Expect to Begin Rolling Submission of New Drug
Application (NDA) for Accelerated Approval to FDA for Avutometinib
and Defactinib Regimen in Recurrent Low-Grade Serous Ovarian Cancer
(LGSOC) in H1 2024; Prepare for Potential Commercial Launch in
2025
Initial Data Read-Out from RAMP 205 Trial of
Avutometinib and Defactinib Combination Plus Standard of Care
Chemotherapy in Frontline Metastatic Pancreatic Cancer Planned for
H1 2024
Data from RAMP 203 and RAMP 204 Trials in KRAS
G12C-Mutant Non-Small Cell Lung Cancer (NSCLC) Planned for
Mid-2024
Verastem Oncology (Nasdaq:VSTM), a biopharmaceutical company
committed to advancing new medicines for patients with cancer,
today outlined key 2024 strategic priorities and upcoming catalysts
to support advancement of its clinical programs in RAS
pathway-driven cancers.
“We have made significant progress in our commitment to
advancing new solutions for RAS pathway-driven cancers and look
forward to an exciting and catalyst-filled year ahead. With plans
to submit an NDA for Accelerated Approval for the combination of
avutometinib and defactinib in recurrent low-grade serous ovarian
cancer in the first half of this year, we are working rapidly to
bring forward the first potential therapy approved by the U.S. Food
and Drug Administration for patients with LGSOC in need of better
treatment options,” said Dan Paterson, President and Chief
Executive Officer of Verastem Oncology. “Further, we plan to
provide data read-outs with avutometinib and defactinib
combinations in KRAS G12C-mutant non-small cell lung cancer and
frontline metastatic pancreatic cancer, as well as supporting our
collaborator GenFleet in advancing oral KRAS G12D inhibitor
GFH375/VS-7375 into a Phase 1 clinical trial this year.”
2023 and Recent Accomplishments
- Presented interim results from the Phase 1/2 RAMP 201 trial of
avutometinib and defactinib in LGSOC, including an objective
response rate (ORR) of 45% (13/29) and disease control in 86%
(25/29) of evaluable patients. Safety and tolerability continued to
be favorable and consistent with previously reported data.
- Finalized design with the FDA and initiated confirmatory Phase
3 RAMP 301 trial to evaluate the efficacy and safety of
avutometinib and defactinib versus standard chemotherapy or
hormonal therapy for the treatment of recurrent LGSOC.
- Launched patient and healthcare professional initiatives,
including Let’s Talk About LGSOC, designed to support clinicians in
the diagnosis and management of LGSOC and to provide information,
resources and support to patients. Engaged with more than 25% of
the recurrent LGSOC patient population.
- Presented initial results from Phase 1/2 RAMP 203 trial
evaluating the efficacy and safety of avutometinib and sotorasib in
patients with KRAS G12C-mutant non-small cell lung cancer (NSCLC)
who have or have not been previously treated with a KRAS G12C
inhibitor. The confirmed ORR was 25% (3/12) across
efficacy-evaluable patients and responses observed in both KRAS
G12C inhibitor resistant (14.3%; 1/7) and naïve (40%; 2/5)
patients. Avutometinib 4.0 mg by mouth biweekly (BIW) 21/28 days
and sotorasib 960 mg by mouth once-daily (QD) 28/28 days was
selected as the recommended Phase 2 dose based on dose limiting
toxicity assessment.
- Received Fast Track designation from the FDA 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.
- Entered into a discovery and development collaboration with
GenFleet Therapeutics (“GenFleet”) to advance three oncology
discovery programs targeting RAS pathway-driven cancers. Completed
investigational new drug (IND)-enabling GLP toxicology studies for
oral KRAS G12D inhibitor GFH375/VS-7375, selected as lead
program.
Strategic Priorities and Anticipated 2024 Milestones and
Catalysts
LGSOC Program
- Begin submission of an NDA to the FDA for Accelerated Approval
for the combination of avutometinib and defactinib in recurrent
LGSOC in H1 2024; prepare for potential commercial launch in
2025.
- Present full data from Part A and Part B of RAMP 201 trial in
LGSOC at a scientific medical conference in H1 2024.
- Initiate discussions with European and Japanese regulatory
authorities for the avutometinib and defactinib regimen in LGSOC to
address patient needs outside the U.S.
KRAS G12C-Mutant NSCLC Program
- Based on stronger tumor regressions in KRAS G12C-mutant NSCLC
preclinical models when FAKi is added along with G12Ci +
avutometinib, add defactinib to the RAMP 203 trial of avutometinib
with sotorasib.
- Data updates from RAMP 203 and RAMP 204 trials planned for
mid-2024.
Frontline Metastatic Pancreatic Cancer
Program
- Present initial safety and efficacy results from RAMP 205 trial
of avutometinib and defactinib in combination with standard of care
gemcitabine and nab-paclitaxel in frontline metastatic pancreatic
cancer in H1 2024.
GenFleet Collaboration
- GenFleet expected to submit an IND for GFH375/VS-7375 in China
for patient with KRAS G12D mutations in H1 2024 and begin a Phase 1
trial for GFH375/VS-7375 in China in H2 2024.
- Companies to continue discovery/lead optimization for second
and third programs.
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
pathway inhibition. In contrast to currently available 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 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 pathway-driven tumors as part
of its (Raf And Mek Program). RAMP 301
is a 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 is
a Phase 2 registration-directed trial of avutometinib in
combination with defactinib in patients with recurrent LGSOC and
has completed enrollment in the dose optimization and expansion
phases and is enrolling for low-dose evaluation. 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. Supported by the
“Therapeutic Accelerator Award” Verastem Oncology received from
PanCAN, the Company 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 the expected outcome and benefits of the
collaboration with GenFleet, the potential clinical value of
various of its 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. 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
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, 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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20240124822477/en/
Investors: Ryan Porter Argot Partners +1 212-600-1902
ryan.porter@argotpartners.com
Media: Verastem Oncology media@verastem.com
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