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Verastem Reports Year-End 2017 Financial Results
“The last year has been marked by significant achievement for
Fourth Quarter 2017 and Recent Highlights:
Duvelisib
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Duvelisib NDA submitted to
FDA – In earlyFebruary 2018 ,Verastem submitted an NDA to theFDA seeking full approval for its lead product candidate duvelisib, a first-in-class oral dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, for the treatment of relapsed or refractory CLL/SLL and accelerated approval for the treatment of relapsed or refractory FL. The NDA is supported by clinical data from the randomized Phase 3 DUO™ study, which met its primary endpoint by demonstrating statistically significant efficacy, along with a consistent and manageable safety profile, for duvelisib monotherapy in patients with relapsed or refractory CLL/SLL. The NDA is also supported by results from the Phase 2 DYNAMO™ study, which also met its primary endpoint by demonstrating a statistically significant improvement in overall response rate (ORR) compared to an historical control in patients with indolent non-Hodgkin’s lymphoma that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy. -
Clinical Data from Pivotal Phase 3 DUO Study Highlighted in an
Oral Presentation at ASH 2017 –
Verastem presented results from the Phase 3 DUO study at theAmerican Society of Hematology 2017 Annual Meeting (ASH 2017). The presentation, titled “Results from the Phase 3 DUO Trial: A Randomized Comparison of Duvelisib vs Ofatumumab in Patients with Relapsed/Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma,” was presented by principal investigatorIan Flinn , M.D., Ph.D., Director of the Blood Cancer Research Program atSarah Cannon Research Institute . The DUO study met its primary endpoint with oral duvelisib monotherapy achieving a statistically significant improvement in progression free survival (PFS) compared to ofatumumab in patients with relapsed or refractory CLL/ SLL per a blinded independent review committee (IRC) using modified international workshop on CLL (iwCLL) and revisedInternational Working Group (IWG) Response Criteria (median PFS=13.3 months versus 9.9 months, respectively; HR=0.52, p<0.0001), representing a 48% reduction in the risk of progression or death. Oral duvelisib monotherapy also achieved a statistically significant improvement in ORR compared to ofatumumab (74% vs 45%, respectively; p<0.0001), and reduced lymph node burden of less than 50% in most patients compared to ofatumumab (85% vs 16%, respectively). Duvelisib monotherapy demonstrated a manageable safety profile, with results from this study consistent with the well-characterized safety profile of duvelisib monotherapy in patients with advanced hematologic malignancies in previous studies. For duvelisib-treated patients, the median time on treatment was 50.3 weeks (range, 0.9 - 160.0) compared to 23.1 weeks (range, 0.1 - 26.1) for ofatumumab. -
Additional Duvelisib Abstracts Presented at ASH 2017 –
Along with the Phase 3 DUO results, two additional duvelisib abstracts
were presented at ASH 2017. The abstract, titled “In Vitro, In Vivo,
and Parallel Phase I Evidence Support the Safety and Activity of
Duvelisib, a PI3K-δ,γ Inhibitor, in Combination with Romidepsin or
Bortezomib in Relapsed/Refractory T-Cell Lymphoma,” was given as an
oral presentation by
Alison Moskovitz , M.D.,Memorial Sloan Kettering Cancer Center . -
Preclinical Data Highlighting the Synergistic Effects in
Combination with Immunotherapy Presented at the
American Society of Clinical Oncology Clinical Immuno-Oncology Symposium (ASCO-SITC) – InJanuary 2018 ,Jonathan Pachter , Ph.D., Chief Scientific Officer ofVerastem , presented preclinical data highlighting the potential synergistic effects of duvelisib in combination with immune checkpoint or co-stimulatory antibodies in B-cell lymphoma. This data, outlined in a poster titled “The Dual PI3K-δ,γ Inhibitor Duvelisib Stimulates Anti-Tumor Immunity and Enhances Efficacy of Immune Checkpoint and Co-Stimulatory Antibodies in a B-Cell Lymphoma Model,” supports the further exploration of duvelisib in combination with anti-PD-1/PD-L1 or co-stimulatory antibodies in patients with B-cell malignancies.
Defactinib
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Defactinib Preclinical Abstract Presented at ASH 2017 – A
poster describing preclinical data in combination with B-cell lymphoma
2 (BCL-2) was presented at ASH 2017. The abstract, titled
“Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in AML,”
was presented by Xiangmeng Wang, Ph.D.,
MD Anderson Cancer Center .
Corporate and Financial
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Joseph Lobacki Appointed Chief Commercial Officer – In
January 2018 ,Verastem announced the appointment ofJoseph Lobacki as Executive Vice President and Chief Commercial Officer. Mr. Lobacki, formerly Chief Commercial Officer and Executive Council Member at Medivation, is responsible for overseeing the commercial strategy and execution for Verastem’s lead product candidate, duvelisib. Mr. Lobacki is a skilled leader in commercializing oncology drugs and his strong experience in hematologic oncology commercialization and marketing make him an invaluable addition to theVerastem team. -
Additional Financing Through Increasing Debt Facility to up to
$50.0 Million and a$25.0 Million Public Offering – InJanuary 2018 ,Verastem amended its loan and security agreement withHercules Capital, Inc. (Hercules), increasing its existing borrowing limit under the loan facility from up to$25.0 million to up to$50.0 million in financing, subject to certain conditions of funding. InDecember 2017 , the Company successfully completed an underwritten public offering of shares of common stock with gross proceeds totaling approximately $25.0 million. -
NgocDiep Le , MD, PhD, Appointed Chief Medical Officer – InOctober 2017 ,Verastem announced the appointment of Dr. Le as its Chief Medical Officer. A trained medical oncologist, Dr. Le is board certified in internal medicine and has 15 years of drug development experience across all phases in both solid and liquid tumors, with specialized expertise in clinical development. Dr. Le joinsVerastem from MedImmune (a wholly owned subsidiary ofAstraZeneca ) where she served as Vice President, Immuno-Oncology Innovative Medicines and led the product development teams for multiple high-priority immuno-oncology assets. Dr. Le oversees the development strategy and activities for Verastem’s core assets, duvelisib and defactinib. -
Paid First Development Milestone to
Infinity Pharmaceuticals – InOctober 2017 ,Verastem paid toInfinity Pharmaceuticals, Inc. (Infinity) a$6.0 million milestone payment, representing the first milestone under the duvelisib license agreement. This milestone is based on the achievement of positive top-line results from the Phase 3 DUO study evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory CLL/SLL. The milestone was paid using funds drawn from Verastem’s existing loan and security agreement with Hercules.
Full Year 2017 Financial Results
Net loss for the year ended
Research and development expense for the 2017 Period was
General and administrative expense for the 2017 Period was
As of December 31, 2017, Verastem had cash, cash equivalents and investments of $86.7 million compared to $80.9 million as of December 31, 2016.
The number of outstanding common shares as of December 31, 2017, was 50,800,908.
Financial Guidance
Based on our current operating plans, we expect to have sufficient cash, cash equivalents and investments to fund our current operating plan and capital expenditure requirements into the second half of 2018.
About Duvelisib
Duvelisib is a first-in-class investigational, dual inhibitor of
phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known
to help support the growth and survival of malignant B-cells and
T-cells. PI3K signaling may lead to the proliferation of malignant B-
and T-cells and is thought to play a role in the formation and
maintenance of the supportive tumor microenvironment.1,2,3
Duvelisib was evaluated in late- and mid-stage extension trials,
including DUO™, a randomized, Phase 3 monotherapy study in patients with
relapsed or refractory chronic lymphocytic leukemia/small lymphocytic
lymphoma (CLL/SLL),4 and DYNAMO™, a single-arm, Phase 2
monotherapy study in patients with refractory indolent non-Hodgkin
lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary
endpoints and
About Defactinib
Defactinib is an investigational inhibitor of focal adhesion kinase
(FAK), a non-receptor tyrosine kinase that mediates oncogenic signaling
in response to cellular adhesion and growth factors.7 Based
on the multi-faceted roles of FAK, defactinib is used to treat cancer
through modulation of the tumor microenvironment and enhancement of
anti-tumor immunity.8,9 Defactinib is currently being
evaluated in three separate clinical collaborations in combination with
immunotherapeutic agents for the treatment of several different cancer
types including pancreatic cancer, ovarian cancer, non-small cell lung
cancer (NSCLC), and mesothelioma. These studies are combination clinical
trials with pembrolizumab and avelumab from
About
This press release includes forward-looking statements about
References
1 Winkler 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.
2
Reif 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.
3 Schmid 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.
4www.clinicaltrials.gov,
NCT02004522
5www.clinicaltrials.gov,
NCT01882803
6www.clinicaltrials.gov,
NCT02783625, NCT02158091
7 Schaller M.D. and Parsons
J.T. Focal adhesion kinase: an integrin-linked protein tyrosine kinase.
Trends Cell Biol. 1993 3: 258-62.
8 Jiang H et al.
Targeting focal adhesion kinase renders pancreatic cancers responsive to
checkpoint immunotherapy. Nat Med 2016:
9
Sulzmaier F.J. et al. FAK in cancer: mechanistic findings and clinical
applications. Nature Rev Cancer. 2014 14: 598-610.
10www.clinicaltrials.gov,
NCT02546531
11www.clinicaltrials.gov,
NCT02943317
12www.clinicaltrials.gov,
NCT02758587
Verastem, Inc. Selected Consolidated Balance Sheets (in thousands) |
||||||||
December 31, | December 31, | |||||||
2017 | 2016 | |||||||
Cash, cash equivalents and investments | $ | 86,672 | $ | 80,897 | ||||
Prepaid expenses and other current assets | 1,115 | 398 | ||||||
Property and equipment, net | 861 | 1,417 | ||||||
Other assets | 1,143 | 917 | ||||||
Total assets | $ | 89,791 | $ | 83,629 | ||||
Accounts payable and accrued expenses | $ | 17,128 | $ | 10,991 | ||||
Long-term debt | 14,828 | — | ||||||
Other liabilities | 151 | 341 | ||||||
Stockholders’ equity | 57,684 | 72,297 | ||||||
Total liabilities and stockholders’ equity | $ | 89,791 | $ | 83,629 | ||||
Verastem, Inc. Consolidated Statements of Operations (in thousands, except per share amounts) |
|||||||||||
Year ended December 31, | |||||||||||
2017 | 2016 | ||||||||||
Operating expenses: | |||||||||||
Research and development | $ | 46,423 | $ | 19,779 | |||||||
General and administrative | 21,381 | 17,223 | |||||||||
Total operating expenses | 67,804 | 37,002 | |||||||||
Loss from operations | (67,804 | ) | (37,002 | ) | |||||||
Interest income | 561 | 562 | |||||||||
Interest expense | (559 | ) | — | ||||||||
Net loss | $ | (67,802 | ) | $ | (36,440 | ) | |||||
Net loss per share—basic and diluted | $ | (1.76 | ) | $ | (0.99 | ) | |||||
Weighted-average number of common shares used in net loss per
share- |
38,422 | 36,988 | |||||||||
View source version on businesswire.com: http://www.businesswire.com/news/home/20180313005585/en/
Source:
Verastem, Inc.
Marianne M. Lambertson
Vice President,
Corporate Communications
Investor Relations/Public Relations
+1
781-292-4273
mlambertson@verastem.com