Positive Interim Data Read-Out of RAMP 201 and
Productive FDA Meeting Support Avutometinib + Defactinib
Combination in Recurrent Low-Grade Serous Ovarian Cancer
(LGSOC)
Studies of Avutometinib Combinations in Other
RAS Pathway-Driven Cancers Advancing
Company Cash, Cash Equivalents, and Investments
of $87.9 Million as of December 31, 2022; Pro-Forma $117.9 Million
Including the Sale of Series B Convertible Preferred Stock
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company
committed to advancing new medicines for patients with cancer,
today reported financial results for the three months and full year
ended December 31, 2022, and highlighted recent progress.
“Building on our breakthrough therapy designation, we have made
significant progress advancing our LGSOC program, including
selecting the combination of avutometinib and defactinib as the
go-forward treatment regimen in recurrent disease regardless of
KRAS mutation with the goal of filing for accelerated approval upon
data maturity in the RAMP 201 trial and initiation of a
confirmatory study,” said Brian Stuglik, Chief Executive Officer of
Verastem Oncology. “We believe we are well-positioned to bring this
potential new combination therapy to patients who have been waiting
for the first U.S. Food and Drug Administration (FDA) approved
medicine for this disease. Our debt facility and recent private
placement offering add flexibility to our financial strength and
are expected to support the continued development of avutometinib
in combinations across RAS pathway-driven cancers of high unmet
need.”
Fourth Quarter 2022 and Recent Highlights
Low Grade Serous Ovarian Cancer (LGSOC)
- The Company held a productive meeting with the FDA to discuss
the encouraging results to date of the ongoing RAMP 201 LGSOC trial
evaluating avutometinib ± defactinib among patients with recurrent
LGSOC, confirmed the go forward treatment regimen selection of
avutometinib and defactinib based on a planned interim analysis
with prespecified criteria and discussed the regulatory path
forward.
- Of the 29 patients evaluable for response by blinded
independent central review (BICR) in the combination arm, the
initial results showed a confirmed objective response rate (ORR) of
28% in all patients and 27% vs 29% in KRAS mutant (n=15) and KRAS
wild-type (n=14) LGSOC, respectively. Three additional patients
with KRAS mutant LGSOC showed an unconfirmed partial response. The
overall disease control rate (stable disease plus partial response)
was 93%. Most evaluable patients (62%) were still on study
treatment on the combination arm at the time of the data cut with a
minimum follow-up of five months. No new safety signals were
reported with a continued favorable safety and tolerability
profile.
- Completed enrollment in primary cohort of 72 patients in the
combination arm of RAMP 201. Continued enrollment in the
combination arm of RAMP 201 is ongoing to expand the clinical
experience in anticipation of initiation of a confirmatory
study.
- The Company intends to include mature data from RAMP 201, the
Verastem sponsored clinical trial, and the FRAME study, led by The
Institute of Cancer Research, London, and The Royal Marsden NHS
Foundation Trust, to potentially support filing for accelerated
approval.
Other Programs
- In the Company’s RAMP 203 and RAMP 204 clinical trials,
evaluating the combination of avutometinib with Amgen’s LUMAKRAS®
(sotorasib) (RAMP 203) and with Mirati’s KRAZATI® (adagrasib) (RAMP
204) in KRAS G12C mutant NSCLC, RAMP 203 progressed to the final
dose escalation cohort and enrollment was initiated and dose
escalation is ongoing in RAMP 204.
- Initiated RAMP 205, a Phase 1b/2 clinical trial of avutometinib
and defactinib to evaluate a more complete blockade of KRAS
signaling with standard of care chemotherapy ((GEMZAR®
(gemcitabine) and ABRAXANE®)). The trial is supported by the
Company’s receipt of the first “Therapeutic Accelerator Award” from
the Pancreatic Cancer Network (PanCAN).
Corporate Updates
- The Company entered into a definitive agreement to sell up to
approximately 2.1 million shares of its Series B convertible
preferred stock to affiliates of BVF Partners L.P. in a private
placement to raise aggregate gross proceeds of up to approximately
$60 million in two tranches. On January 27, 2023, Verastem Oncology
closed on the initial tranche of 1.2 million shares of its Series B
convertible preferred stock with gross proceeds of $30
million.
- The Company has achieved the Term B Milestone pursuant to its
credit facility with Oxford and plans to draw down an additional
$15 million in March 2023. Under the credit facility, Verastem has
the ability to access up to an additional $110 million in a series
of tranches, $60 million of which are based on certain
pre-determined milestones and $50 million of which are at the
lender’s discretion.
- Intermittent dosing intellectual property for both avutometinib
alone and in combination with defactinib was recently allowed,
extending patent coverage up to 2038 and 2040, respectively.
- Appointed Anil Kapur, Executive Vice President, Corporate
Strategy and Chief Commercial Officer at Geron Corporation and
Robert Gagnon, the Chief Financial Officer and Operating Partner at
Gurnet Point Capital to its Board of Directors.
Fourth Quarter 2022 Financial Results
Verastem Oncology ended the fourth quarter of 2022 with cash,
cash equivalents and investments of $87.9 million. On a pro forma
basis, inclusive of the $30 million gross proceeds raised through
issuance of Series B convertible preferred stock, cash, cash
equivalents and investments were $117.9 million as of December 31,
2022. Additionally, the Company plans to draw down $15 million on
the Oxford Loan and Security Agreement in March 2023.
Total operating expenses for the three months ended December 31,
2022 (the “2022 Quarter”) were $16.8 million, compared to $17.1
million for the three months ended December 31, 2021 (the “2021
Quarter”).
Research & development expenses for the 2022 Quarter were
$10.7 million, compared to $11.4 million for the 2021 Quarter. The
decrease of $0.7 million, or 6.1%, primarily resulted from a
decrease in investigator fees and drug product and drug substance
costs.
Selling, general & administrative expenses for the 2022
Quarter were $6.1 million, compared to $5.7 million for the 2021
Quarter. The increase of $0.4 million, or 7.0%, was primarily
related to additional costs in anticipation of potential
commercialization of avutometinib and defactinib in LGSOC.
Net loss for the 2022 Quarter was $16.8 million, or $0.08 per
share (basic and diluted), compared to net loss of $16.5 million,
or $0.09 per share (basic and diluted) for the 2021 Quarter.
For the 2022 Quarter, non-GAAP adjusted net loss was $15.4
million, or $0.08 per share (diluted), compared to non-GAAP
adjusted net loss of $14.9 million, or $0.08 per share (diluted)
for the 2021 Quarter. Please refer to the GAAP to Non-GAAP
Reconciliation attached to this press release.
Full-Year 2022 Financial Results
Total revenue for the year ended December 31, 2022 (“2022
Period”) was $2.6 million, compared to $2.1 million for the year
ended December 31, 2021 (“2021 Period”). Revenue for the 2022
Period was primarily comprised of one regulatory milestone achieved
by Secura Bio, Inc.’s (“Secura”) sublicensee, CSPC Pharmaceutical
Group Limited. Revenue for the 2021 Period was primarily comprised
of two regulatory milestones achieved by Secura’s sublicensee,
Sanofi, and transition services revenue for certain support
functions provided to Secura pursuant to the Secura transition
services agreement, which was entered into in connection with the
sale of COPIKTRA to Secura.
Total operating expenses for the 2022 Period were $75.5 million,
compared to $63.5 million for the 2021 Period.
Research & development expenses for the 2022 Period were
$50.6 million, compared to $39.3 million for the 2021 Period. The
increase of $11.3 million, or 28.8%, was primarily related to an
increase in drug substance and drug product costs and contract
research organization costs.
Selling, general & administrative expenses for the 2022
Period were $25.0 million, compared to $24.1 million for the 2021
Period. The increase of $0.9 million, or 3.7%, was primarily
related to additional costs in anticipation of potential
commercialization of avutometinib and defactinib in LGSOC.
Net loss for the 2022 Period was $73.8 million, or $0.38 per
share (basic and diluted), compared to $71.2 million, or $0.41 per
share (basic and diluted) for the 2021 Period.
For the 2022 Period, non-GAAP adjusted net loss was $67.4
million, or $0.35 per share (diluted), compared to non-GAAP
adjusted net loss of $54.1 million, or $0.31 per share (diluted),
for the 2021 Period. 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: [pro-forma cash], 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, 2022 and 2021 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 potential
borrowings, the potential clinical value of various of its clinical
trials, the timing of commencing and completing trials, including
topline data reports, interactions with regulators and potential
for additional development programs involving Verastem Oncology’s
lead compound. 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 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 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 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)
December 31,
December 31,
2022
2021
Cash, cash equivalents, &
investments
$
87,894
$
100,256
Accounts receivable, net
31
516
Prepaid expenses and other current
assets
4,945
4,968
Property and equipment, net
92
210
Right-of-use asset, net
1,789
2,302
Restricted cash and other assets
299
410
Total assets
$
95,050
$
108,662
Current Liabilities
$
21,663
$
18,590
Long term convertible senior notes
—
249
Long term debt
24,526
—
Lease Liability, long-term
1,470
2,264
Stockholders’ equity
47,391
87,559
Total liabilities and stockholders’
equity
$
95,050
$
108,662
Verastem Oncology
Condensed Consolidated
Statements of Operations (in thousands, except per share
amounts) (unaudited)
Three months ended December
31,
Year ended December
31,
2022
2021
2022
2021
Revenue:
Sale of COPIKTRA license and related
assets revenue
$
—
$
545
$
2,596
$
1,447
Transition services revenue
—
—
—
606
Total revenue
—
545
2,596
2,053
Operating expenses:
Research and development
10,740
11,396
50,558
39,347
Selling, general and administrative
6,106
5,660
24,975
24,115
Total operating expenses
16,846
17,056
75,533
63,462
Loss from operations
(16,846)
(16,511)
(72,937)
(61,409)
Other income (expense)
(7)
—
47
—
Interest income
769
40
1,215
181
Interest expense
(724)
(10)
(2,137)
(9,972)
Net loss
$
(16,808)
$
(16,481)
$
(73,812)
$
(71,200)
Net loss per share—basic and diluted
$
(0.08)
$
(0.09)
$
(0.38)
$
(0.41)
Weighted average common shares outstanding
used in computing:
Net loss per share – basic and diluted
204,501
182,672
193,654
174,406
Verastem Oncology
Reconciliation of GAAP to
Non-GAAP Financial Information
(in thousands, except per share
amounts) (unaudited)
Three months ended December
31,
Year ended December
31,
2022
2021
2022
2021
Net loss reconciliation
Net loss (GAAP basis)
$
(16,808)
$
(16,481)
$
(73,812)
$
(71,200)
Adjust:
Stock-based compensation expense
1,287
1,574
6,047
7,711
Non-cash interest, net
(3)
44
228
9,331
Severance and other
109
—
109
40
Adjusted net loss (non-GAAP
basis)
$
(15,415)
$
(14,863)
$
(67,428)
$
(54,118)
Reconciliation of net loss per
share
Net loss per share – diluted (GAAP
basis)
$
(0.08)
$
(0.09)
$
(0.38)
$
(0.41)
Adjust per diluted share
Stock-based compensation expense
—
0.01
0.03
0.04
Non-cash interest, net
—
—
—
0.06
Severance and other
—
—
—
—
Adjusted net loss per share –
diluted (non-GAAP basis)
$
(0.08)
$
(0.08)
$
(0.35)
$
(0.31)
Weighted average common shares outstanding
used in computing net loss per share—diluted
204,501
182,672
193,654
174,406
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version on businesswire.com: https://www.businesswire.com/news/home/20230314005307/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
781-292-4205 lbuffington@verastem.com
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