Randomized Phase 3 Trial (RAMP 301) Will
Evaluate the Combination of Avutometinib and Defactinib vs Standard
of Care Treatment; Study to Commence in 2H 2023
Recently Published Data from Part A of RAMP 201
Trial Show an Objective Response Rate of 45%, and Manageable Safety
and Tolerability Profile with the Combination
Company Intends to File for Accelerated FDA
Approval for the Combination Based on Mature Data from RAMP 201 and
the FRAME Trial
Verastem Oncology (Nasdaq: VSTM) (the “Company”), a
biopharmaceutical company committed to advancing new medicines for
patients with cancer, announced today that it has finalized with
the U.S. Food and Drug Administration (FDA) the design of its
confirmatory Phase 3 trial to evaluate the combination of
avutometinib and defactinib for the treatment of recurrent
low-grade serous ovarian cancer (LGSOC). RAMP 301, a randomized
global confirmatory trial, will evaluate the efficacy and safety of
avutometinib and defactinib versus standard of care (SOC)
chemotherapy and hormonal therapy in patients with recurrent LGSOC.
RAMP 301 is expected to begin enrollment in the second half of this
year.
RAMP 301 is the follow-up confirmatory study for full approval
in recurrent LGSOC. The Company intends to file for Accelerated FDA
Approval for the combination of avutometinib and defactinib based
on mature data from the Company’s Phase 2 registration-directed
RAMP 201, together with the results of the investigator-initiated
FRAME trial. The Company recently reported results of Part A of
RAMP 201 including confirmed objective response rates (ORR) by
blinded independent central review of 45% (13/29; 95% CI: 26%,64%)
with a tolerable safety profile.
“We are pleased to partner with GOG and ENGOT and announce the
final study design for RAMP 301, another important milestone in
advancing our avutometinib and defactinib program and bringing us
closer to addressing the unmet needs of patients living with
LGSOC,” said Brian Stuglik, Chief Executive Officer, Verastem
Oncology. “This trial builds on the encouraging results of Part A
of the RAMP 201 trial and our breakthrough therapy designation,
after one or more prior lines of therapy, and we are committed to
bringing the first FDA-approved therapy for LGSOC to patients as
quickly as possible.”
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 chemotherapy (pegylated liposomal doxorubicin,
paclitaxel, topotecan) or hormone therapy (letrozole,
anastrozole). Selection of the SOC regimen will be based on
the preference of the investigator. The primary endpoint is
progression free survival (PFS) by Blinded Independent Central
Review. Secondary endpoints include overall response rates,
duration of response, disease control rate, safety and
tolerability, patient reported outcomes and overall survival. The
RAMP 301 trial will be led globally and in the U.S. by Rachel
Grisham, MD, Section Head, Ovarian Cancer and Director, Gynecologic
Medical Oncology at Memorial Sloan Kettering Cancer
Center in Westchester, NY. Susana Banerjee, MBBS, MA PhD,
FRCP, global and lead investigator of the RAMP 201 study,
Consultant Medical Oncologist at The Royal Marsden NHS
Foundation Trust and Team Leader in Women’s Cancers
at The Institute of Cancer Research, London, will be
leading the RAMP 301 trial in Europe.
Financial Update
As of March 31, 2023, Verastem Oncology had cash and short-term
investments of $111.2 million. With the net proceeds from the
underwritten public offering completed in June 2023 of
approximately $91.5 million after deducting estimated underwriting
discounts and commissions and estimated offering expenses, the
Company has pro-forma cash and short-term investments as of March
31, 2023 of approximately $202.7 million.
Recent historical operating expenses have ranged between $16.0M
-- $20.0M per quarter, which the Company does not anticipate will
change significantly in the near term as the RAMP 301 trial
commences.
Dr. Banerjee and Dr. Grisham have consulting relationships with
Verastem Oncology.
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 pathway-driven tumors as part
of its (Raf And Mek Program). RAMP 201
is a registration-directed trial of avutometinib alone and 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.
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.
About The GOG Foundation, Inc. (www.gog.org)
The GOG Foundation, Inc. is a not-for-profit organization with
the purpose of promoting excellence in the quality and integrity of
clinical and translational scientific research in the field of
gynecologic malignancies. The GOG Foundation is committed to
maintaining the highest standards in clinical trials development,
execution, analysis, and distribution of results. The GOG
Foundation is the only clinical trialist group in the United States
that focuses its research on patients with pelvic malignancies,
such as cancer of the ovary (including surface peritoneal
malignancies), uterus (including endometrium, soft tissue sarcoma,
and gestational trophoblastic neoplasia), cervix, and vulva. The
GOG Foundation is multi-disciplinary in its approach to clinical
trials, and includes gynecologic oncologists, medical oncologists,
pathologists, radiation oncologists, oncology nurses,
biostatisticians (including those with expertise in
bioinformatics), basic scientists, quality of life experts, data
managers, and administrative personnel.
About the GOG Partners Program
Supported by industry, GOG Partners program is structured to
work directly with pharmaceutical organizations and operate
clinical trials outside the National Cancer Institute (NCI)
framework. The GOG Partners program promotes the mission of the GOG
Foundation dedicated to transforming the standard of care in
Gynecologic Oncology. By providing an alternative venue for patient
accrual and site infrastructure support, GOG Partners has helped
provide additional trials and opportunities for patients outside
the national gynecologic clinical trials network.
About ENGOT (www.engot.esgo.org)
The European Network for Gynaecological Oncological Trial
(ENGOT) groups is a research network of the European Society of
Gynaecological Oncology and was founded in Berlin in October 2007.
Currently, ENGOT consists of 21 trial groups from 31 European
countries that perform cooperative clinical trials. ENGOT’s
ultimate goal is to bring the best treatment to gynecological
cancer patients through the best science and enabling every patient
in every European country to access a clinical trial.
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 potential clinical value of various of
its clinical trials, the timing of commencing and completing trials
and the Company’s anticipated operating expenses. 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, LUMAKRASTM 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 Bio, Inc. will achieve the
milestones that result in payments to us under our asset purchase
agreement with Secura Bio, Inc.; 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 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.
Dr. Banerjee and Dr. Grisham have consulting relationships with
Verastem Oncology.
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version on businesswire.com: https://www.businesswire.com/news/home/20230705791373/en/
Investors:
Dan Calkins Investor Relations +1 781-469-1694
dcalkins@verastem.com
Argot Partners 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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