Expect to report updated data from the ongoing
RAMP 205 trial in first-line metastatic pancreatic cancer in Q1
2025
Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company
committed to advancing new medicines for patients, today announced
that the U.S. Food and Drug Administration (FDA) has granted Orphan
Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, in
combination with defactinib, a selective FAK inhibitor, for the
treatment of pancreatic cancer.
“At the ASCO 2024 Annual Meeting, we reported positive initial
interim results from the ongoing RAMP 205 trial evaluating
avutometinib and defactinib in combination with standard of care
chemotherapy in first-line metastatic pancreatic cancer,” said Dan
Paterson, president and chief executive officer of Verastem
Oncology. “The FDA Orphan Drug Designation for the combination of
avutometinib and defactinib for the treatment of pancreatic cancer
recognizes the substantial unmet treatment need for patients with
pancreatic cancer. We believe avutometinib and defactinib in
combination with standard of care has an opportunity to provide a
different approach in treating this challenging cancer. We look
forward to reporting updated data from across dose cohorts in the
ongoing RAMP 205 trial in the first quarter of 2025.”
At the American Society of Clinical Oncology (ASCO) Annual
Meeting in June 2024, Verastem presented initial interim safety and
efficacy results from the ongoing RAMP 205 trial of avutometinib
and defactinib in combination with current standard of care
gemcitabine and nab-paclitaxel in first-line metastatic pancreatic
cancer. As of the data cutoff of May 14, 2024, 41 patients had been
treated in one of four dose cohort regimens and only patients in
dose cohort 1 had a minimum follow up of six months. In the dose
level 1 cohort, 83% (5/6) of patients achieved a confirmed partial
response with more than six months of follow up at the time of data
cutoff. One dose-limiting toxicity (DLT) was observed in the dose
level 1 cohort, and the dose level was subsequently cleared after
additional patients were enrolled. Of the 26 patients in all
cohorts who have had the opportunity to have their first scan while
on treatment, 21 have experienced a reduction of the change in
target lesion sum of diameters. Read the press release here.
FDA Orphan Drug Designation is granted to certain
investigational treatments for diseases or conditions that affect
fewer than 200,000 people in the United States. Orphan Drug
Designation provides benefits to drug developers, including tax
credits for qualified clinical trials, exemptions from certain FDA
user fees and the potential for seven years of market exclusivity
upon approval.
About Metastatic Pancreatic Cancer
Pancreatic cancer is the third leading cancer in the U.S. and
seventh leading cause of cancer-associated mortality worldwide.
Metastatic pancreatic cancer is defined as stage IV cancer, where
the cancer spreads to other organs. In the U.S., over 30,000
patients are diagnosed with metastatic pancreatic cancer each year,
for which the five-year survival rate is 3%. Globally, over 240,000
patients are diagnosed with metastatic pancreatic cancer each year.
More than 90% of pancreatic cancers have a KRAS mutation. The
standard of care consists of surgery, chemotherapy, radiation or a
combination of these approaches.
About RAMP 205 Phase 1/2 Study
RAMP 205 is a multicenter, open-label, single arm Phase 1b/2a
study designed to evaluate the safety, tolerability and efficacy of
avutometinib and defactinib in combination with standard of care
chemotherapy (gemcitabine and Nab-paclitaxel) in patients with
previously untreated metastatic pancreatic ductal adenocarcinoma.
Part A of the study will evaluate different dose and schedule
combinations to determine the recommended Phase 2 dose for
expansion into Part B. RAMP 205 is supported by a PanCAN
Therapeutic Accelerator Award.
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 has initiated a rolling NDA
submission for avutometinib and defactinib combination in adults
with recurrent LGSOC and expects to complete its NDA submission in
the second half of 2024 with a potential FDA decision in the first
half of 2025.
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 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
This press release includes forward-looking statements about,
among other things, Verastem Oncology’s programs and product
candidates, strategy, future plans and prospects, including
statements related to the potential clinical value of various of
the Company’s clinical trials, including the RAMP 205 trial, the
timing of commencing and completing trials, including the RAMP 205
trial 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.
Forward-looking statements are not guarantees of future
performance and are subject to risks and uncertainties that could
cause our actual results could differ materially from those
expressed or implied in the forward-looking statements we make.
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 the market
opportunities of our drug candidates are based on internal and
third-party estimates which may prove to be incorrect; 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 or
that the FDA may require the Company to enroll additional patients
in the Company’s ongoing RAMP-301 confirmatory Phase 3 clinical
trial prior to submitting or the FDA taking Action on our NDA
seeking accelerated approval; risks associated with preliminary and
interim data, which may not be representative of more mature data,
including with respect to interim duration of therapy data; 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 we may be unable to successfully validate,
develop and obtain regulatory approval for companion diagnostic
tests for our product candidates that require or would commercially
benefit from such tests, or experience significant delays in doing
so; that the mature RAMP 201 data and associated discussions with
the FDA may not support the scope of our rolling NDA submission for
the avutometinib and defactinib combination in LGSOC, including
with respect to KRAS wild type LGSOC; 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 or our decisions regarding execution
of such commercialization; that we may not have sufficient cash to
fund our contemplated operations, including certain of our product
development programs; 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 the total addressable and target markets for our product
candidates might be smaller than we are presently estimating; that
we or Secura Bio, Inc. (“Secura”) will fail to fully perform under
the asset purchase agreement with Secura, 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 Therapeutics
(Shanghai), Inc. (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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240729473398/en/
For Investor and Media Inquiries: Julissa Viana Vice
President, Corporate Communications and Investor Relations
investors@verastem.com or media@verastem.com
Verastem (NASDAQ:VSTM)
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