Verastem Will Receive $70 Million Up-Front with
Total Deal Value Up to $311 Million, Plus Double-Digit Sales
Royalties
Upon Closing, Verastem’s Current Programs Will
Be Funded Until At Least 2024 to Develop VS-6766 and Defactinib in
Low-Grade Serous Ovarian Cancer and KRAS Mutant Non-Small Cell Lung
Cancer
Phase 2 Registration-Directed Trials Expected
to Commence by Year End 2020 in Both Low-Grade Serous Ovarian
Cancer and KRAS Mutant Non-Small Cell Lung Cancer
Enrollment in Ongoing Investigator-Initiated
Phase 1/2 FRAME Study of VS-6766 and Defactinib Now Expanding to
Include Pancreatic, KRAS Mutant Endometrial and KRAS-G12V Non-Small
Cell Lung Cancer Cohorts
Verastem, Inc. (Nasdaq:VSTM) (also known as Verastem Oncology),
a biopharmaceutical company committed to advancing new medicines
for patients battling cancer, today announced that it has entered
into a definitive agreement to sell its global commercial and
development rights to COPIKTRA (duvelisib), its marketed oral
inhibitor of phosphoinositide 3-kinase (PI3K), and the first
FDA-approved dual inhibitor of PI3K-delta and PI3K-gamma, to Secura
Bio, Inc., an integrated biopharmaceutical company dedicated to the
worldwide commercialization of significant oncology therapies.
Verastem’s sale of COPIKTRA follows the Company’s previously
announced strategic direction to focus on maximizing the broad
potential of its RAF/MEK inhibitor (VS-6766) and FAK inhibitor
(defactinib) program in KRAS mutant (KRASmt) solid tumors. Upon
closing of the transaction with Secura Bio, Verastem will be
dedicated to the development of this program and to deliver on
clinical and regulatory milestones for the first potential
indications in low-grade serous ovarian cancer (LGSOC) and KRASmt
non-small cell lung cancer (NSCLC). Both LGSOC and KRASmt NSCLC are
areas of high unmet patient need as there are no approved
treatments and existing therapies have low response rates.
“By focusing our expertise and efforts on rapidly advancing the
RAF/MEK/FAK development program, we believe we will be providing
the best path forward for patients, customers, our shareholders and
our company. These strategic decisions will enable us to best
deliver on our mission to advance new medicines on behalf of cancer
patients,” said Brian Stuglik, Chief Executive Officer of Verastem
Oncology. “The agreement with Secura Bio will ensure COPIKTRA
continues to help more patients, leveraging the established
commercial structure, support of ongoing clinical study and
potential expansion into new indications.”
Terms of the Definitive Sale Agreement
Verastem will receive an up-front payment of $70 million upon
the closing of the transaction and is eligible to receive up to a
total deal value of $311 million if certain regulatory and
sales-based milestones are successfully met by Secura Bio and
COPIKTRA’s other rest-of-world partners, including:
- A total of $45 million from two separate milestone payments for
U.S. Food and Drug Administration (FDA) and European Medicines
Agency approvals of COPIKTRA with label indicated for peripheral
T-cell lymphoma
- A total of $50 million for cumulative worldwide net sales of
COPIKTRA beginning at $100 million of cumulative net sales
- Verastem will receive low double-digit royalties on net sales
over $100 million in U.S., Europe and the United Kingdom
- Verastem will also receive 50% of licensing milestones (up to
$146 million) and royalties outside of U.S., Europe and the United
Kingdom
In exchange, Secura Bio will receive an exclusive worldwide
license for the research, development, commercialization and
manufacture of COPIKTRA in all oncology indications. Secura Bio
will assume all operational and financial responsibility for
activities that were previously part of Verastem’s duvelisib
program, including commercialization efforts in the United States
and Europe, ongoing clinical trials, Verastem’s partnerships with
Yakult, CSPC and Sanofi and existing royalty obligations. Secura
Bio and Verastem are also in discussions related to the transfer of
Verastem’s field sales and medical professionals.
The transaction with Secura Bio is subject to customary closing
conditions and is expected to close in the third quarter of
2020.*
VS-6766 and Defactinib Program Progress and
Registration-Directed Trials
Verastem announced today that the company met with the FDA in
July 2020 to discuss the registration-directed study design for the
VS-6766/defactinib combination in patients with LGSOC. The FDA was
supportive of the Company’s development strategy and adaptive
design for LGSOC.
Verastem’s NSCLC study will also be an adaptive design with a
focus on patients with KRAS-G12V mutant tumors. Verastem intends to
seek input from the FDA after completing the initial cohort of the
lung cancer study. Verastem expects to commence
registration-directed clinical trials for potential accelerated
approval in LGSOC and KRASmt NSCLC by the end of 2020.
Verastem is continuing its clinical collaboration with the Drug
Development Unit at ICR/Royal Marsden Hospital. The ongoing
investigator-initiated Phase 1/2 FRAME study evaluating the
combination of VS-6766 with defactinib in LGSOC, KRASmt NSCLC and
colorectal cancer (CRC) has resumed normal accrual and reporting
rates following the global lockdown resulting from the COVID-19
pandemic. The FRAME study is now expanding to include new cohorts
in pancreatic cancer, KRASmt endometrial cancer and KRAS-G12V
NSCLC. Verastem expects that additional data from the LGSOC cohort
of the FRAME study will be made available in September, including
presentation at the 2nd Annual RAS-Targeted Drug Development
Conference. The Company also expects that additional data from the
NSCLC cohort of the FRAME study will be submitted to the
International Association for the Study of Lung Cancer (IASLC)
World Lung Cancer Conference, taking place in January 2021.
The Company has also begun preclinical combination studies
investigating VS-6766 and defactinib in combination with KRAS-G12C
inhibitors and initial data will be presented at the 2nd Annual
RAS-Targeted Drug Development Conference. Based on the positive
preclinical data presented at the AACR 2020 Virtual Annual Meeting
II, Verastem plans to support a Phase 2 investigator-initiated
study evaluating the combination of VS-6766 and defactinib in uveal
melanoma, which is expected to begin in late 2020.
Corporate and Financial Overview
With the sale of COPIKTRA, Verastem will become a focused
development company with reduced annual expenses of approximately
$50 million. The company is in a position of financial strength
with a cash runway expected to fund the clinical and regulatory
milestones and development of VS-6766 and defactinib in LGSOC and
KRASmt NSCLC until at least 2024.
About VS-6766
VS-6766 (formerly known as CH5126766, CKI27 and RO5126766) is a
unique inhibitor of the RAF/MEK signaling pathway. In contrast to
other MEK inhibitors in development, VS-6766 blocks both MEK kinase
activity and the ability of RAF to phosphorylate MEK. This unique
mechanism allows VS-6766 to block MEK signaling without the
compensatory activation of MEK that appears to limit the efficacy
of other inhibitors.
About Defactinib
Defactinib (VS-6063) is an oral small molecule inhibitor of FAK
and PYK2 that is currently being evaluated as a potential
combination therapy for various solid tumors. The Company has
received Orphan Drug designation for defactinib in ovarian cancer
and mesothelioma in the US, EU and Australia. Preclinical research
by Verastem Oncology scientists and collaborators at world-renowned
research institutions has described the effect of FAK inhibition to
enhance immune response by decreasing immuno-suppressive cells,
increasing cytotoxic T cells, and reducing stromal density, which
allows tumor-killing immune cells to enter the tumor.1,2
About the VS-6766/Defactinib Combination
RAS mutant tumors are present in 30% of all human cancers and
have historically presented a difficult treatment challenge and are
often associated with significantly worse prognosis. Challenges
associated with identifying new treatment options for these types
of cancers include resistance to single agents, identifying
tolerable combination regimens with MEK inhibitors and new RAS
inhibitors in development addressing only a minority of all RAS
mutated cancers.
The combination of VS-6766 and defactinib has been found to be
clinically active in KRASmt. In an ongoing investigator-initiated
Phase I/2 FRAME study, the combination of VS-6766 and defactinib is
being evaluated in patients with LGSOC, KRASmt NSCLC and colorectal
cancer (CRC). Preliminary data from this study presented at the
American Association for Cancer Research (AACR) 2020 Virtual Annual
Meeting I demonstrated a 67% overall response rate and long
duration of therapy among patients with KRASmt LGSOC. Based on an
observation of higher response rates seen in patients with
KRAS-G12V mutations in the study, Verastem will also be further
exploring the role of VS-6766 and defactinib in KRAS-G12V NSCLC.
The FRAME study is expanding in August 2020 to include new cohorts
in pancreatic, KRASmt endometrial and KRAS-G12V NSCLC.
About COPIKTRA® (duvelisib)
COPIKTRA is an oral inhibitor of phosphoinositide 3-kinase
(PI3K), and the first approved dual inhibitor of PI3K-delta and
PI3K-gamma, two enzymes known to help support the growth and
survival of malignant B-cells. PI3K signaling may lead to the
proliferation of malignant B-cells and is thought to play a role in
the formation and maintenance of the supportive tumor
microenvironment.3,4,5 COPIKTRA is indicated for the treatment of
adult patients with relapsed or refractory chronic lymphocytic
leukemia/small lymphocytic lymphoma (CLL/SLL) after at least two
prior therapies and relapsed or refractory follicular lymphoma (FL)
after at least two prior systemic therapies. COPIKTRA is also being
developed by Verastem Oncology for the treatment of peripheral
T-cell lymphoma (PTCL), for which it has received Fast Track status
and Orphan Drug Designation, and is being investigated in
combination with other agents through investigator-sponsored
studies.6 For more information on COPIKTRA, please visit
www.COPIKTRA.com. Information about duvelisib clinical trials can
be found on www.clinicaltrials.gov.
*MTS Health Partners, L.P and Ropes & Gray acted as advisors
to Verastem Oncology on this transaction.
About Verastem Oncology
Verastem Oncology (Nasdaq: VSTM) is a commercial
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
phosphoinositide 3-kinase (PI3K), focal adhesion kinase (FAK) and
RAF/MEK inhibition.
Our first FDA approved product is available for the treatment of
patients with certain types of indolent non-Hodgkin’s lymphoma
(iNHL).
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 sale of COPIKTRA, the Company’s
future funding requirements and the potential clinical value of the
RAF/MEK/FAK combination. 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 satisfaction of
closing conditions with respect to the sale of the COPIKTRA assets
to Secura Bio; the ability of Secura Bio to achieve the clinical
and sales milestones necessary to result in additional
consideration payable to Verastem; the inherent uncertainty in
forecasting expected funding needs of the Company in advancing its
product candidates; the success in the development and potential
commercialization of our product candidates, including defactinib
in combination with VS-6766; 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 labeling and other matters that could affect
the 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; that we may not have sufficient cash to fund our
contemplated operations; that we may be unable to make additional
draws under our debt facility or obtain adequate financing in the
future through product licensing, co-promotional arrangements,
public or private equity, debt financing or otherwise; that we will
be unable to execute on our partnering strategies for defactinib in
combination with VS-6766; 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, 2019 as filed with the Securities
and Exchange Commission (SEC) on March 11, 2020 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.
1 Gerber D. et al. Phase 2 study of the focal adhesion kinase
inhibitor defactinib (VS-6063) in previously treated advanced KRAS
mutant non-small cell lung cancer. Lung Cancer 2020: 139:60-67.
2 Chénard-Poirier, M. et al. Results from the biomarker-driven
basket trial of RO5126766 (CH5127566), a potent RAF/MEK inhibitor,
in RAS- or RAF-mutated malignancies including multiple myeloma.
Journal of Clinical Oncology 2017: 35.
10.1200/JCO.2017.35.15_suppl.2506.
3 Winkler D.G., Faia K.L., DiNitto J.P. et al. PI3K-delta and
PI3K-gamma inhibition by IPI-145 abrogates immune responses and
suppresses activity in autoimmune and inflammatory disease models.
Chem Biol 2013; 20:1-11.
4 Reif K et al. Cutting Edge: Differential Roles for
Phosphoinositide 3 kinases, p110-gamma and p110-delta, in
lymphocyte chemotaxis and homing. J Immunol 2004:173:2236-2240.
5 Schmid M et al. Receptor Tyrosine Kinases and TLR/IL1Rs
Unexpectedly activate myeloid cell PI3K, a single convergent point
promoting tumor inflammation and progression. Cancer Cell
2011;19:715-727.
6 www.clinicaltrials.gov, NCT03372057.
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version on businesswire.com: https://www.businesswire.com/news/home/20200810005338/en/
Investors: John Doyle Vice President, Investor Relations &
Finance +1 781-469-1546 jdoyle@verastem.com
Media: Lisa Buffington Corporate Communications +1
781-292-4205
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