On March 13, 2018 Verastem, Inc. (NASDAQ:VSTM), focused on developing and commercializing drugs to improve the survival and quality of life of cancer patients, reported financial results for the year ended December 31, 2017 and provided an overview of certain corporate developments and plans (Press release, Verastem, MAR 13, 2018, View Source;p=RssLanding&cat=news&id=2337744 [SID1234524706]).
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"The last year has been marked by significant achievement for Verastem with the reporting of positive data from the pivotal Phase 3 DUO study and culminating in the recent submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking full approval for duvelisib for the treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and accelerated approval for the treatment of relapsed or refractory follicular lymphoma (FL)," said Robert Forrester, President and Chief Executive Officer of Verastem. "As we await the potential acceptance and approval of the duvelisib NDA, we are diligently working to build our commercial infrastructure and preparing for our first potential product launch. I am delighted that Joe Lobacki has joined the team. Joe’s formidable expertise in commercialising oncology drugs at Medivation, Micromet and Genzyme positions Verastem to successfully execute on our launch plan for duvelisib in the US."
Fourth Quarter 2017 and Recent Highlights:
Duvelisib
Duvelisib NDA submitted to FDA – In early February 2018, Verastem submitted an NDA to the FDA 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 (Free ASH Whitepaper) 2017 – Verastem presented results from the Phase 3 DUO study at the American Society of Hematology (ASH) (Free ASH Whitepaper) 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 investigator Ian Flinn, M.D., Ph.D., Director of the Blood Cancer Research Program at Sarah 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 revised International 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 (Free ASH Whitepaper) 2017 – Along with the Phase 3 DUO results, two additional duvelisib abstracts were presented at ASH (Free ASH Whitepaper) 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 (ASCO) (Free ASCO Whitepaper) Clinical Immuno-Oncology Symposium (ASCO-SITC) – In January 2018, Jonathan Pachter, Ph.D., Chief Scientific Officer of Verastem, 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
Defactinib Preclinical Abstract Presented at ASH (Free ASH Whitepaper) 2017 – A poster describing preclinical data in combination with B-cell lymphoma 2 (BCL-2) was presented at ASH (Free ASH Whitepaper) 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
Joseph Lobacki Appointed Chief Commercial Officer – In January 2018, Verastem announced the appointment of Joseph 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 the Verastem team.
Additional Financing Through Increasing Debt Facility to up to $50.0 Million and a $25.0 Million Public Offering – In January 2018, Verastem amended its loan and security agreement with Hercules 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. In December 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 – In October 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 joins Verastem from MedImmune (a wholly owned 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. Dr. Le oversees the development strategy and activities for Verastem’s core assets, duvelisib and defactinib.
Paid First Development Milestone to Infinity Pharmaceuticals – In October 2017, Verastem paid to Infinity 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 December 31, 2017 (2017 Period) was $67.8 million, or $1.76 per share, as compared to a net loss of $36.4 million, or $0.99 per share, for the year ended December 31, 2016 (2016 Period). Net loss includes non-cash stock-based compensation expense of $5.0 million and $6.2 million for the 2017 Period and 2016 Period, respectively. Verastem used $57.3 million of cash for operating activities during the 2017 Period.
Research and development expense for the 2017 Period was $46.4 million compared to $19.8 million for the 2016 Period. The $26.6 million increase from the 2016 Period to the 2017 Period was primarily related to an increase of $13.4 million in external clinical research organization expense for outsourced biology, chemistry, development and clinical services, which includes our clinical trial costs, the achievement of a $6.0 million milestone pursuant to our license agreement with Infinity, an increase of $5.1 million in consulting fees, and an increase in personnel related costs of $1.9 million.
General and administrative expense for the 2017 Period was $21.4 million compared to $17.2 million for the 2016 Period. The increase of $4.2 million from the 2016 Period to the 2017 Period primarily resulted from increases in consulting and professional fees of $4.4 million, including $2.5 million related to commercial launch preparation, and an increase in personnel costs of $1.0 million. These increases were partially offset by a decrease in stock-based compensation expense of $1.5 million.
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 Verastem has submitted 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 follicular lymphoma (FL). Duvelisib is also being developed by Verastem for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov.
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 Merck & Co. and Pfizer/Merck KGaA, respectively.10,11,12 Information about these and additional clinical trials evaluating the safety and efficacy of defactinib can be found on www.clinicaltrials.gov.