Full Press Release Details
Verastem Oncology Signs Definitive Agreement to Sell COPIKTRA (duvelisib) Rights to Secura Bio to Focus on Development of
VS-6766 and Defactinib in KRAS Mutant Solid Tumors
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
- August 10, 2020 - 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.
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.
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
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 |
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.
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.
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.
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
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.
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.
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
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.
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)
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.
Health Partners, L.P and Ropes & Gray acted as advisors to Verastem Oncology on this transaction.
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.