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Verastem Reports Third Quarter 2017 Financial Results
“The third quarter was a pivotal time for
Mr. Forrester concluded, “At the upcoming
Third Quarter 2017 and Recent Highlights:
Announced Regulatory Strategy for Duvelisib NDA –
Verastemrecently announced that a meeting was held with the U.S. Food and Drug Administration( FDA) regarding the regulatory path for duvelisib. Based on the meeting with, and written feedback from the FDA, Verastemintends to submit a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL and accelerated approval for the treatment of patients with relapsed or refractory FL. Along with the clinical data from the DUO study, the duvelisib NDA submission will also contain the favorable results from the Phase 2 DYNAMO™ study in double-refractory indolent non-Hodgkin’s lymphoma (iNHL), which also achieved its primary endpoint with an ORR of 46% (p<0.0001). In the subset of patients enrolled in the DYNAMO study with double-refractory FL (n=83), duvelisib demonstrated an ORR of 41%. The Company expects to submit the duvelisib NDA during the first quarter of 2018.
Reported Positive Top-line Data from the Pivotal Phase 3 DUO
Study in Relapsed or Refractory CLL/SLL – In
September 2017, Verastemreported that the Phase 3 DUO study met its primary endpoint with oral duvelisib monotherapy demonstrating superiority over ofatumumab for PFS in patients with CLL/SLL. In this study, duvelisib achieved a statistically significant improvement in median PFS of 13.3 months, compared to 9.9 months for ofatumumab with a hazard ratio of 0.52 (p<0.0001), representing a 48% reduction in the risk of progression or death. Duvelisib monotherapy had 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.
Expanded Duvelisib Program to Include Peripheral T-Cell Lymphoma
September 2017, Verastemannounced the expansion of its duvelisib development program to include targeting the treatment of patients with Peripheral T-Cell Lymphoma (PTCL). Duvelisib was granted Fast Track designation by the FDAfor the treatment of patients with PTCL who have received at least one prior therapy. Development of duvelisib in PTCL is supported by encouraging Phase 1 clinical data which demonstrated a 50% investigator-assessed overall response rate in 16 heavily pre-treated patients with relapsed or refractory PTCL, including 3 (19%) complete responses and 5 (31%) partial responses. Verastemintends to initiate an open-label, multicenter, Phase 2 clinical trial evaluating the efficacy and safety of duvelisib in patients with relapsed or refractory PTCL by the end of 2017.
Clinical Data from Pivotal Phase 3 DUO Study Selected for Oral
Presentation at ASH 2017 –
Verastemrecently announced that an abstract highlighting clinical data from the Phase 3 DUO study was selected for oral presentation at 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,” will be presented by principal investigator, Ian Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program at Sarah Cannon Research Institute, on Sunday, December 10, 2017at 4:30pm ETat the Georgia World CongressCenter, in Building B, Level 5, Murphy BR 3-4.
Additional Duvelisib Abstracts Selected for Presentation at ASH
2017 – Along with the Phase 3 DUO results, two additional
duvelisib abstracts were selected for presentation 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,” will be given as an oral
Steven Horowitz, M.D., Memorial Sloan Kettering Cancer Center, on Monday, December 11, 2017at 5:00pm ETat the Georgia World Congress Center, in Building A, Level 4, Marcus Auditorium. The abstract, 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,” will be given as a poster presentation by Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem, on Saturday, December 9, 2017from 5:30-7:30pm ETat the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Verastemto Host Key Opinion Leader Event at ASH 2017 – On Sunday, December 10, 2017, Verastemwill host an investor and analyst reception, which will feature a moderated panel discussion/Q&A including Ian Flinn, MD, Director of the Blood Cancer Research Program at Sarah Cannon Cancer Centerin Nashville, TN.The event will take place during the ASH 2017 annual meeting and interested parties can access a live webcast of the event beginning December 10, 2017at 8pm ETby going to the “News and Press” section of the Verastemwebsite at www.verastem.com.
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.
Defactinib Preclinical Abstract Selected for Presentation at ASH
2017 – An abstract describing preclinical data in combination
with BCL-2 was selected for presentation at ASH 2017. The abstract,
titled “Combinatorial Inhibition of Focal Adhesion Kinase and BCL-2 in
AML,” will be given as a poster presentation by Xiangmeng Wang, Ph.D.,
Sunday, December 10, 2017from 6:00-8:00pm ETat the Georgia World Congress Center, in Building A, Level 1, Hall A2.
Corporate and Financial
Brian Stuglik, R.Ph. Appointed to the Board of Directors – In September 2017, Verastemannounced the appointment of Mr. Stuglik to its Board. Mr. Stuglik, former Chief Marketing Officer for Lilly Oncology, brings to Verastem35 years of experience in pharmaceutical and oncology commercialization in both the U.S. and international markets. He has successfully launched several multi-billion dollar brands over his career, including Gemzar®, Alimta® and Erbitux®.
NgocDiep Le, M.D., Ph.D., Appointed Chief Medical Officer – In October 2017, Verastemannounced 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 hematologic malignancies as well as IND and NDA submissions. Dr. Le joins Verastemfrom MedImmune (a subsidiary of AstraZeneca) where she served as Vice President, Immuno-Oncology Innovative Medicines and led the product development teams for multiple high-priority immuno-oncology assets. Prior to joining MedImmune, Dr. Le held roles of increasing responsibilities at Novartisand at GlaxoSmithKline where she led the MEK inhibitor, trametinib (MekinistTM), from the first-in-human studies to FDAapproval. Dr. Le received a Bachelor in Science degree from the California Institute of Technology, and earned both MD and PhD degrees from Stanford University School of Medicine. Dr. Le will oversee the development strategy and activities for Verastem’s duvelisib and defactinib.
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 function. 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.
Achieved First Development Milestone Related to the Duvelisib
License Agreement – In
September 2017, upon achievement of positive top-line results from the Phase 3 DUO study, Verastemdetermined that the pre-specified criteria for the first milestone under the license agreement with Infinity Pharmaceuticals, Inc.(Infinity) had been met and recorded $6.0 millionas research and development expense. Subsequently, in October 2017, Verastemmade the milestone payment of $6.0 millionto Infinity. The milestone was paid using funds drawn from Verastem’s existing loan and security agreement with Hercules Capital, Inc.
Third 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 September 30, 2017, Verastem had cash, cash equivalents and investments of $60.3 million compared to $80.9 million as of December 31, 2016.
The number of outstanding common shares as of September 30, 2017, was 39,945,028.
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 the second half of 2018.
Duvelisib is an 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-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
chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (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
Defactinib 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, 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
|Condensed Consolidated Balance Sheets|
|Cash, cash equivalents and investments||$||60,264||$||80,897|
|Prepaid expenses and other current assets||940||398|
|Property and equipment, net||989||1,417|
|Accounts payable and accrued expenses||$||19,618||$||10,991|
|Total liabilities and stockholders’ equity||$||63,139||$||83,629|
|Unaudited Condensed Consolidated Statements of Operations|
(in thousands, except per share amounts)
|Three months ended September 30,||Nine months ended September 30,|
|Research and development||$||17,743||$||4,216||$||35,170||$||12,887|
|General and administrative||5,394||3,843||14,582||12,315|
|Total operating expenses||23,137||8,059||49,752||25,202|
|Loss from operations||(23,137)||(8,059)||(49,752)||(25,202)|
|Net loss per share—basic and diluted||$||(0.61)||$||(0.21)||$||(1.33)||$||(0.67)|
|Weighted-average number of common shares used in net loss per share-basic and diluted||37,630||36,992||37,207||36,986|
Brian Sullivan, 781-292-4214
Director, Corporate Development