
201 Trial of Avutometinib and Defactinib in Recurrent Low-Grade
Serous Ovarian Cancer (LGSOC) at American Society of Clinical
Oncology Meeting
Established Design for RAMP 301 Phase 3
Confirmatory Trial of Avutometinib and Defactinib in Recurrent
LGSOC
Strengthened Balance Sheet, Including Receipt
of Gross Proceeds of $97.8M from June 2023 Public Offering,
Bringing Company Cash, Cash Equivalents, and Investments to $183.1M
as of June 30, 2023
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company
committed to advancing new medicines for patients with cancer,
today reported financial results for the second quarter ending June
30, 2023 and highlighted recent progress.
“We made significant advancements in the second quarter,
including presenting positive results from the RAMP 201 trial of
avutometinib and defactinib in recurrent LGSOC and finalizing the
design of the confirmatory Phase 3 trial. Both are important
milestones in our plan to file for accelerated approval in LGSOC
based on mature results from RAMP 201 and data from the
investigator sponsored FRAME trial,” said Dan Paterson, President
and Chief Executive Officer, Verastem Oncology. “Our work to
strengthen our balance sheet will enable our continued progress
across our RAMP programs in LGSOC, non-small lung cancer and
pancreatic cancer and support continued preparation for a potential
commercial launch in LGSOC. We are encouraged by the progress we
have made and believe we are well positioned to address significant
unmet needs in RAS pathway-driven cancers.”
Second Quarter 2023 and Recent Highlights
Low Grade Serous Ovarian Cancer (LGSOC)
- The Company finalized the design of the Phase 3 confirmatory
trial (RAMP 301) of avutometinib and defactinib in LGSOC versus
standard of care (SOC) chemotherapy (pegylated liposomal
doxorubicin, paclitaxel, topotecan) or hormone therapy (letrozole,
anastrozole). RAMP 301 is an international collaboration between
The GOG Foundation, Inc. (GOG) and the European Network of
Gynaecological Oncological Trial groups (ENGOT) sponsored by
Verastem Oncology. The trial will enroll approximately 270 patients
who will be randomized to either the combination of avutometinib
and defactinib or SOC. The primary endpoint is progression free
survival (PFS) by blinded independent central review (BICR).
Secondary endpoints include overall response rates, duration of
response, disease control rate, safety and tolerability, patient
reported outcomes and overall survival.
- RAMP 301 is the follow-up confirmatory study being conducted
for full regulatory approval in recurrent LGSOC. The Company
intends to file for accelerated approval with the U.S. Food and
Drug Administration (FDA) for the combination of avutometinib and
defactinib based on mature data from the Company’s Phase 2
registration-directed trial, RAMP 201, together with the results of
the investigator-initiated FRAME trial.
- Data from Part A of the RAMP 201 trial were presented at the
American Society of Clinical Oncology Meeting in June. Results
included confirmed objective response rates (ORR) by BICR of 45%
(13/29; 95% CI: 26%-64%). Overall, patients were heavily
pretreated with a median of 4 prior systemic regimens (up to 11).
Tumor shrinkage was observed in the majority of patients, 86%
(25/29). The safety profile was tolerable and consistent with
previously reported safety data. These results are consistent with
the data that supported the Breakthrough Therapy Designation
granted by the FDA for the combination in recurrent LGSOC after one
or more prior lines of therapy, including platinum-based
chemotherapy.
Other Programs
- In the Company’s RAMP 203 and RAMP 204 Phase 1/2 clinical
trials, the combinations of avutometinib with Amgen’s LUMAKRAS®
(sotorasib) (RAMP 203) and with Mirati’s KRAZATI® (adagrasib) (RAMP
204) are evaluated in patients with KRAS G12C mutant non-small cell
lung cancer (NSCLC). RAMP 203 progressed to the recommendation of
the Phase 2 dose (avutometinib 4 mg BIW PO and sotorasib 960 mg QD
PO) and continues enrollment in Part B dose expansion in patients
who are G12C inhibitor treatment naïve and in patients who
experienced disease progression on prior G12C inhibitor
monotherapy. Dose escalation is ongoing in RAMP 204.
- Enrollment is ongoing in the Company’s RAMP 205 Phase 1b/2
clinical trial evaluating avutometinib and defactinib in
combination with SOC chemotherapy (GEMZAR® (gemcitabine) and
ABRAXANE®) in patients with metastatic adenocarcinoma of the
pancreas. The trial is supported by the Company’s receipt of the
first “Therapeutic Accelerator Award” from the Pancreatic Cancer
Action Network (PanCAN).
Corporate Updates
- Dan Paterson was promoted to President and Chief Executive
Officer in July. During his tenure as President and Chief Operating
Officer of Verastem Oncology, he spearheaded the acquisition of
lead compound avutometinib and led strategic direction designed to
accelerate the program’s advancement. In connection with his
appointment, Dan was also appointed to the Board of Directors. Dan
succeeds Brian Stuglik who has retired from his role as Chief
Executive Officer but remains a member of the Company’s Board of
Directors and leads the Board’s recently designated
Commercialization Committee.
- The Company strengthened the balance sheet in June 2023 by
raising gross proceeds of approximately $97.8 million in a public
offering of 8,489,409 shares of common stock and, in lieu of common
stock to certain investors, pre-funded warrants to purchase an
aggregate of 1,538,591 shares of common stock.
- Karin Tollefson was elected to Verastem Oncology’s Board of
Directors at the Company’s annual meeting, alongside returning
Board members Robert Gagnon and Brian Stuglik. Dr. Tollefson is the
Senior Vice President and Head of Global Medical Affairs at Seagen
Inc. Karin has 30 years of experience in the pharmaceutical
industry and is a proven leader in global oncology development and
medical affairs.
Second Quarter 2023 Financial Results
Verastem Oncology ended the second quarter of 2023 with cash,
cash equivalents and investments of $183.1 million. Total operating
expenses for the three months ended June 30, 2023 (the “2023
Quarter”) were $20.3 million, compared to $21.4 million for the
three months ended June 30, 2022 (the “2022 Quarter”). Recent
historical operating expenses have ranged between $16.0M and $20.0M
per quarter, which the Company does not anticipate will change
significantly in the near term as the RAMP 301 trial commences.
Research & development expenses for the 2023 Quarter were
$12.9 million, compared to $14.9 million for the 2022 Quarter. The
decrease of $2.0 million, or 13.4%, primarily resulted from a
decrease in drug product and drug substance costs and contract
research organization costs.
Selling, general & administrative expenses for the 2023
Quarter were $7.4 million, compared to $6.5 million for the 2022
Quarter. The increase of $0.9 million, or 13.8%, was primarily
related to increased consulting and professional fees as well as
additional costs in anticipation of a potential launch of
avutometinib and defactinib in LGSOC.
Net loss for the 2023 Quarter was $24.3 million, or $1.37 per
share (basic and diluted, each as adjusted for the Company’s
reverse stock split), compared to net loss of $22.0 million, or
$1.41 per share (basic and diluted, each as adjusted for the
Company’s reverse stock split) for the 2022 Quarter.
For the 2023 Quarter, non-GAAP adjusted net loss was $18.8
million, or $1.06 per share (diluted, as adjusted for the Company’s
reverse stock split), compared to non-GAAP adjusted net loss of
$20.1 million, or $1.29 per share (diluted, as adjusted for the
Company’s reverse stock split) for the 2022 Quarter. Please refer
to the GAAP to Non-GAAP Reconciliation attached to this press
release.
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 adjusted 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 and six months ended June 30,
2023, and 2022 are included in the tables accompanying this press
release, after the unaudited condensed consolidated financial
statements.
About Avutometinib (VS-6766)
Avutometinib is a RAF/MEK clamp that induces inactive complexes
of MEK with ARAF, BRAF and CRAF potentially creating a more
complete and durable anti-tumor response through maximal RAS
pathway inhibition. Avutometinib is currently in late-stage
development.
In contrast to other MEK inhibitors, avutometinib blocks both
MEK kinase activity and the ability of RAF to phosphorylate MEK.
This unique mechanism allows avutometinib to block MEK signaling
without the compensatory activation of MEK that appears to limit
the efficacy of other inhibitors. The U.S. Food and Drug
Administration granted Breakthrough Therapy designation for the
combination of Verastem Oncology’s investigational RAF/MEK clamp
avutometinib, with defactinib, its FAK inhibitor, for the treatment
of all patients with recurrent low-grade serous ovarian cancer
(LGSOC) regardless of KRAS status after one or more prior lines of
therapy, including platinum-based chemotherapy.
Verastem Oncology is currently conducting clinical trials with
its RAF/MEK clamp avutometinib in RAS- driven tumors as part of its
(Raf And Mek Program). RAMP 201 is a
registration-directed trial of avutometinib in combination with
defactinib in patients with recurrent LGSOC. Verastem Oncology has
established clinical collaborations with Amgen and Mirati to
evaluate LUMAKRAS® (sotorasib) and KRAZATI® (adagrasib) in
combination with avutometinib in KRAS G12C mutant NSCLC as part of
the RAMP 203 and RAMP 204 trials, respectively. As part of the
“Therapeutic Accelerator Award” Verastem Oncology received from
PanCAN, Verastem Oncology is conducting RAMP 205, a Phase 1b/2
clinical trial evaluating avutometinib and defactinib with
gemcitabine/nab-paclitaxel in patients with front-line metastatic
pancreatic cancer.
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 its financial condition, its future operating
expenses, the potential clinical value of various of its clinical
trials, the timing of commencing and completing trials, including
topline data reports and interactions with regulators. 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 avutometinib in combination with other
compounds, including defactinib, LUMAKRAS® and others; 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 trial design, labeling and
other matters that could affect the timing, 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 we may not attract and retain high quality
personnel; 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, including as a result of conducting
additional studies; that our target market for our product
candidates might be smaller than we are presently estimating; that
we or Chugai Pharmaceutical Co., Ltd. will fail to fully perform
under the avutometinib license agreement; that we or our other
collaboration partners may fail to perform under our collaboration
agreements; that any of our third party contract research
organizations, contract manufacturing organizations, clinical
sites, or contractors, among others, who we rely on fail to fully
perform; 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 Secura will achieve the milestones that result in
payments to us under our asset purchase agreement with Secura; that
we will be unable to execute on our partnering strategies for
avutometinib in combination with other compounds; 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 Verastem Oncology’s Annual Report on Form
10-K for the year ended December 31, 2022 as filed with the
Securities and Exchange Commission (SEC) on March 14, 2023 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 Verastem Oncology 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 Oncology
Condensed Consolidated Balance
Sheets
(in thousands)
(unaudited)
June 30, 2023
December 31, 2022
Cash, cash equivalents, &
investments
$
183,086
$
87,894
Accounts receivable, net
2
31
Prepaid expenses and other current
assets
6,875
4,945
Property and equipment, net
40
92
Right-of-use asset, net
1,494
1,789
Restricted cash and other assets
261
299
Total assets
$
191,758
$
95,050
Current Liabilities
$
20,787
$
21,663
Long term debt
39,739
24,526
Lease liability, long-term
1,022
1,470
Preferred stock tranche liability
7,460
—
Convertible preferred stock
21,159
—
Stockholders’ equity
101,591
47,391
Total liabilities, convertible
preferred stock and stockholders’ equity
$
191,758
$
95,050
Verastem Oncology
Condensed Consolidated
Statements of Operations
(in thousands, except per share
amounts)
(unaudited)
Three months ended June
30,
Six months ended June
30,
2023
2022
2023
2022
Revenue:
Sale of COPIKTRA license and related
assets revenue
$
—
$
—
$
—
$
2,596
Total revenue
—
—
—
2,596
Operating expenses:
Research and development
12,893
14,888
24,908
28,530
Selling, general and administrative
7,399
6,514
14,728
12,448
Total operating expenses
20,292
21,402
39,636
40,978
Loss from operations
(20,292
)
(21,402
)
(39,636
)
(38,382
)
Other income (expense)
(40
)
6
(47
)
34
Interest income
1,122
84
2,098
130
Interest expense
(1,121
)
(640
)
(1,890
)
(696
)
Change in fair value of preferred stock
tranche liability
(3,950
)
—
(520
)
—
Net loss
$
(24,281
)
$
(21,952
)
$
(39,995
)
$
(38,914
)
Net loss per share—basic and
diluted(1)
$
(1.37
)
$
(1.41
)
$
(2.32
)
$
(2.51
)
Weighted average common shares outstanding
used in computing:
Net loss per share – basic and
diluted(1)
17,732
15,539
17,231
15,530
(1) Amounts have been retroactively
restated to reflect the 1-for-12 reverse stock split effected on
May 31, 2023
Verastem Oncology
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,
2023
2022
2023
2022
Net loss reconciliation
Net loss (GAAP basis)
$
(24,281
)
$
(21,952
)
$
(39,995
)
$
(38,914
)
Adjust:
Stock-based compensation expense
1,432
1,758
2,745
3,404
Non-cash interest, net
112
94
76
111
Change in fair value of preferred stock
tranche liability
3,950
—
520
—
Severance and Other
—
—
38
—
Adjusted net loss (non-GAAP
basis)
$
(18,787
)
$
(20,100
)
$
(36,616
)
$
(35,399
)
Reconciliation of net loss per
share
Net loss per share – diluted (GAAP
Basis)(1)
(1.37
)
(1.41
)
(2.32
)
(2.51
)
Adjust per diluted share:
Stock-based compensation expense(1)
0.08
0.11
0.16
0.22
Non-cash interest, net(1)
0.01
0.01
—
0.01
Change in fair value of preferred stock
tranche liability(1)
0.22
—
0.03
—
Severance and Other(1)
—
—
—
—
Adjusted net loss per share –
diluted
(non-GAAP basis)(1)
$
(1.06
)
$
(1.29
)
$
(2.13
)
$
(2.28
)
Weighted average common shares outstanding
used in computing net loss per share—diluted(1)
17,732
15,539
17,231
15,530
(1) Amounts have been retroactively
restated to reflect the 1-for-12 reverse stock split effected on
May 31, 2023
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230808137851/en/
Investors:
Dan Calkins +1 781-469-1694 Investor Relations
dcalkins@verastem.com
Nate LiaBraaten +1 212-600-1902 nate@argotpartners.com
Media:
Lisa Buffington Corporate Communications +1 781-292-4502
lbuffington@verastem.com
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