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Verastem Reports Second Quarter 2017 Financial Results
Company Expects to Report Top-line Data from Phase 3 DUO™ Study in Late Summer 2017
“The second quarter of 2017 was marked by several duvelisib data
presentations at top hematology-focused medical meetings,” said Robert
Second Quarter 2017 and Recent Highlights:
Ongoing Phase 3 DUO Study in Relapsed or Refractory CLL/SLL –
The efficacy and safety of duvelisib is currently being evaluated in
the randomized Phase 3 DUO study in patients with relapsed or
refractory CLL/SLL. In the DUO study, approximately 300 patients were
randomized 1:1 to receive duvelisib or ofatumumab. The trial was fully
November 2015. The primary endpoint of this study is progression free survival (PFS). Key secondary endpoints include overall response rate (ORR), overall survival (OS), duration of response (DOR) and safety. Verastemexpects to report top-line data from the DUO study in the latter part of summer 2017.
Presented Long-Term Follow Up Data in Patients with
Double-Refractory FL and SLL at EHA 2017 –
Long-term follow up data from the subsets of patients with FL or SLL
who were enrolled in the ongoing Phase 2 DYNAMO study were the subject
of presentations at EHA 2017 in
Madrid, Spain. In an oral presentation, titled “DYNAMO: A Phase 2 Study Demonstrating the Clinical Activity of Duvelisib in Patients with Double-Refractory Follicular Lymphoma,” Pier Luigi Zinzani, M.D., Ph.D., of the University of Bologna Instituteof Hematology, reported that duvelisib monotherapy demonstrated an ORR of 43%, as determined by an independent review committee, with 83% of patients experiencing a reduction in the size of target lymph nodes. The median DOR was 7.9 months, the median PFS was 8.3 months, and the median overall survival (OS) was 27.8 months. In an e-poster presentation, titled “DYNAMO: The Clinical Activity of Duvelisib in Patients with Double-Refractory Small Lymphocytic Lymphoma in a Phase 2 Study,” Dr. Zinzani reported that duvelisib as a monotherapy demonstrated an ORR of 68%, as determined by an independent review committee, with 100% of patients experiencing a reduction in the size of target lymph nodes. With a median time on duvelisib of 12 months, median DOR was 10.1 months, median PFS was 11.7 months, and median OS was 28.9 months. The safety profile of duvelisib monotherapy remained consistent with what has been previously reported in iNHL and other hematologic malignancies. Copies of Dr. Zinzani’s oral and e-poster presentations are available here and here.
Presented Long-Term Follow Up Data from the Phase 2 DYNAMO Study
at ICML 2017 – Long-term follow up data from the ongoing Phase
2 DYNAMO study was highlighted in an oral presentation at ICML 2017 in
Switzerland. In the presentation, titled “DYNAMO: A Phase 2 Study Demonstrating the Clinical Activity of Duvelisib in Patients with Double-Refractory Indolent Non-Hodgkin Lymphoma,” Dr. Zinzani, reported that duvelisib as a monotherapy demonstrated an ORR of 47%, as determined by an independent review committee, with 88% of patients experiencing a reduction in the size of target lymph nodes. Overall, the median DOR was 10 months, the median PFS was 9 months, and the median overall survival was 27.8 months. With additional follow-up (median 18 months), the safety profile of duvelisib monotherapy remained consistent with what has been previously reported in iNHL and other hematologic malignancies. The DYNAMO study met its primary ORR endpoint (p=0.0001) at the primary analysis. A PDF of Dr. Zinzani’s oral presentation is available here.
Published Scientific Data Highlighting Potential Role of Focal
Adhesion Kinase (FAK) Inhibition in Pancreatic and Breast Cancer –
July 2017, Verastemannounced the publication of two papers in the peer-reviewed journals, PLoS One and Oncotarget. The two published articles reported scientific findings from studies evaluating FAK inhibition in preclinical models of pancreatic and breast cancer and continue to validate the underlying thesis for ongoing clinical collaborations evaluating Verastem’s lead FAK inhibitor defactinib in combination with chemotherapeutic and leading immunotherapeutic agents in several difficult to treat types of cancer. The PLoS One paper in pancreatic cancer is available here and the Oncotarget paper in breast cancer is available here.
Presented Preclinical Data at the 2017
American Association for Cancer ResearchAnnual Meeting – In an oral presentation titled, “Reprogramming the tumor microenvironment to improve responses to therapy,” Verastemscientific collaborator David G. DeNardo, Ph.D., Associate Professor of Medicine, Division of Oncology, Department of Immunology, Washington University School of Medicinein St. Louis, described data demonstrating that FAK inhibition can enable efficacy of PD-1 inhibition in preclinical models of pancreatic cancer that, like the clinical disease, are otherwise refractory to checkpoint inhibition. Verastem’s FAK inhibitor, defactinib, is currently being evaluated in combination with Merck’s PD-1 inhibitor, pembrolizumab, and gemcitabine in patients with advanced pancreatic ductal adenocarcinoma (PDAC). Initial analysis of immune biomarkers from matched pairs of metastatic biopsies, taken either pre- or post-treatment, from patients with PDAC showed an increase in activated proliferating cytotoxic T cells together with a reduction in tumor-associated macrophages (TAMs). A PDF copy of Dr. DeNardo’s oral presentation is available here.
Corporate and Financial
Julie B. Feder Appointed Chief Financial Officer – In
July 2017, Verastemannounced the appointment of Ms. Feder as its Chief Financial Officer. Ms. Feder is an accomplished financial professional with invaluable leadership experience in the healthcare industry. She joins Verastemfrom the Clinton Health Access Initiative, Inc.(CHAI), where she served as Chief Financial Officer. Prior to joining CHAI, Ms. Feder held finance roles of increasing responsibility at Genzyme Corporationincluding leading the internal audit process. Ms. Feder began her career at Deloitte & Touche LLPand she holds a Bachelor of Science in Accounting from Yeshiva University’s Sy Syms School of Business.
Eric K. Rowinsky, M.D. Appointed to the Board of Directors – Verastemannounced the appointment of Eric K. Rowinsky, M.D., to its Board of Directors. Dr. Rowinsky brings to Verastemnearly 30 years of experience in the development of cancer treatments, such as cetuximab (Erbitux®) when he was Chief Medical Officer of ImClone Systems, as well as Cyramza®, Portrazza®, Taxol®, Taxotere®, Hycamtin®, Tarceva®, Camptosar®, Tykerb®, and cixutumumab, among others. He is a member of the board of directors of Biogen, Navidea, and Fortress Biotech, all public life sciences companies, and has served on the board of directors of BIND Therapeutics, a life-science company acquired by Pfizer. Dr. Rowinsky replaced Paul A. Friedman, M.D. who transitioned from his role as Director to become a member of Verastem’s Clinical and Scientific Advisory Board.
Second Quarter 2017 Financial Results
Net loss for the three months ended
Research and development expense for the 2017 Quarter was
General and administrative expense for the 2017 Quarter was
As of June 30, 2017, Verastem had cash, cash equivalents and investments of $57.9 million compared to $80.9 million as of December 31, 2016.
The number of outstanding common shares as of June 30, 2017, was 36,992,418.
Based on our current operating plans, we expect to have sufficient cash, cash equivalents and investments to fund our research and development programs and operations into 2018.
About the Tumor Microenvironment
The tumor microenvironment encompasses various cellular populations and extracellular matrices within the tumor or cancer niche that support cancer cell survival. This includes immunosuppressive cell populations such as regulatory T-cells, myeloid-derived suppressor cells, M2 TAMS, as well as tumor-associated fibroblasts and extracellular matrix proteins which can hamper the entry and therapeutic benefit of cytotoxic immune cells and anti-cancer drugs. In addition to targeting the proliferative and survival signaling of cancer cells, Verastem’s compounds duvelisib and defactinib target the tumor microenvironment as a mechanism of action to potentially improve a patient’s response to therapy.
Duvelisib is an investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes that are 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-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib is currently being evaluated in late- and mid-stage clinical trials, including DUO®, a randomized, Phase 3 monotherapy study in patients with relapsed/refractory CLL/SLL,4 and DYNAMO®, a single-arm, Phase 2 monotherapy study in patients with refractory iNHL that achieved its primary endpoint of ORR upon topline analysis of efficacy data.5 Duvelisib is also being evaluated for the treatment of hematologic malignancies through investigator-sponsored studies, including T-cell lymphoma.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.
Defactinib (VS-6063) is an investigational inhibitor of 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, enhancement of
anti-tumor immunity, and reduction of cancer stem cells.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,
ovarian, non-small cell lung cancer, and mesothelioma. These studies are
combination clinical trials with pembrolizumab and avelumab from
This press release includes forward-looking statements about
1 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.
2 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.
3 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.
6www.clinicaltrials.gov, NCT02783625, 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.
11 www.clinicaltrials.gov, NCT02943317
Note: Presentations referenced in this press release can be downloaded at www.verastem.com/research/posters.aspx
Condensed Consolidated Balance Sheets
|June 30,||December 31,|
|Cash, cash equivalents and investments||$||57,918||$||80,897|
|Prepaid expenses and other current assets||1,835||398|
|Property and equipment, net||1,127||1,417|
|Accounts payable and accrued expenses||$||11,066||$||10,991|
|Total liabilities and stockholders’ equity||$||61,839||$||83,629|
Unaudited Condensed Consolidated Statements of Operations
(in thousands, except per share amounts)
|Three months ended June 30,||Six months ended June 30,|
|Research and development||$||9,042||$||4,492||$||17,427||$||8,671|
|General and administrative||4,425||4,217||9,188||8,472|
|Total operating expenses||13,467||8,709||26,615||17,143|
|Loss from operations||(13,467||)||(8,709||)||(26,615||)||(17,143||)|
|Net loss per share—basic and diluted||$||(0.36||)||$||(0.23||)||$||(0.71||)||$||(0.46||)|
Weighted-average number of common
shares used in net loss per share-basic and diluted
Brian Sullivan, 781-292-4214