Plan to announce topline RAMP 201 data with the
start of planned rolling NDA submission for avutometinib and
defactinib combination in recurrent low-grade serous ovarian cancer
in Q2 2024
FDA Fast Track Designation granted for
avutometinib in combination with adagrasib for the treatment of
KRAS G12C-mutated metastatic non-small cell lung cancer
FDA Fast Track Designation granted for
avutometinib plus defactinib in combination with sotorasib for the
treatment of KRAS G12C-mutated metastatic non-small cell lung
cancer
Initial safety and efficacy results from the
RAMP 205 trial of avutometinib and defactinib in combination with
current standard of care in first-line metastatic pancreatic cancer
to be presented at the 2024 ASCO Annual Meeting
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company
committed to advancing new medicines for patients with cancer,
today announced business updates and reported financial results for
the first quarter ended March 31, 2024.
“In the first quarter of 2024, we received FDA Orphan Drug
Designation for avutometinib and defactinib combination in
recurrent low-grade serous ovarian cancer, which recognizes this
rare cancer as different and distinct from other forms of ovarian
cancer and reinforces the need for new treatment options,” said Dan
Paterson, president and chief executive officer of Verastem
Oncology. “We look forward to starting our planned rolling NDA
submission and sharing topline data for avutometinib and defactinib
combination in recurrent low-grade serous ovarian cancer. We also
plan to announce initial data from the RAMP 205 trial in first-line
metastatic pancreatic cancer at ASCO and plan to provide updates
across our other clinical programs in the second half of 2024.”
First Quarter 2024 and Recent Updates
Avutometinib and Defactinib Combination in Low-Grade Serous
Ovarian Cancer (LGSOC)
- Enrollment and site activations are underway in the U.S.,
Australia, and the UK, for the international confirmatory Phase 3
RAMP 301 trial evaluating the avutometinib and defactinib
combination versus standard of care chemotherapy or hormonal
therapy for the treatment of recurrent LGSOC.
- Granted Orphan Drug Designation from the U.S. Food and Drug
Administration (FDA) for avutometinib alone or in combination with
defactinib for the treatment of all patients with recurrent LGSOC,
in March 2024.
- Multiple abstracts were selected for oral and poster
presentations at the Society of Gynecologic Oncology (SGO) 2024
Annual Meeting on Women’s Cancer on March 16-18 in San Diego. These
presentations included a late-breaking oral presentation on a
planned subgroup analysis of Part A of the Phase 2 RAMP 201 trial
of avutometinib and defactinib combination of heavily pretreated
patients with LGSOC and a plenary oral presentation of preclinical
efficacy data of avutometinib in combination with a focal adhesion
kinase (FAK) inhibitor in recurrent LGSOC as well as a
trials-in-progress poster about the Phase 3 RAMP 301 trial.
- Plan to announce updated topline data from the RAMP 201 trial
in LGSOC to coincide with the start of our planned rolling New Drug
Application (NDA) submission for Accelerated Approval for the
avutometinib and defactinib combination in recurrent LGSOC in Q2
2024.
- Preparations for a potential U.S. commercial launch in 2025 are
ongoing and plans to initiate discussions with European and
Japanese regulatory authorities to address patient needs outside
the U.S. continue to advance.
Avutometinib in Combination with KRAS G12C Inhibitors in
Non-Small Cell Lung Cancer (NSCLC)
- Verastem Oncology announced today it has received Fast Track
Designation from the FDA for avutometinib in combination with
Mirati’s (BMS) G12C inhibitor, KRAZATI™ (adagrasib) for the
treatment of patients with KRAS G12C-mutated metastatic NSCLC who
have received at least one prior systemic therapy and have not been
previously treated with a KRAS G12C inhibitor, in April 2024.
- Verastem Oncology announced today it has received Fast Track
Designation from the FDA for the combination of avutometinib plus
defactinib with Amgen’s G12C inhibitor, LUMAKRAS™ (sotorasib) for
the treatment of patients with KRAS G12C-mutated metastatic NSCLC
who have received at least one prior systemic therapy, in April
2024.
- The FDA granted Fast Track Designation for avutometinib in
combination with Amgen’s G12C inhibitor, LUMAKRAS™ (sotorasib), for
the treatment of patients with KRAS G12C-mutant metastatic NSCLC
who have received at least one prior systemic therapy and have not
been previously treated with a KRAS G12C inhibitor, in January
2024.
- Data updates from patients with KRAS G12C-mutant NSCLC in the
Phase 1/2 RAMP 203 trial evaluating avutometinib plus defactinib
and sotorasib are planned for H2 2024.
- Data from patients with KRAS G12C-mutant NSCLC in the Phase 1/2
RAMP 204 trial evaluating avutometinib and adagrasib are planned
for H2 2024.
Avutometinib and Defactinib Combination in First-Line
Metastatic Pancreatic Cancer
- Verastem Oncology today announced the acceptance of an abstract
that will include initial safety and efficacy results from the RAMP
205 trial of avutometinib and defactinib in combination with
current standard of care gemcitabine and nab-paclitaxel in
first-line metastatic pancreatic cancer at the upcoming American
Society of Clinical Oncology (ASCO) Annual Meeting.
GFH375 (VS-7375): Oral KRAS G12D (ON/OFF) Inhibitor
- GenFleet Therapeutics investigational new drug (IND)
application for GFH375 (VS-7375) was submitted in China and
accepted for review. Expect to begin a Phase 1 trial in China in H2
2024.
- Discovery/lead optimization continues for second and third
programs with GenFleet collaboration.
Upcoming Presentations
Verastem Oncology today announced the acceptance of an abstract
for poster presentation at the ASCO Annual Meeting being held from
May 31 to June 4, 2024, in Chicago, IL.
- Title: Avutometinib/defactinib and
gemcitabine/nab-paclitaxel combination in first-line metastatic
pancreatic ductal adenocarcinoma: Initial safety and efficacy of
phase 1b/2 study (RAMP 205).
- Abstract Number: 4140
- Date/Time: Saturday, June 1, 2024, 1:30 to 4:30 pm
CDT
Corporate Updates
- Strengthened the executive leadership team with the appointment
of John Hayslip, M.D., to chief medical officer in April 2024.
First Quarter 2024 Financial Results
Verastem Oncology ended the first quarter of 2024 with cash,
cash equivalents and investments of $110.1 million.
Total operating expenses for the three months ended March 31,
2024 (the “2024 Quarter”) were $28.1 million, compared to $19.3
million for the three months ended March 31, 2023 (the “2023
Quarter”).
Research & development expenses for the 2024 Quarter were
$17.7 million, compared to $12.0 million for the 2023 Quarter. The
increase of $5.7 million, or 47.5%, was primarily related to
increased contract research organization costs, increased
investigator fees and increased personnel costs, including non-cash
stock compensation.
Selling, general & administrative expenses for the 2024
Quarter were $10.4 million, compared to $7.3 million for the 2023
Quarter. The increase of $3.1 million, or 42.5%, was primarily
related to additional costs in anticipation of a potential launch
of avutometinib and defactinib in LGSOC, increased personnel costs,
including non-cash stock compensation, and increased consulting and
professional fees.
Net loss for the 2024 Quarter was $33.9 million, or $1.26 per
share (basic and diluted), compared to $15.7 million, or $0.94 per
share (basic and diluted, each as adjusted for the Company’s
reverse stock split) for the 2023 Quarter.
For the 2024 Quarter, non-GAAP adjusted net loss was $26.2
million, or $0.98 per share (diluted) compared to non-GAAP adjusted
net loss of $17.8 million, or $1.07 per share (diluted, as adjusted
for the Company’s reverse stock split), for the 2023 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 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, 2024 and 2023 are included in the tables accompanying
this press release after the unaudited condensed consolidated
financial statements.
About the Avutometinib and Defactinib Combination
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/MAPK
pathway inhibition. In contrast to currently available MEK-only
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
MEK-only inhibitors. The U.S. Food and Drug Administration (FDA)
granted Breakthrough Therapy Designation of the investigational
combination of avutometinib and defactinib, a selective 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. Avutometinib alone or in combination with defactinib
was also granted Orphan Drug Designation by the FDA for the
treatment of LGSOC.
Verastem Oncology is currently conducting clinical trials with
avutometinib in RAS/MAPK driven tumors as part of its Raf
And Mek Program or RAMP. RAMP 301
(NCT06072781) is an international Phase 3 confirmatory trial
evaluating the combination of avutometinib and defactinib versus
standard chemotherapy or hormonal therapy for the treatment of
recurrent LGSOC. RAMP 201 (NCT04625270) is a Phase 2
registration-directed trial of avutometinib in combination with
defactinib in patients with recurrent LGSOC and enrollment has been
completed in each of the dose optimization and expansion phases and
the low-dose evaluation.
Verastem Oncology has established clinical collaborations with
Amgen and Mirati to evaluate LUMAKRAS™ (sotorasib) in combination
with avutometinib and defactinib and KRAZATI™ (adagrasib) in
combination with avutometinib in KRAS G12C mutant NSCLC as part of
the RAMP 203 (NCT05074810) and RAMP 204 (NCT05375994) trials,
respectively. The RAMP 205 (NCT05669482), a Phase 1b/2 clinical
trial evaluating avutometinib and defactinib with
gemcitabine/nab-paclitaxel in patients with front-line metastatic
pancreatic cancer, is supported by the PanCAN Therapeutic
Accelerator Award.
About GFH375 (VS-7375)
GFH375 (VS-7375) is a potential best-in-class, potent and
selective oral KRAS G12D (ON/OFF) inhibitor, identified as the lead
program from the Verastem Oncology discovery and development
collaboration with GenFleet Therapeutics. Upon approval of the
investigational new drug (IND) application in China (which is
currently under review), GenFleet is expected to initiate a Phase 1
trial in China in the second half of 2024. The collaboration
includes three discovery programs, the first being the KRAS G12D
inhibitor, and will provide Verastem Oncology with exclusive
options to obtain licenses to each of the three compounds in the
collaboration after successful completion of pre-determined
milestones in Phase 1 trials. The licenses would give Verastem
Oncology development and commercialization rights outside of the
GenFleet territories of mainland China, Hong Kong, Macau, and
Taiwan.
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a late-stage development
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 RAS/MAPK-driven
cancers, specifically novel small molecule drugs that inhibit
critical signaling pathways in cancer that promote cancer cell
survival and tumor growth, including RAF/MEK inhibition and FAK
inhibition. For more information, please visit www.verastem.com and
follow us on LinkedIn.
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 timing of the planned rolling
New Drug Application (NDA) submission for the avutometinib and
defactinib combination in low-grade serous ovarian cancer, the
outcome and benefits of the collaboration with GenFleet, including
the approval of the IND in China, the potential clinical value of
various of the Company’s clinical trials, the timing of commencing
and completing trials, including topline data reports, interactions
with regulators, the potential for and timing of commercialization
of product candidates 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. Forward-looking statements are not
guarantees of future performance and are 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
uncertainties inherent in research and development, such as
negative or unexpected results of clinical trials, the occurrence
or timing of applications for our product candidates that may be
filed with regulatory authorities in any jurisdictions; whether and
when regulatory authorities in any jurisdictions may approve any
such applications that may be filed for our product candidates,
and, if approved, whether our product candidates will be
commercially successful in such jurisdictions; 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 cause adverse safety events and/or unexpected
concerns may arise from additional data or analysis, or result in
unmanageable safety profiles as compared to their levels of
efficacy; that our product candidates may experience manufacturing
or supply interruptions or failures; 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 face substantial
competition, which may result in others developing or
commercializing products before or more successfully than we do
which could result in reduced market share or market potential for
our product candidates; 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 may not have sufficient cash to fund
our contemplated operations; that we may not attract and retain
high quality personnel; that we or Chugai Pharmaceutical Co., Ltd.
will fail to fully perform under the avutometinib license
agreement; that our target market for our product candidates might
be smaller than we are presently estimating; that Secura Bio, Inc.
will fail to fully perform under the asset purchase agreement with
Secura Bio, Inc., including in relation to milestone payments; that
we will not see a return on investment on the payments we have and
may continue to make pursuant to the collaboration and option
agreement with GenFleet or that GenFleet will fail to fully perform
under the agreement; 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 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, 2023 as filed with the Securities
and Exchange Commission (SEC) on March 14, 2024 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 Oncology Condensed
Consolidated Balance Sheets (in thousands) (unaudited)
March 31,
2024
December 31,
2023
Cash, cash equivalents, &
investments
$
110,125
$
137,129
Grant receivable
226
—
Prepaid expenses and other current
assets
7,323
6,553
Property and equipment, net
52
37
Right-of-use asset, net
997
1,171
Restricted cash and other assets
4,816
4,828
Total assets
$
123,539
$
149,718
Current Liabilities
$
26,725
$
26,380
Long term debt
40,123
40,086
Lease liability, long-term
270
530
Preferred stock tranche liability
10,200
4,189
Convertible preferred stock
21,159
21,159
Stockholders’ equity
25,062
57,374
Total liabilities, convertible
preferred stock and stockholders’ equity
$
123,539
$
149,718
Verastem Oncology Condensed
Consolidated Statements of Operations (in thousands, except per
share amounts) (unaudited)
Three months ended March
31,
2024
2023
Operating expenses:
Research and development
$
17,707
$
12,015
Selling, general and administrative
10,352
7,329
Total operating expenses
28,059
19,344
Loss from operations
(28,059
)
(19,344
)
Other income (expense)
(30
)
(7
)
Interest income
1,367
976
Interest expense
(1,130
)
(769
)
Change in fair value of preferred stock
tranche liability
(6,011
)
3,430
Net loss
$
(33,863
)
$
(15,714
)
Net loss per share—basic and diluted
$
(1.26
)
$
(0.94)(1
)
Weighted average common shares outstanding
used in computing:
Net loss per share – basic and diluted
$
26,832
$
16,723(1
)
(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 March
31,
2024
2023
Net loss reconciliation
Net loss (GAAP basis)
$
(33,863
)
$
(15,714
)
Adjust:
Stock-based compensation expense
1,483
1,313
Non-cash interest, net
(419
)
(36
)
Change in fair value of preferred stock
tranche liability
6,011
(3,430
)
Severance and other
553
38
Adjusted net loss (non-GAAP
basis)
$
(26,235
)
$
(17,829
)
Reconciliation of net loss per
share
Net loss per share – diluted (GAAP
Basis)
$
(1.26
)
$
(0.94)(1
)
Adjust per diluted share:
Stock-based compensation expense
0.06
0.08(1
)
Non-cash interest, net
(0.02
)
—
Change in fair value of preferred stock
tranche liability
0.22
(0.21)(1
)
Severance and other
0.02
—
Adjusted net loss per share –
diluted
(non-GAAP basis)
$
(0.98
)
$
(1.07)(1
)
Weighted average common shares outstanding
used in computing net loss per share—diluted
$
26,832
$
16,723(1
)
(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/20240509453333/en/
For Investor and Media Inquiries: Julissa Viana Vice
President, Corporate Communications and Investor Relations
investors@verastem.com or media@verastem.com
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