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Verastem Reports Year-End 2016 Financial Results
“2016 was a year of significant achievement for
Mr. Forrester continued, “On the financial front, we ended 2016 with
Fourth Quarter 2016 and Recent Highlights:
In-licensed Late-stage, Complementary Oncology Product Candidate
Verastemand Infinity Pharmaceuticals, Inc.(Infinity) announced the signing of an agreement under which Verastemlicensed exclusive worldwide rights to develop and commercialize duvelisib, an investigational product candidate currently in development for hematologic malignancies. Duvelisib is well aligned with Verastem'sstrategic focus of developing novel anti-cancer therapeutics that modulate the tumor microenvironment. The transaction provides a new oncology product candidate with demonstrated activity in lymphoid malignancies.
Ongoing Phase 3 DUO Study in Relapsed or Refractory CLL –
The safety and efficacy of duvelisib is currently being evaluated in
the randomized Phase 3 DUO study in patients with relapsed or
refractory CLL. In the DUO study, approximately 300 patients were
randomized 1:1 to receive duvelisib (25mg BID) or ofatumumab (8 weekly
infusions, starting with an initial intravenous dose of 300mg on day 1
followed by 7 weekly doses of 2,000mg, then 2,000mg monthly for 4
cycles). The primary endpoint of this study is progression free
survival (PFS). Key secondary endpoints include overall response rate
(ORR), overall survival, duration of response (DOR) and safety.
Verastemexpects to report top-line data from the DUO study in mid-year 2017.
Positive Phase 2 DYNAMO™ Data Reported at ASH 2016
– Positive Phase 2 clinical data from the DYNAMO study demonstrating
the clinical activity of duvelisib in patients with relapsed
refractory indolent non-Hodgkin lymphoma (iNHL) were presented at the
American Society of Hematology(ASH) Annual Meeting in December 2016. In an oral presentation, titled “A phase 2 study demonstrating the clinical activity of duvelisib in patients with relapsed refractory indolent non-Hodgkin lymphoma,” (Publication ID: 1218) Ian Flinn, M.D., Ph.D. (Director, Hematologic Malignancies Program, Sarah Cannon Research Institute), described results from 129 patients with double refractory iNHL (median 3 prior anti-cancer regimens, range 1-18). The study met its primary endpoint, achieving an ORR of 46% as determined by an independent review committee (IRC) (p=0.0001; 95% CI 0.37-0.55). Among disease subgroups, the ORR was 41% in follicular lymphoma (n=83), 68% in small lymphocytic lymphoma (n=28), and 33% in marginal zone lymphoma (n=18). The median DOR among all patients was 9.9 months. Notably, 83% of patients had reductions in the size of their target lymph nodes per the IRC. Duvelisib was generally well tolerated, with an expected and manageable safety profile with appropriate risk mitigation. The DYNAMO study showed that duvelisib monotherapy has a favorable benefit-risk profile in refractory iNHL patients and may represent an important treatment option in this population.
Dosed the First Patient in Combination Trial of Defactinib and
Avelumab in Patients with Ovarian Cancer – As announced
January 2017, the first patient was dosed in a new clinical trial evaluating avelumab, an investigational fully human anti-PD-L1 IgG1 monoclonal antibody, in combination with Verastem’s defactinib, an investigational focal adhesion kinase (FAK) inhibitor, in patients with advanced ovarian cancer. This multicenter, open-label, dose-escalation and dose-expansion Phase 1/2 clinical trial is designed to assess the safety, pharmacokinetics, pharmacodynamics, and initial observations of clinical activity of the avelumab/defactinib combination in patients with recurrent or refractory stage III-IV ovarian cancer. The study is being conducted in collaboration with the alliance between Merck KGaA, Darmstadt, Germany, which in the U.S. and Canadaoperates as EMD Serono, and Pfizer, and is expected to enroll approximately 100 patients at up to 15 sites across the U.S.
Corporate and Financial
Hagop Youssoufian, MSc, M.D., Named Head of Hematology and Oncology Development– In January 2017, Dr. Youssoufian assumed this leadership role at Verastemto oversee the clinical and regulatory development of Verastem’s pipeline, including duvelisib, and provide overall strategic and tactical leadership to our hematology-oncology clinical programs. Dr. Youssoufian brings over 25 years of product development and commercialization experience to Verastem, having served in senior leadership roles at several oncology-focused companies, including BIND Therapeutics, Progenics Pharmaceuticals, Ziopharm Oncology, Imclone Systems, Sanofi Aventis and Bristol-Myers Squibbwhere he was involved in the development of Sprycel®, Taxotere® and Erbitux®.
Additional Key Personnel Appointments – Recently,
Michael Ferraressojoined Verastemas Vice President, Commercial Operations, and Verastemalso appointed several highly experienced individuals to the Clinical and Scientific Advisory Boardincluding:
Lori Kunkel, M.D., Former Chief Medical Officer, Pharmacyclics
Edmund J. Pezalla, M.D., MPH, Former VP, Pharmaceutical Policy and Strategy at Aetna
Greg Berk, M.D., Former Chief Medical Officer, Verastem
Cheryl Cohen, Former Chief Commercial Officer, Medivation
Brian Stuglik, PharmD., Former VP and Chief Marketing Officer, Oncology Global Marketing, Eli Lilly
$25 MillionLoan Facility – In March 2017, Verastementered into a Loan and Security Agreement with Hercules Capital, Inc.for up to $25.0 millionin financing. Verastemreceived the first $2.5 millionof financing under the Loan and Security Agreement when the transaction closed. The proceeds will be used for Verastem’s ongoing research and development programs and for general corporate purposes. Additional tranches of up to $22.5 millionin aggregate will be available subject to certain conditions, including positive data from the Phase 3 DUO clinical trial evaluating duvelisib in patients with relapsed or refractory CLL.
Full Year 2016 Financial Results
Net loss for the year ended
Research and development expense for the 2016 Period was
General and administrative expense for the 2016 Period was
As of December 31, 2016, Verastem had cash, cash equivalents and investments of $80.9 million compared to $110.3 million as of December 31, 2015. Verastem used $29.5 million for operating activities during the 2016 Period.
The number of outstanding common shares as of December 31, 2016, 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.
Conference Call Information
The live, listen-only webcast of the conference call can be accessed by visiting the investors section of the Company’s website at www.verastem.com. A replay of the webcast will be archived on the Company’s website for 90 days following the call.
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 tumor-associated macrophages, 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 product candidates, including duvelisib and defactinib, also 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 or refractory CLL4, and DYNAMO™, a single-arm, Phase 2 monotherapy study in patients with refractory iNHL that achieved its primary endpoint of ORR upon top-line analysis of efficacy data5. 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 is an investigational inhibitor of FAK, a non-receptor
tyrosine kinase encoded by the PTK-2 gene 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 cancer, ovarian
cancer, 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
7Schaller MD and Parsons JT. Focal adhesion kinase: an integrin-linked protein tyrosine kinase. Trends Cell Biol. 1993 3: 258-62.
8Jiang H et al. Targeting focal adhesion kinase renders pancreatic cancers responsive to checkpoint immunotherapy. Nat Med 2016:
9Sulzmaier FJ et al. FAK in cancer: mechanistic findings and clinical applications. Nature Rev Cancer. 2014 14: 598-610.
11 www.clinicaltrials.gov, NCT02943317
Unaudited Selected Consolidated Balance Sheets
|December 31,||December 31,|
|Cash, cash equivalents and investments||$||80,897||$||110,258|
|Prepaid expenses and other current assets||398||585|
|Property and equipment, net||1,417||2,048|
|Accounts payable and accrued expenses||$||10,991||$||10,040|
|Total liabilities and stockholders’ equity||$||83,629||$||113,094|
Unaudited Condensed Consolidated Statements of Operations
(in thousands, except per share amounts)
|Year ended December 31,|
|Research and development||$||19,779||$||40,565|
|General and administrative||17,223||17,634|
|Total operating expenses||37,002||58,199|
|Loss from operations||(37,002)||(58,199)|
|Net loss per share—basic and diluted||$||(0.99)||$||(1.61)|
Weighted-average number of common shares used in net loss per
Brian Sullivan, 781-292-4214