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Verastem Reports Third Quarter 2016 Financial Results
Company Adds Late-Stage, Complementary Oncology Product Candidate Duvelisib to Pipeline
“Last week, we announced the inlicensing of the late-stage oncology
product candidate duvelisib from
Mr. Forrester continued, “Since the beginning of 2016, we have made
significant progress with our immuno-oncology focused clinical
development program aimed at advancing our FAK inhibitors in combination
with immunotherapies and other current and emerging standard of care
treatments. We recently announced a new clinical collaboration with
Third Quarter 2016 and Recent Highlights:
Inlicensed Late-stage, Complementary Oncology Product Candidate
Duvelisib – Last week,
Verastemand Infinity Pharmaceuticals, Inc.(Infinity) announced the signing of an agreement under which Verastemlicensed exclusive worldwide rights to develop and commercialize Infinity’s duvelisib, an investigational product candidate currently in development for hematologic malignancies. In consideration for duvelisib, Verastemwill pay to Infinity no upfront payment, and up to $28 millionin milestones. Positive data from DUO®, a Phase 3, randomized monotherapy study of duvelisib in patients with relapsed or refractory CLL, triggers the first milestone payment. Verastemwill also pay royalties on worldwide net sales. Duvelisib is well aligned with Verastem'sstrategic focus of developing novel anti-cancer therapeutics that modulate the tumor microenvironment.
Phase 2 DYNAMO Data to be Reported at ASH 2016 – Phase 2
clinical data for duvelisib will be presented at the 58th
American Society of Hematology(ASH) Annual Meeting, which is being held December 3-6, 2016in San Diego. 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, MD, PhD, Director, Hematologic Malignancies Program, Sarah Cannon Research Institute, will describe the results from DYNAMO®, a Phase 2 study evaluating the efficacy and safety of duvelisib in relapsed/refractory iNHL. The oral presentation will take place on Monday, December 5, 2016, at 7:30 PM PTat the San Diego Convention Center, Ballroom 20BC.
New Clinical Collaboration with
Cancer Research UKand MSD to Evaluate Defactinib in Combination with Immunotherapy in Mesothelioma, Non-small Cell Lung and Pancreatic Cancer – In September 2016, the companies announced a new clinical trial collaboration agreement to evaluate the combination of Verastem’s defactinib and MSD’s PD-1 immunotherapy pembrolizumab (Keytruda®). This clinical collaboration is based on discoveries by scientists at the Edinburgh Cancer ResearchUK Centre at the University of Edinburghwho showed that inhibiting FAK increases the effectiveness of anti-PD-1 agents. The trial is expected to enroll up to 60 patients and will commence in early 2017.
Published Preclinical Researchin Nature Medicine – In July 2016, Verastemannounced the publication of preclinical research conducted by its scientific collaborator, David G. DeNardo, PhD, Assistant Professor of Medicine, Division of Oncology, Department of Immunology, Washington University School of Medicinein St. Louis. In the published study, Dr. DeNardo demonstrates that FAK inhibition decreases fibrosis and immunosuppressive cell populations in pancreatic ductal adenocarcinoma, rendering previously unresponsive tumors sensitive to chemo- and immunotherapy. These findings provide important support and rationale for the ongoing Phase 1 dose-escalation clinical studies evaluating Verastem’s FAK inhibitors in combination with pembrolizumab and gemcitabine, and, gemcitabine and Abraxane® in patients with pancreatic cancer.
Third Quarter 2016 Financial Results
Net loss for the third quarter ended
Research and development expense for the 2016 Quarter was
General and administrative expense for the 2016 Quarter was
As of September 30, 2016, Verastem had cash, cash equivalents and investments of $86.9 million compared to $110.3 million as of December 31, 2015. Verastem used $6.0 million for operating activities during 2016 Quarter.
The number of outstanding common shares as of September 30, 2016, was 36,992,418.
Based on current operating plans, we expect to have sufficient cash, cash equivalents and short-term 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 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 compounds duvelisib, defactinib, VS-4718 and VS-5584 also target the tumor microenvironment as a mechanism of action to potentially improve a patients 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 chronic lymphocytic leukemia (CLL)4, and DYNAMO®, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL) that achieved its primary endpoint of overall response rate upon topline analysis of efficacy data5. Duvelisib is also being evaluated for the treatment of hematologic malignancies through investigator-sponsored studies, including T cell lymphoma. Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.6
Defactinib (VS-6063) is an investigational inhibitor of Focal Adhesion
Kinase (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, 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
1Winkler 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.
2Reif 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.
3Schmid 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: Aug 22(8) 851-60.
9Sulzmaier FJ et al. FAK in cancer: mechanistic findings and clinical applications. Nature Rev Cancer. 2014 14: 598-610.
|Unaudited Selected Consolidated Balance Sheets|
|September 30,||December 31,|
|Cash, cash equivalents and investments||$||86,882||$||110,258|
|Prepaid expenses and other current assets||431||585|
|Property and equipment, net||1,569||2,048|
|Accounts payable and accrued expenses||$||6,220||$||10,040|
|Total liabilities and stockholders’ equity||$||89,044||$||113,094|
|Unaudited Condensed Consolidated Statements of Operations|
(in thousands, except per share amounts)
|Three months ended September 30,||Nine months ended September 30,|
|Research and development||$||4,216||$||11,304||$||12,887||$||32,877|
|General and administrative||3,843||4,230||12,315||13,361|
|Total operating expenses||8,059||15,534||25,202||46,238|
|Loss from operations||(8,059)||(15,534)||(25,202)||(46,238)|
|Net loss per share—basic and diluted||$||(0.21)||$||(0.42)||$||(0.67)||$||(1.29)|
Weighted-average number of
Brian Sullivan, 781-292-4214