Verastem Oncology
Annual Report 2013

Plain-text annual report

Use these links to rapidly review the documentTABLE OF CONTENTS Verastem, Inc. (A development stage company)Table of Contents UNITED STATESSECURITIES AND EXCHANGE COMMISSIONWASHINGTON, D.C. 20549FORM 10-KCommission file number 001-35403Verastem, Inc.(Exact name of registrant as specified in its charter)Delaware(State or other jurisdiction ofincorporation or organization) 27-3269467(I.R.S. EmployerIdentification No.)215 First Street, Suite 440Cambridge, Massachusetts(Address of principal executiveoffices) 02142(Zip Code)Registrant's telephone number, including area code: (617) 252-9300 Securities registered pursuant to Section 12(b) of the Act:Title of each class Name of each exchange on whichregisteredCommon Stock, $0.0001 par value NASDAQ Global Market Securities registered pursuant to Section 12(g) of the Act: None Indicate by check mark if the registrant is a well-known seasoned issuer, as defined in Rule 405 of the Securities Act. o Yes  No(Mark One)  ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the fiscal year ended December 31, 2013oro TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934For the transition period from to  Indicate by check mark if the registrant is not required to file reports pursuant to Section 13 or Section 15(d) of the Act. o Yes  No Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to suchfiling requirements for the past 90 days.  Yes o No Indicate by check mark whether the registrant has submitted electronically and posted on its corporate Website, if any, every Interactive Data Filerequired to be submitted and posted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for suchshorter period that the registrant was required to submit and post such files).  Yes o No Indicate by check mark if disclosure of delinquent filers pursuant to Item 405 of Regulation S-K is not contained herein, and will not becontained, to the best of registrant's knowledge, in definitive proxy or information statements incorporated by reference in Part III of this Form 10-K orany amendment to this Form 10-K.  Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, or a smaller reportingcompany. See definitions of "large accelerated filer," "accelerated filer" and "smaller reporting company" in Rule 12b-2 of the Exchange Act. (Checkone): Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). o Yes  No Aggregate market value of the voting and non-voting common equity held by non-affiliates of the registrant as of June 28, 2013 was$203,917,000. The number of shares outstanding of the registrant's common stock as of February 28, 2014 was 25,822,939.DOCUMENTS INCORPORATED BY REFERENCE Portions of our definitive proxy statement to be delivered to stockholders in connection with the 2014 Annual Meeting of Stockholders areincorporated by reference into Part III of this Annual Report on Form 10-K. Large accelerated filer o Accelerated filer  Non-accelerated filer o(Do not check if asmaller reporting company) Smaller reporting company oTable of ContentsTABLE OF CONTENTS 2PART I Item 1. Business 4 Item 1A. Risk Factors 46 Item 1B. Unresolved Staff Comments 73 Item 2. Properties 73 Item 3. Legal Proceedings 73 Item 4. Mine Safety Disclosures 73 PART II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuers Purchases ofEquity Securities 74 Item 6. Selected Financial Data 76 Item 7. Management's Discussion and Analysis of Financial Condition and Results of Operations 77 Item 7A. Quantitative and Qualitative Disclosures About Market Risk 87 Item 8. Financial Statements and Supplementary Data 88 Item 9. Changes in and Disagreements With Accountants on Accounting and Financial Disclosure 88 Item 9A. Controls and Procedures 88 Item 9B. Other Information 89 PART III Item 10. Directors, Executive Officers and Corporate Governance 90 Item 11. Executive Compensation 90 Item 12. Security Ownership of Certain Beneficial Owners and Management and Related StockholderMatters 90 Item 13. Certain Relationships and Related Transactions, and Director Independence 90 Item 14. Principal Accountant Fees and Services 90 PART IV Item 15. Exhibits and Financial Statement Schedules 91 SIGNATURES 92 Table of ContentsFORWARD-LOOKING STATEMENTS This Annual Report on Form 10-K contains forward-looking statements that involve substantial risks and uncertainties. All statements, other thanstatements related to present facts or current conditions or of historical facts, contained in this Annual Report on Form 10-K, including statementsregarding our strategy, future operations, future financial position, future revenues, projected costs, prospects, plans and objectives of management, areforward looking statements. Such statements relate to, among other things, the development of our compounds and diagnostics programs, the timelinefor clinical development and regulatory approval of our compounds, the expected timing for the reporting of data from ongoing trials, the structure ofour planned or pending clinical trials and our ability to fund operations. The words "anticipate," "believe," "estimate," "expect," "intend," "may," "plan,""predict," "project," "target," "potential," "will," "would," "could," "should," "continue" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements are not guarantees of future performance and our actual results could differ materially from the results discussed in theforward-looking statements. Factors that could cause actual results to differ materially from those in the forward-looking statements include, but are notlimited to, our ability to raise additional capital to support our clinical development programs and other operations, our ability to develop products ofcommercial value and to identify, discover and obtain rights to additional potential product candidates, our ability to protect and maintain our intellectualproperty and the ability of our licensors to obtain and maintain patent protection for the technology or products that we license from them, the outcomeof research and development activities, the fact that the preclinical and clinical testing of our compounds and preliminary data from clinical trials may notbe predictive of the success of ongoing or later clinical trials, that data may not be available when we expect it to be, our reliance on third-parties,competitive developments, the effect of current and future legislation and regulation and regulatory actions, as well as other risks described in thisAnnual Report on Form 10-K and other filings with the Securities and Exchange Commission, or SEC. As a result of these and other factors, we may not actually achieve the plans, intentions or expectations disclosed in our forward-lookingstatements, and you should not place undue reliance on our forward-looking statements. Our forward-looking statements do not reflect the potentialimpact of any future acquisitions, mergers, dispositions, joint ventures or investments we may make. We do not assume any obligation to update anyforward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.3Table of ContentsPART I Item 1. Business OVERVIEW We are a biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Acancer stem cell is a particularly aggressive type of tumor cell, resistant to conventional cancer therapy, that we believe is an underlying cause of tumors,their recurrence and metastasis. We have proprietary technology to create a stable population of cancer stem cells that we use to screen for and identifysmall molecule compounds that target cancer stem cells. Our most advanced programs target the Focal Adhesion Kinase, or FAK, and the PI3K/mTORsignaling pathways. Our lead FAK inhibitor, VS-6063, has been assigned defactinib as the United States Adopted Name (USAN). We have receivedorphan drug designation for the use of VS-6063 in mesothelioma in the European Union and in the United States. VS-6063 is currently in aregistration-directed trial (COMMAND) in patients with mesothelioma, a Phase 1b trial in combination with weekly paclitaxel for patients with ovariancancer, a Phase 2 study in patients with non-small cell lung cancer and a Phase 1 trial in Japan in patients with solid tumors. In addition to VS-6063,both our FAK inhibitor VS-4718 and our dual mTORC1/2 and PI3K inhibitor VS-5584 are in Phase 1 clinical trials in patients with advanced cancers. Cancer is a group of diseases characterized by uncontrolled growth and spread of abnormal cells. The American Cancer Society estimated that inthe United States in 2013, approximately 1.7 million new cases of cancer would be diagnosed and nearly 600,000 people would die from the disease.Current treatments for cancer include surgery, radiation therapy, chemotherapy, hormone therapy and targeted therapy. According to estimates by theNational Institutes of Health, in the United States in 2010, the direct medical costs of cancer of all types exceeded $100 billion. IMS Health estimatesthat in the United States in 2010, approximately $22 billion was spent on drugs to treat cancer, representing the largest class of drug spending in theUnited States. Despite years of intensive research and clinical use, current treatments often fail to cure cancer. We believe that a key reason for the ultimate failure of many current cancer therapies to achieve a durable clinical response is the presence of cancerstem cells, or CSCs, which are also sometimes referred to as tumor-initiating cells, within tumors. CSCs have been identified in many types of cancer,including mesothelioma, ovarian, breast, pancreatic, colon, brain, lung and leukemia. Following many cancer treatments, the tumor can remain with ahigh percentage of CSCs and become more aggressive and resistant to further treatment. In addition, patients who relapse often develop metastaticdisease in which the cancer spreads to other sites in the body. Tumor metastasis to critical organs is the cause of more than 90% of cancer deaths. Webelieve that it is the drug resistance and ability of CSCs to spread to other sites in the body that may be the root causes of these therapies failing.Accordingly, our mission is to develop drugs targeting CSCs that either in combination with other cancer treatments or alone can target all of the cellscomprising a tumor and, thus, create a durable clinical response. Our most advanced product candidates are VS-6063, VS-4718 and VS-5584. We are currently evaluating these compounds in both preclinical andclinical studies as potential therapies for certain cancers, including mesothelioma, ovarian and lung. We believe that these compounds may be especiallybeneficial as therapeutics in aggressive cancers driven by CSCs that have a poorer prognosis and lower overall survival rate than other types of cancer. An important element of our business strategy is the development and use of proprietary, companion diagnostics in connection with thedevelopment of our therapeutic drug candidates. We plan to use these diagnostics as part of a personalized medicine approach to identify patients that webelieve will benefit most from our therapies. We also believe that our diagnostics may be used to select patients, to monitor patients' progress on therapyand aid physicians' ongoing treatment decisions.4Table of ContentsOUR MANAGEMENT TEAM AND SCIENTIFIC CO-FOUNDERS AND ADVISORS Our experienced management team includes our Executive Chairman and co-founder Christoph Westphal, M.D., Ph.D., our President and ChiefExecutive Officer, Robert Forrester, our Chief Financial Officer, John "Jack" Green, C.P.A, our Chief Medical Officer, Joanna Horobin, M.B., Ch.B.,our Chief Business Officer, Daniel Paterson, our Vice President, Head of Research, Jonathan Pachter, Ph.D, and our General Counsel, Monica Singh. Dr. Westphal has been involved in founding a number of biotechnology companies as chief executive officer, including SirtrisPharmaceuticals, Inc., which was acquired by GlaxoSmithKline plc in 2008, as well as Alnylam Pharmaceuticals, Inc., Momenta Pharmaceuticals, Inc.and Acceleron Pharma Inc. Dr. Westphal also co-founded Alnara Pharmaceuticals, Inc., which was acquired by Eli Lilly and Co. in 2010 andOvaScience, Inc. Mr. Forrester has been the chief executive officer, chief operating officer and chief financial officer of both private and public life sciencecompanies, including Forma Therapeutics, Inc., CombinatoRx, Inc., now Zalicus Inc., and Coley Pharmaceutical Group, Inc., which was acquired byPfizer Inc. in 2007. Mr. Green was the Senior Vice President and Chief Financial Officer of GTC Biotherapeutics (formerly Genzyme Transgenics Corporation),which was spun out from Genzyme Corporation as a stand-alone public company. Mr. Green is a Certified Public Accountant (CPA) with over30 years of financial experience, including 20 within the biotechnology industry. Dr. Horobin has 30 years of pharmaceutical drug development experience in senior development and executive roles in both public and privatecompanies. Dr. Horobin previously held positions as President and Chief Executive Officer at Syndax Pharmaceuticals, Vice President, Oncology atRhone-Poulenc Rorer (now Sanofi,), Chief Operating Officer of CombinatoRx (now Zalicus) and Executive Vice President at EntreMed withexperience spanning clinical development, marketing, and general management in both public and private companies. Dr. Horobin has been involved inthe development, marketing and commercial launch of 10 approved products. Dr. Horobin received her medical degree from the University ofManchester. Mr. Paterson has over 20 years of experience in management roles at healthcare and biotechnology companies, including as chief executive officerand chief operating officer, and specific expertise in oncology drug and diagnostic product development. Mr. Paterson was Head of Global Strategy forSpecialty Market and Patient-Level Data at IMS Health after playing a key role in the acquisition of PharMetrics by IMS Health as VP, Marketing andCorporate Development. Dr. Pachter has over 20 years of experience in leading the discovery and translational research of small molecule and monoclonal antibodytherapeutics for the treatment of cancer, most recently as the Senior Director of Cancer Biology at OSI Pharmaceuticals Inc., which was acquired byAstellas Pharma Inc. in 2010. Dr. Pachter is an author of over 40 peer-reviewed publications and inventor on numerous patents. Dr. Pachter did hispostdoctoral work in Pharmacology at Yale University School of Medicine and he holds a Ph.D. from Baylor College of Medicine. Ms. Singh has served as our General Counsel since March 2014. Prior to joining the Company, Ms. Singh was a member of the securities andpublic company group at Ropes & Gray LLP where she advised on corporate transactions involving public companies, investment banks and privateequity funds and provided counsel on securities law compliance and corporate governance matters. Ms. Singh holds a B.A. from Amherst College anda J.D. from Harvard Law School. Our scientific co-founders are recognized leaders in the field of cancer biology. Robert Weinberg, Ph.D., Founding Member of the WhiteheadInstitute and Professor of Biology at MIT, has played a key role in identifying the genetic basis of cancer. Dr. Weinberg discovered the first tumoroncogene, the5Table of Contentsfirst tumor suppressor gene, the role of a protein related to the cell surface receptor HER2 in preclinical studies and the mechanisms underlying theformation of CSCs. Eric Lander, Ph.D., Founding Director of the Broad Institute, Professor of Biology at MIT and Professor of Systems Biology atHarvard Medical School, played a central role in the Human Genome Project. Collaborative research in the labs of Dr. Lander and Dr. Weinbergdeveloped our proprietary technology for use in the identification of drugs targeting CSCs and a genetic expression signature, useful as a biomarker, tomonitor the effect of treatment. Our management team is supported by our scientific advisory board comprised of leading academic and industry scientists. Our scientific advisoryboard consists of:6Scientific advisory board Robert Weinberg, Ph.D.Scientific co-founder and chair Founding Member of the Whitehead Institute for Biomedical Research,Professor of Biology at the Massachusetts Institute of Technology andrecipient of the 1997 National Medal of ScienceEric Lander, Ph.D.Scientific co-founder Founding Director of the Broad Institute, Professor of Biology at theMassachusetts Institute of Technology and Professor of Systems Biology atHarvard Medical SchoolJose Baselga, M.D., Ph.D.Senior Medical Advisor Physician in Chief at Memorial Sloan Kettering Cancer Center. Formerly theBruce A. Chabner Chair and Chief of the Division of Hematology/Oncologyat Massachusetts General Hospital and associate director of the MGH CancerCenter.George Daley, M.D., Ph.D. Professor of Hematology and Oncology and Director of the Stem CellTransplantation Program at Children's Hospital and Professor of BiologicalChemistry and Molecular Pharmacology at Harvard Medical SchoolPeter Elliott, Ph.D. Former Senior Vice President and Head of Research and Development ofSirtris Pharmaceuticals, Inc., former Vice President of Pharmacology andDrug Development of Millennium Pharmaceuticals, Inc. and co-developer ofVelcadeDaniel Haber, M.D., Ph.D. Director of the Massachusetts General Hospital Cancer Center and Professorof Medicine at Harvard Medical SchoolRichard Sackler, M.D. Co-chairman, Purdue Pharma and Adjunct Professor of Genetics atRockefeller UniversityJoseph (Yossi) Schlessinger, Ph.D. Chairman and Professor in the Department of Pharmacology at Yale Schoolof MedicinePhillip A. Sharp, Ph.D. Institute Professor at the David H. Koch Institute for Integrative CancerResearch at the Massachusetts Institute of Technology and recipient of the1993 Nobel Prize in Medicine and PhysiologyRoger Tung, Ph.D. President and Chief Executive Officer of Concert Pharmaceuticals, Inc.,former Vice President of Drug Discovery of Vertex Pharmaceuticals, Inc.and co-inventor of Lexiva and AgeneraseChristopher Walsh, Ph.D. Hamilton Kuhn Professor in the Department of Biological Chemistry andMolecular Pharmacology at Harvard Medical SchoolTable of ContentsTHE PROBLEM The cancer death rate in the United States has only decreased modestly since the early 1990s. Cancer remains one of the world's most serioushealth problems and is the second most common cause of death in the United States after heart disease. The American Cancer Society estimated that inthe United States in 2013, approximately 1.7 million new cases of cancer would be diagnosed and nearly 600,000 people would die from the disease.According to estimates by the National Institutes of Health, in the United States in 2010, the direct medical cost of cancer of all types exceeded$100 billion and the cancer type responsible for the highest individual disease costs was breast cancer at $16.5 billion. The following table sets forth theU.S. annual incidence, based on 2013 estimates from the American Cancer Society, and the prevalence, or the number of people in the United Stateswho have been previously diagnosed with cancer, based on 2010 estimates from the National Cancer Institute, for select cancers in which CSCs havebeen implicated. For tumors that have not yet metastasized and remain localized to the site of original tumor formation, current treatments for cancer can be effectivein initially reducing tumor burden. However, for many forms of cancer, current treatments lack sufficient efficacy to achieve a durable clinical response.Following initial treatment, the tumor may recur at the same site or metastasize and spread to other sites in the body. The vast majority of patients whosuccumb to cancer are killed by tumors that have metastasized. This is illustrated by the information in the following table, which shows, according tothe National Cancer Institute's SEER Cancer Statistics Review, 2001-2007, the reduction in five-year survival rate for breast cancer patients based onthe stage of the disease at the time at which the disease is diagnosed. The percentage of patients diagnosed at each stage of disease, referred to as stagedistribution, is included below for comparative purposes. With the application of new technologies and key discoveries, we believe that we are now entering an era of cancer research characterized by amore sophisticated understanding of the biology of cancer.7Scientific advisory board Max Wicha, M.D. Director, University of Michigan Comprehensive Cancer Center andDistinguished Professor of Oncology at the University of MichiganEric Winer, M.D. Director of the Breast Oncology Center at the Dana Farber Cancer Instituteand Professor of Medicine at Harvard Medical SchoolCancer type U.S. annual incidence U.S. prevalence Breast 234,580 2,645,621 Lung and bronchus 228,190 373,489 Colorectal 142,820 1,110,077 Leukemia 48,610 253,350 Pancreatic 45,220 34,657 Brain and other nervous system cancers 23,130 128,193 Breast cancer stage at diagnosis Stagedistribution(1) Five-year relativesurvival rate Localized (confined to primary site) 60% 98.6%Regional (spread to regional lymph nodes) 33% 83.8%Distant (cancer has metastasized) 5% 23.4%(1)2% of breast cancer cases were designated as unknown stage.Table of ContentsWe believe that the discovery of CSCs and the role that they play in cancer development are important new insights that present the opportunity todevelop more effective treatments.Cancer stem cells We believe that CSCs, which are sometimes referred to as tumor-initiating cells, are responsible for the initiation, metastasis and recurrence ofmany cancers. CSCs have the ability to:•move freely and proliferate without attachment to other cells or surfaces; •initiate a tumor; •self-renew; •produce other cancer cell types; and •resist many current cancer treatments. CSCs are often characterized by a distinctive set of biomarkers, which we believe may be a key to identifying patients with tumors that are likely torespond to therapies targeting CSCs. CSCs may be more resistant to current cancer treatments than other types of cancer cells. Thus, as illustrated in the figure below, while currenttreatments may succeed at initially decreasing tumor burden, they may leave behind a population of CSCs that can regenerate tumors. Therefore, thepresence of a mixture of CSCs and other types of cancer cells within a tumor may necessitate a therapeutic approach combining drugs that can kill bothcell populations. The need to target CSCs may apply across the treatment of a broad range of cancers. CSCs have been isolated and characterized from many typesof cancer, including mesothelioma, ovarian, breast, pancreatic, colon, brain, lung and leukemia. The CSCs isolated from each of these tumor types havebeen found to confer greater tumor-forming capability when transplanted into mice than other types of cancer cells from the same tumor. Several specific signaling pathways have been implicated in CSC biology. FAK is a central signaling node in the development of CSCs and criticalfor the establishment of metastases. In 2012, research conducted in Dr. Weinberg's laboratory and published in Cancer Discovery elucidated the role ofFAK in breast cancer disease progression. This work found that increased FAK activity leads to increases in the metastatic capability of cancer cellsfollowing the process of epithelial to mesenchymal transition, an underlying mechanism in CSC development. Similarly, the role of PI3K/mTORsignaling has also been linked to disease progression and the survival of CSCs in multiple types of solid tumors and leukemia. An increase in the proportion of CSCs in response to chemotherapy has been observed in clinical studies. For example, at the Baylor College ofMedicine, breast cancer biopsies were taken from patients at the time of initial diagnosis and again following 12 weeks of treatment with docetaxel, astandard cancer chemotherapy widely used to treat breast cancer. The biopsies taken after 12 weeks of treatment showed increased expression ofbiomarkers for CSCs and an increased number of chemoresistant cells as compared to biopsies taken at the time of initial diagnosis. This result indicates8Table of Contentsthat the CSC component of the tumor was relatively resistant to the chemotherapy. Moreover, it supports our belief that a combination of treatments thatcan effectively target both CSCs and other types of cancer cells is critical to create a durable clinical response.OUR SOLUTION Our solution is to discover and develop a next generation of oncology therapeutics targeting CSCs along with companion diagnostics. We believethat by developing therapeutics that target CSCs we can address the problem of cancer recurrence and metastasis. To achieve a durable clinical response,we believe that it may be necessary to kill both CSCs and other types of cancer cells in a tumor, as illustrated in the figure below, either with acombination of current cancer treatments and CSC-targeted drugs or a single therapeutic found to target both cancer cell populations.Our proprietary technology A persistent problem in the discovery of drugs targeting CSCs is the difficulty of isolating large numbers of CSCs. Without such large numbers,the discovery of drugs targeting CSCs using high-throughput screening is extremely difficult. Moreover, when CSCs are isolated, they typically do notremain stable in culture. Instead, over a short period of time, CSCs convert into other types of cancer cells. To address this problem, our scientific co-founders developed proprietary technology to create a stable population of CSCs that are suitable for use in high-throughput screening of smallmolecule compounds. These stable CSCs are similar to natural CSCs in that they are drug resistant and capable of initiating new tumors. We andscientists at the Whitehead Institute and the Broad Institute have used our technology and high-throughput screening methods to evaluate the ability ofover 300,000 compounds to kill CSCs.Biomarkers and diagnostics Because of the high level of toxicity of traditional chemotherapies and the variability in response of tumors to these treatments, it is criticallyimportant to get the right cancer drug to the right patient. As a result, the oncology field has been at the forefront of developing diagnostics to selectpatients who may benefit from specific therapies, which is sometimes referred to as personalized medicine. We are developing diagnostics designed toenhance our ability to deliver the right drug to the right patient. We believe that our diagnostics will enable us to identify patients that are the most likely to benefit from our drug candidates. By screening toidentify these patients, we expect to be able to select appropriate patients for enrollment in our clinical trials and ultimately, if we obtain marketingapproval, patients who are likely to respond to our therapies. We also plan to use these diagnostics to measure the selective killing of CSCs by ourproduct candidates as one of the ways of determining their efficacy. We expect that our use of proprietary diagnostics may accelerate the clinical development process. We believe that use of these diagnostics mayprovide early, objective signals of drug activity to guide us9Table of Contentsto optimal dosing and the sequencing of agents more quickly. We also believe that this approach may ultimately enable physicians to identify patientswho are likely to benefit most from these therapies and make better clinical decisions during therapy.We are working on companion diagnostics for ourtherapeutic programs based on both in-licensed and internally developed technology and science.OUR STRATEGY We believe that a key reason for the failure of many current cancer treatments is that they fail to kill CSCs, which we believe are responsible for theinitiation, metastasis and recurrence of many cancers. Our goal is to build a leading biopharmaceutical company focused on the discovery, developmentand, ultimately, commercialization of novel drugs and companion diagnostics targeting CSCs. Key elements of our strategy to achieve this goal are:•Execute on the registration-directed trial of VS-6063 in mesothelioma named COMMAND (Control of Mesothelioma withMAinteNance Defactinib). We have met with the regulatory agencies in the United States and the United Kingdom and interacted withthe regulatory agencies in ten other countries. Based on these discussions, we believe that positive results from this trial, if obtained, mayenable us to seek regulatory approval for VS-6063 in mesothelioma. •Rapidly advance our product candidates through clinical development. We have initiated clinical trials of VS-6063 both as a single agentand in combination with the standard chemotherapy paclitaxel and are conducting additional clinical trials with VS-4718 and VS-5584.We expect to initiate further clinical trials over the next several years. •Expand the indications in which our product candidates may be used. In parallel to the mesothelioma, ovarian and non-small cell lungcancer trials that we are currently conducting, we plan to pursue additional disease indications as we believe CSCs are implicated inmany types cancers, including breast, pancreatic, colon, brain, lung and leukemia. •Develop diagnostics for therapeutic products targeting CSCs. We plan to develop companion diagnostic products to support ourtherapeutic product candidates. We believe that use of these diagnostics may aid in the selection of patients for enrollment in our clinicaltrials and, if we obtain marketing approval, patients who are most likely to benefit from therapy with our drugs. We also believe thatthese diagnostics may be used to monitor patients' progress on therapy and aid physicians' ongoing treatment decisions. •Collaborate selectively to augment and accelerate translational research, development and commercialization. We may seek third-partycollaborators for the development and eventual commercialization of our product candidates. In particular, we may enter into third-partyarrangements for target oncology indications in which our potential collaborator has particular expertise or for which we need access toadditional research, development or commercialization resources. •Consider acquiring or in-licensing rights to additional compounds. We may pursue the acquisition or in-license from third parties ofrights to additional compounds that target CSCs. We believe that our approach of identifying CSC targeting product candidates fromexternal sources at various stages of development to supplement our internal programs may allow us to initiate clinical development of adiverse pipeline of compounds more quickly than if we were to focus solely on internally developed candidates. •Maintain scientific leadership in the CSC field. We plan to continue to conduct research in the CSC field to further our understanding ofthe underlying biology of cancer progression and metastasis. We also plan to continue fostering relationships with top scientificadvisors, researchers and physicians. We believe that exceptional advisors, employees and management are critical to leadership in theCSC field.10Table of ContentsOUR PRODUCT CANDIDATESOverview Using our proprietary technology and high-throughput screening methods, we are evaluating compounds for their activity against CSCs in a waythat we believe has not been previously possible. We are focused on the discovery and development of small molecules to allow flexibility in the designof molecules for optimized efficacy and safety primarily as orally available drug candidates. We have three product candidates currently in clinical trials including the focal adhesion kinase (FAK) inhibitors VS-6063 and VS-4718, and thedual mTORC1/2 and PI3K inhibitor VS-5584. We are running clinical trials in cancers where cancer stem cells are implicated in disease progression,including mesothelioma, ovarian, non-small cell lung and other advanced cancers. We intend to incorporate patient selection markers and CSC-specific biomarkers into companion diagnostics for our product candidates for use inidentifying patients who are likely to benefit from treatment. We may use this information to aid in the selection of patients for late stage clinical trials.For example, we may use a clinical test for the expression status of the protein merlin in patients with mesothelioma in our COMMAND trial. We alsoplan to utilize diagnostics to measure the effect that our product candidates have on CSCs in a tumor. Conventional chemotherapy works by stopping the function of cancer cells through a variety of mechanisms. Chemotherapies are usually nottargeted at any specific differences between cancer cells and normal cells. Rather, they kill cancer cells because cancer cells generally grow more rapidlythan normal cells and, as a result, are relatively more affected by the chemotherapy than normal cells. Because CSCs exhibit mechanisms of resistance,including a slower rate of growth than other cancer cells, they are often not susceptible to conventional chemotherapy. As a result, the treatments maysucceed at initially decreasing tumor burden but ultimately fail to kill the CSCs. To enhance therapeutic benefit, we may use our product candidates in combination with existing therapies in an effort to target both CSCs andother types of cancer cells. This approach is being utilized in our ongoing ovarian cancer trial of VS-6063 in combination with paclitaxel. A secondapproach to using CSC-targeting drugs in combination with chemotherapy is based upon the timing (or sequencing) of drug treatment. We believe thatusing our CSC-targeting drugs immediately following chemotherapeutic treatments (known as maintenance therapy) may complement the overall effectof treatment by eliminating the residual CSCs following chemotherapeutic treatment. We are using this approach in patients with mesothelioma in ourCOMMAND trial for VS-6063. We are developing our product candidates for the treatment of multiple cancer types, initially mesothelioma, ovarian and non-small cell lung cancer,and in the future, other cancers driven by CSCs. We believe that our product candidates target CSCs that have been implicated in aggressive cancers,metastasis and chemotherapeutic resistance.MESOTHELIOMAOverview Malignant mesothelioma is a malignant tumor of mesothelial cells which make up the pleura, or tissue lining, of many internal organs.Mesothelioma most commonly occurs in the pleura surrounding the lung, but occasionally involving the peritoneum surrounding internal organs of thedigestive tract, the lining of the heart or the lining of the testis. Asbestos fibers are the cause of most cases of mesothelioma. The proportion of men with mesothelioma directly attributable to occupationalasbestos exposure is approximately 85%. However, mesothelioma can also result from para-occupational exposures (for example, people who havelaundered contaminated clothing) and non-industrial environmental exposures. The latency period11Table of Contentsbetween first exposure to asbestos and death from mesothelioma is typically very long (20 to more than 40 years). Mesothelioma is a relatively rare disease; it is estimated that the prevalence within the United States is approximately 2,500-3,000 cases per yearand in the European Union it is approximately 3,500-4,000 cases per year. The World Health Organization estimates that there are approximately 59,000cases of mesothelioma each year worldwide. A large majority of mesothelioma patients present with the pleural form around the lungs. Typically, symptoms are chest pain, shortness of breath,or both. The pain is usually dull, diffuse and characteristically worsens during the course of the illness. The pain may be described as heaviness oraching in the shoulder, arm, chest wall and upper abdomen. A 2011 study published in Oncogene reported that 90% of mesothelioma patient samples contained CSCs and implicated these cells in thedevelopment of drug resistance. In addition, the treatment of mesothelioma cells with Alimta led to an increase in the tumor-initiating capability of thedrug resistant cells, as compared to those treated with placebo control. A recent report published in the journal Cancer Research describes that the dualloss of merlin and p16 in mesothelioma leads to highly aggressive tumors enriched in cancer stem cells.Biomarkers Neurofibromatosis 2 (NF2) is a tumor suppressor gene that encodes the protein merlin. Inactivation of NF2 by mutation and/or deletion, leading toa reduction in the merlin protein, occurs in approximately 50% of malignant mesotheliomas. Merlin has been demonstrated to play roles in cell adhesion,invasion and cell motility in tumor cell lines partially through regulation of FAK. In turn, FAK mediates signal transduction by integrins and growthfactor receptors. Increased activation of FAK has been demonstrated in merlin-low mesothelioma cells, indicating that FAK may represent an importanttherapeutic target for malignant mesothelioma. We are developing a diagnostic for merlin to aid the use of VS-6063 in mesothelioma. We believe thepatients with tumors that are merlin-low may respond better to treatment with VS-6063.Current Treatment Malignant mesothelioma carries a poor prognosis with a median survival of approximately 12 months from diagnosis. Mesothelioma is currentlytreated with a combination of surgery, radiotherapy and chemotherapy. The only approved drug treatment regimen for mesothelioma is Alimta incombination with carboplatinum or cisplatinum, referred to here collectively as cisplatin. A study by Vogelzang in 2003 reported that the mediansurvival of patients treated with the combination of Alimta and cisplatin was 12.1 months as compared to the median survival of patients treated withcisplatin alone, which was 9.3 months. Following this first-line treatment, the duration of which is often limited by toxicity, patients are monitored butdo not receive further therapy until disease progression. As reported by Buikhuisen et al., 2013, the median time to disease progression (progressionfree survival, or PFS) is approximately 4 months. Once progression occurs, there is no approved standard of12Table of Contentscare. As reported by Krug et al., in 2011 in an evaluation of 332 patients, this second-line setting has a median PFS of 6 weeks.OVARIAN CANCEROverview Ovarian cancer forms in tissues of the ovary, one of a pair of female reproductive glands in which the ova, or eggs, are formed. Most ovariancancers are either ovarian epithelial carcinoma, cancer that begins in the cells on the surface of the ovary, or malignant germ cell tumors that begin in eggcells. According to the National Cancer Institute, epithelial carcinoma of the ovary is one of the most common gynecologic malignancies and the fifthmost frequent cause of cancer death in women, with 50% of all cases occurring in women older than 65 years. The National Cancer Institute estimated that in January 2009 there were approximately 182,000 women in the United States with a history ofovarian cancer and in 2013 approximately 22,000 new cases of ovarian cancer will be diagnosed and approximately 14,000 women will die from thedisease.Prognosis For patients with ovarian cancer, the most important prognostic factor is stage of the disease. Unfortunately, most patients with ovarian cancer havewidespread disease at diagnosis. This may be partly explained by relatively early spread to the rest of the abdominal cavity. General symptoms such asabdominal pain and swelling, gastrointestinal symptoms, and pelvic pain often go unrecognized, leading to delays in diagnosis. This is illustrated by theinformation in the following table, which shows, according to the National Cancer Institute's SEER Cancer Statistics Review, 2002-2008, the reductionin five-year survival rate for ovarian cancer patients based on the stage of the disease at the time at which the disease is diagnosed. The percentage ofpatients diagnosed at each stage of disease, referred to as stage distribution, is included below for comparative purposes.Current Treatment Most patients are treated with a combination of surgery, chemotherapy and radiation therapy. Surgery is often comprehensive to remove as muchof the tumor as possible and may include removal of the ovaries or a total hysterectomy where the uterus is also removed.13Stage at Diagnosis StageDistribution (%) 5-yearRelative Survival (%) Localized (confined to primary site) 15 91.5 Regional (spread to regional lymph nodes) 17 71.9 Distant (cancer has metastasized) 61 26.9 Unknown (unstaged) 7 22.0 Table of Contents Unfortunately, chemotherapy is rarely curative in the treatment of ovarian cancer and many tumors become resistant to platinum-basedchemotherapy, which is the primary treatment regimen. Further therapy with conventional chemotherapy is generally palliative, not curative, as thetumor is able to metastasize and spread to other sites in the body. A report by McNeish et al., at the 2013 American Society of Clinical Oncologyreported that the use of paclitaxel in patients that had previously failed platinum-based chemotherapy resulted in less than 1% of patients achieving acomplete response and an overall response rate of approximately 12% as defined by Response Evaluation Criteria In Solid Tumors (RECIST)guidelines. We believe it is the presence of CSCs that lead to this resistance and eventual disease progression.NON-SMALL CELL LUNG CANCEROverview According to the National Cancer Institute, the most common types of non-small cell lung cancer (NSCLC) are squamous cell carcinoma, large cellcarcinoma, and adenocarcinoma. Although NSCLCs are associated with cigarette smoke, adenocarcinomas may be found in patients who have neversmoked. As a class, NSCLCs are relatively insensitive to chemotherapy and radiation therapy compared with small cell lung cancer (SCLC). TheNational Cancer Institute estimates that in 2013 there were 228,190 new cases of lung cancer (both NSCLC and SCLC) in the United States and159,480 deaths. Lung cancer is the leading cause of cancer-related mortality in the United States. The 5-year relative survival rate from 1995 to 2001 forpatients with lung cancer was 15.7% A study published in Cancer Discovery in 2013 described the role of genetic mutations in several genes that drive carcinogenicity in NSCLCincluding Kras, p16 and p53. The researchers published data from preclinical models demonstrating that tumors that harbor both a Kras mutation inaddition to a mutation in either p16 or p53 are particularly sensitive, as determined by overall survival, to treatment with a FAK inhibitor (VS-6062; anearly compound in the same chemical series as VS-6063).Current treatment of NSCLC Patients with resectable disease may be cured by surgery or surgery followed by chemotherapy. Local control can be achieved with radiationtherapy in a large number of patients with unresectable disease, but cure is seen only in a small number of patients. Patients with locally advancedunresectable disease may achieve long-term survival with radiation therapy combined with chemotherapy. Patients with advanced metastatic disease mayachieve improved survival and palliation of symptoms with14Table of Contentschemotherapy, targeted agents, and other supportive measures. The disease becomes resistant to therapy and returns in the vast majority of patients. Webelieve it is the presence of CSCs that lead to this resistance and eventual disease progression.BREAST CANCEROverview The National Cancer Institute estimated that in January 2008 there were approximately 2.6 million women in the United States with a history ofbreast cancer. Breast cancer is currently the second most frequently diagnosed and the second most deadly cancer among women in the United States.The American Cancer Society estimated that in the United States in 2011, approximately 230,500 new cases of invasive breast cancer would bediagnosed in women and approximately 39,500 women would die from the disease. Breast cancers can be segregated into subtypes based upon the positive presence of three protein receptors:•estrogen receptor, or ER; •progesterone receptor, or PR; and •human epidermal growth factor receptor 2, or HER2. Triple negative breast cancer, or TNBC, is a type of breast cancer that does not express any of these three receptors. According to results from apopulation-based study of the California Cancer Registry published by the American Cancer Society in 2007, approximately 15% of all breast cancerswere classified as TNBC. In comparison with other breast cancers, TNBC tends to grow faster and has a higher rate of metastases. Furthermore, TNBCtends to recur more often than other subtypes of breast cancer. Patients with TNBC generally have a poorer prognosis and lower overall survival ratethan patients with breast cancers that are positive for the hormone receptors ER and PR. We believe that the natural disease progression of TNBC exhibits the key hallmarks of CSCs. Specifically, we believe that:•TNBC is initially responsive to chemotherapy because chemotherapy kills the majority of cancer cells, but not the CSCs. •TNBC returns more often than other types of breast cancer in part because there are CSCs that are not killed by current cancertreatments. •The site of recurrence is often at another place in the body as compared to the original tumor because the CSCs which are not killed areable to metastasize. •The recurring tumor may be resistant to therapy because it contains a high percentage of CSCs.Current treatment of breast cancer Surgery, radiation therapy, targeted therapy, hormone therapy and combinations of conventional chemotherapy are often used to treat breast cancer.However, these therapies carry significant side effects and frequently do not result in a durable clinical response, especially for patients with TNBC. The choice of cancer drugs used to treat breast cancer is guided by clinical classification of the tumor as ER positive or negative, PR positive ornegative and HER2 positive or negative. The presence, absence or combination of these biomarkers in patient tumors informs the selection of prescribeddrugs, which include the anti-estrogen therapies Tamoxifen and aromatase inhibitors, as well as agents that directly target HER2, such as Herceptin,Tykerb and Kadcyla. These treatments may slow or stop cancer growth and are currently considered the most successful treatments for breast15Table of Contentscancer. However, because TNBC patients are negative for ER, PR and HER2, the treatment options for these patients are limited. In particular, thetargeted therapies, including Herceptin, Tykerb, Kadcyla and anti-estrogen treatments, are not effective for these patients. For example, in a studyconducted at Baylor College of Medicine, in which biopsies were taken from breast cancer patients both before and after conventional chemotherapytreatment, the percentage of CSCs increased over the 12-week treatment period, indicating the survival of these cells. If tumors recur, which happens more often in TNBC than other breast cancers, further therapy with conventional chemotherapy is generallypalliative, not curative, as the CSCs are able to metastasize and spread to other sites in the body.FAK Inhibition ProgramOverview FAK expression is greater in many tumor types compared to normal tissue, particularly in cancers that have a high invasive and metastaticcapability. The contact between cancer cells and connective tissue stimulates FAK signaling. However, CSCs acquire the ability to survive in theabsence of contact with connective tissue. We believe that FAK signaling in CSCs may be maintained through alternative mechanisms, thus providingCSCs the ability to survive in the absence of cell contact. Accordingly, we believe that FAK signaling may be a central component of CSC biology thatallows CSCs to survive after exiting from a tumor mass and enable metastatic growth at other sites in the body. In 2012, our scientific co-founder Robert Weinberg reported in Cancer Discovery that FAK is a critical regulator of CSCs and inhibition of thissignaling pathway prevented disease progression and initiation of new metastatic lesions. An additional study by Dr. Weinberg reported in PNAS in2009, demonstrated that in a mouse model of breast cancer FAK signaling was required to enable lung metastasis. Non-CSCs, which lack the ability toincrease their FAK signaling activity through alternative mechanisms, remained non-metastatic in this model and did not survive dissemination to thelungs. Researchers at McGill University reported in PNAS that in a genetically modified mouse model the specific deletion of FAK from the mammarycells prevented primary tumor formation and metastasis. Similarly, researchers at the University of Michigan demonstrated that specific deletion of FAKfrom mammary tumors prevented tumor initiating capacity in recipient mice. Scientific research suggests that increased FAK expression and activity is associated with metastatic progression and poor prognosis in multiplecancer types. For example, a 2009 retrospective study published in the Journal of Clinical Investigation identified the amplification, or increase innumber, of the gene encoding FAK in breast cancers. This gene amplification, and resulting high FAK expression, significantly correlated with theprogression of early stage, primary breast cancer to advanced metastatic disease. In an analysis of 295 breast cancer patients that was part of this study,elevated FAK expression was a marker of poor survival. The correlation of elevated FAK expression with poor survival was more significant than, andindependent of, other commonly used clinical parameters such as hormone receptor status. A similar finding was described in the Journal of ClinicalInvestigation in 2010 where it was reported that in women with ovarian cancer elevated FAK expression is correlated with advanced disease, metastasesand poor prognosis. A comparison of FAK activity in biopsies from16Table of Contentspatients revealed that high FAK activity correlated with a decrease in overall survival, from 3.2 to 1.7 years.VS-6063Overview VS-6063, or defactinib, is an orally-available small molecule compound designed to target cancer stem cells through the inhibition of FAKsignaling. We are currently evaluating VS-6063 as a potential therapy for mesothelioma, ovarian cancer, NSCLC and other solid tumors driven byCSCs. We have initiated clinical trials of VS-6063 in patients with cancer: including the registration-directed COMMAND study in patients withmesothelioma, a Phase 1/1b clinical trial of VS-6063 in combination with paclitaxel for patients with ovarian cancer, a Phase 2 study of VS-6063 inpatients with NSCLC and a Phase 1 study of VS-6063 in Japanese patients with advanced solid tumors. We have received orphan drug designation forVS-6063 in mesothelioma in both the United States and the European Union.Development status of VS-6063 Phase 1 clinical trial in advanced solid tumors. VS-6063 demonstrated a good safety profile and initial signs of clinical activity in a Phase 1 trialin 46 patients with advanced solid tumors. Patients were selected on the basis of a confirmed diagnosis of a non-hematologic malignancy that wasunresponsive to currently available therapies or for which no standard therapy existed. VS-6063 was administered on a twice daily (BID) schedule anddose cohorts initially included a minimum of 3 patients. Dosing was based on flat milligram increments without adjustment for body size, the startingdose being 12.5 mg BID in the initial cohort. Safety, activity and pharmacokinetics, the process by which a drug is absorbed, distributed andmetabolized in the body, were measured.17Table of Contents 16 of the 37 subjects enrolled, or 43%, at doses 100 mg BID (predicted active dose) experienced stable disease as their best response to treatment.The recommended Phase 2 dose based on safety, pharmacokinetics and activity was determined to be 400 mg/BID. Phase 1/1b clinical trial of VS-6063 in combination with paclitaxel in patients with ovarian cancers. Ovarian cancer rapidly develops resistanceto chemotherapy. Following development of resistance to front-line platinum therapy, there is not an approved standard of care although treatment withthe chemotherapeutic paclitaxel is often used. We are currently conducting a clinical trial to evaluate the safety and efficacy of VS-6063 administered incombination with paclitaxel in patients with advanced ovarian cancer. This clinical study is comprised of two parts. Part 1 is a dose escalation of oral VS-6063 twice daily in addition to the standard dose of weeklypaclitaxel. The purpose of part 1 is to assess the safety, evaluate pharmacokinetic parameters and determine the recommended Phase 2 dose incombination with paclitaxel. Part 2 includes patients treated with the recommended Phase 2 dose of VS-6063 in combination with paclitaxel. Thepurpose of part 2 is to evaluate the pharmacodynamic effect, the biochemical and physiological effect of VS-6063 through measurement of biomarkersfrom tumor biopsies pre- and post-treatment and assess the anti-cancer activity of VS-6063 when administered in combination with paclitaxel accordingto RECIST guidelines. We believe this approach will allow us to treat both the non-CSCs and CSCs simultaneously by the combination of chemotherapy (eg. paclitaxel)and a CSC-targeted agent (VS-6063) to hopefully enable a more durable clinical response. We reported data from the dose escalation phase of VS-6063 in combination with paclitaxel at the NCI/AACR/EORTC meeting in 2013. In thisstudy, the addition of VS-6063 to weekly paclitaxel demonstrated a good safety profile with no worsening of the well-known side effects of paclitaxelat either the 200mg/BID or 400 mg/BID dose level of VS-6063 in addition to the standard dose of weekly paclitaxel of 80mg/m2. 400mg/BID is therecommended Phase 2 dose of VS-6063 both as a single agent and in combination with weekly paclitaxel. In addition, initial signs of clinical activitywere observed18Table of Contentsincluding a complete response as determined by RECIST. The patient who achieved a complete response was on drug therapy for a total of 37 weeks. Registration-directed study in mesothelioma (COMMAND). We are conducting a randomized, double-blind, multi-center, multi-national study ofVS-6063 in patients with mesothelioma and at least stable disease post first-line therapy. We have met with the regulatory agencies in the United Statesand the United Kingdom and interacted with the regulatory agencies in ten other countries to discuss COMMAND and, based on these discussions, webelieve that positive results, if obtained, from this trial will enable us to seek regulatory approval for VS-6063 in mesothelioma. The study is currentlyopen and recruiting patients in 8 of 11 countries and 28 of 35-40 planned study centers. In total, we expect to enroll 350-400 patients intoCOMMAND. Even when front line therapy of Alimta plus cisplatin produces an objective response or stable disease, patients are noted to have progression ofdisease within approximately four months of completing therapy. The ability to maintain the responses seen with first line therapy and delay furtherprogression is therefore a critical goal in this disease. We believe it is the presence of chemotherapy-resistant CSCs in the residual tumor that lead toprogression and it is our goal to extend the amount of disease control time for patients through treatment with VS-6063. Researchers hypothesized in 2010 in the International Journal of Oncology that early relapse of mesothelioma and further progression followingAlimta plus cisplatin results from the presence of a relatively small number of CSCs which exhibit resistance to standard therapy. This hypothesis issupported by our experiments with human mesothelioma cell lines which show that chemotherapeutic agents such as Alimta and cisplatin enrich forCSCs. As shown below, in contrast to Alimta, inhibition of FAK by VS-6063 effectively reduces the proportion of CSCs, as determined by thebiomarker19Table of ContentsAldefluor. This suggests that targeting FAK might be expected to reduce the CSC population and overcome the resistance that develops to Alimta andcisplatin in mesothelioma. We believe the sequential dosing of chemotherapy (eg. Alimta plus cisplatin) and a CSC-targeted agent (VS-6063) in the maintenance settingimmediately following front-line therapy will allow us to treat both the non-CSCs and CSCs. In a report at the NCI-AACR-EORTC conference in 2012, in a Phase 1 study of the orally available FAK inhibitor GSK2256098 byGlaxoSmithKline (GSK), a median PFS of 17.7 weeks was observed in 29 patients with recurrent mesothelioma in comparison to the previouslyreported median PFS of just 6.1 weeks in 332 similar patients receiving placebo. In addition, GSK evaluated the response to treatment based upon thestatus of the biomarker merlin. Patients who were deemed to be merlin-low had a median PFS of 24.1 weeks compared with 11.4 weeks for thosepatients whose tumors were deemed to be merlin-high as shown in the figure below.20Table of Contents These observations generally suggest that FAK inhibition in mesothelioma may be helpful to slow disease progression and indicate that use of adiagnostic to identify patients with low levels of the biomarker merlin may predict for those most likely to have the greatest response to treatment with aFAK inhibitor. COMMAND is recruiting patients who have not progressed on at least 4 cycles of Alimta plus cisplatin as their most recent therapy. Immediatelyfollowing completion of the front-line chemotherapy, patients begin to take VS-6063 or placebo by mouth, twice a day. This treatment strategy isknown as switch maintenance therapy and there is no current standard of care recommended for patients with mesothelioma in this setting. The patientsare stratified by their merlin expression, as described below, and within each stratum are then randomized to either VS-6063 or matched placebo. Mesothelioma is a rare disease making it important to derive as much information as possible, with as great a certainty as possible, from eachclinical trial. It is hypothesized that the treatment effect in the subjects with merlin low tumors is likely to be greater than that in the merlin high group.Therefore, our design incorporates a sample size reassessment at a pre-planned interim analysis. The goals of the enrichment design approach are:•To identify if the overall population is benefitting or if the merlin low sub population is responding better to treatment. •To modify patient enrollment and only continue enrolling patients with merlin low tumors if those patients are responding better totreatment. This study design enables an analysis that could support approval in either the full treatable mesothelioma population or the approximately half ofthe patients with merlin low tumors. We believe the use of a companion diagnostic test to select the merlin low patients who may respond better to FAKtreatment in mesothelioma may increase our ability to properly treat this patient population. The key endpoints of the study are overall survival, progression free survival and quality of life. Based on discussions with the regulatory agenciesin the United States and United Kingdom and ten21Table of Contentsother countries, we believe that if VS-6063 significantly increases clinical benefit, it may allow us to seek regulatory approval. Phase 2 study of VS-6063 in patients with non-small cell lung cancer (NSCLC). We are conducting a multi-center study of VS-6063 in patientswith recurrent NSCLC following previous chemotherapy. There are 4 study arms, including patients with a KRAS-mutation, or a KRAS-mutation withaccompanying secondary mutations in p16, p53 or both p16 and p53. The trial is being conducted at 8-10 sites in the US and is expected to enroll up to44 patients (11 per arm) in the first stage of the Simon's two-stage trial design. A Simon's two-stage trial design incorporates a single pre-plannedinterim analysis to determine which enrollment arms are exhibiting a favorable response to treatment and allows for enrollment of an additional 12patients with the same tumor mutation profile per arm. The Phase 2 study is designed to assess the effect of VS-6063 on progression freesurvival,overall response rate, and overall survival.. Phase 1 study of VS-6063 in Japanese patients. We are conducting a Phase 1 open-label, dose-escalation study of VS-6063 at Kinki Universityin Osaka, Japan. This study is designed to assess the safety, pharmacokinetics and pharmacodynamics of single agent VS-6063 in Japanese patientswith advanced solid tumors. The study is expected to enroll 12-18 patients.VS-4718Overview VS-4718 is an orally-available small molecule compound designed to target cancer stem cells through the inhibition of FAK signaling. We areevaluating VS-4718 as a potential therapy for cancers driven by CSCs.Development status of VS-4718 We are currently conducting a Phase 1 clinical trial with VS-4718 in patients with advanced solid tumors. The dose escalation portion of thePhase 1 clinical trial is designed to determine the biologically active dose and maximum tolerated dose. Additional patients will be enrolled to assesssafety, tolerability and to evaluate initial signs of activity.22Table of ContentsPreclinical development We are conducting a comprehensive program to study VS-4718 as a potential treatment for cancers associated with CSCs and increased FAKactivity. Key results to date from preclinical studies of VS-4718 are summarized below. Biochemical and cellular tests. In biochemical testing, VS-4718 inhibited purified FAK and demonstrated in vitro selectivity against a panel of107 different protein kinases. In addition, in various in vitro assessments of drug effects on CSCs, VS-4718 exhibited potent activity and a preferentialeffect, or selectivity, for CSCs as compared to other types of cancer cells. Effect of FAK inhibition in merlin low cell lines and mouse tumor models of mesothelioma. VS-4718 has exhibited in vitro cytotoxic effects onmesothelioma cell lines and tumor growth inhibition in mouse models of mesothelioma. Merlin low cells lines derived from mesothelioma tumors wereobserved to be particularly sensitive to FAK inhibition. VS-4718 was also tested in models in which merlin low mesothelioma cells were implanted intoa mouse and the tumor was allowed to develop. Upon tumor formation, the mice were treated with VS-4718 by oral administration at concentrations of25 or 50 mg/kg of body weight or a placebo control through the end of the experiment. As shown in the figure below, single agent VS-4718 treatmentcaused tumor regression in a dose-dependent fashion in contrast to placebo control. Mouse models of breast cancer. VS-4718 has exhibited tumor growth inhibition and reduction of metastatic burden in several mouse models ofbreast cancer. In one experiment, VS-4718 was tested in a model in which breast cancer cells were implanted into a mouse and the tumor was allowedto develop. Upon tumor formation, the mice were treated with VS-4718 in drinking water at a concentration of 0.5 mg/ml or a placebo control beginningat day 12 through the end of the experiment. As shown in the figure below, the tumor volume in the VS-4718 treatment group was significantly smallerthan in the placebo group from day 27 through the end of the experiment. In23Table of Contentsaddition, at day 70 the weight of the primary tumor and the number of lung metastases in the VS-4718 treatment group were both significantly less thanin the placebo group. The vertical line on each data point in the tumor volume figure above represents the standard deviation from the mean. The box and vertical line foreach data point in the tumor weight and metastases figures above show the distribution of the data. The square data point inside the box represents themean. The bottom of the box represents the 25th percentile, the middle line in the box represents the median and the top of the box represents the75th percentile. The vertical lines projecting from the bottom and top of the box represent the 5th and 95th percentiles.PI3K/mTOR Inhibition ProgramOverview The PI3 kinase/mTOR pathway plays a central role in cancer cell proliferation and survival. Depending on the mode of pathway activation,different PI3K isoforms and mTOR complexes have been shown to play essential roles in oncogenesis and survival of CSCs. There are four Class 1PI3K enzymes, known as alpha, beta, gamma and delta, and two mTOR complexes known as mTORC1 and mTORC2. Studies published in Nature in 2006 reported that genetic activation of the PI3K/mTOR pathway in adult blood cells led to generation of leukemiaCSCs. In addition, multiple studies have shown that PI3K pathway activity contributes to the maintenance of CSCs in breast, lung, and prostate cancersVS-5584Overview VS-5584 is an orally available small molecule that potently and selectively inhibits both mTORC1/2 and PI3K signaling. A 2013 study publishedin Science Translational Medicine described findings that the observed resistance to PI3K-alpha inhibitors in breast cancer is mediated by signalingthrough the mTOR complex. We believe that the dual inhibition of mTORC1/2 and PI3K by VS-5584 may provide a greater clinical benefit for patients.24Table of ContentsDevelopment status of VS-5584 VS-5584 is currently in a Phase 1 clinical trial in patients with advanced cancers. The dose escalation portion of the Phase 1 clinical trial isdesigned to determine the biologically active dose and maximum tolerated dose. Additional patients will be enrolled to assess safety, tolerability and toevaluate initial signs of activity.Preclinical development We are conducting a comprehensive preclinical program to study VS-5584 as a potential treatment for cancer. Key results of this program to dateare summarized below. Biochemical testing. In biochemical testing, VS-5584 was equipotent against all four human Class 1 PI3K isoforms and mTOR kinase, theenzymatic component of both mTORC1 and mTORC2 (IC50 values displayed below in nM). In general, the more potent a drug is, the lower the doserequired for a therapeutic effect. An IC50 value measures the dose at which the activity of the enzyme is reduced by 50%, a commonly used measure ofpotency. VS-5584 has demonstrated a selective effect for these kinases versus a panel of over 400 kinases. In vitro killing of CSCs. In an in vitro test, SUM159 triple negative breast cancer cells were treated with VS-5584 or paclitaxel for 4 days, andthe percentage of Aldefluor-positive CSCs was assessed. VS-5584 dose-dependently decreased the percentage of CSCs, while paclitaxel increased thepercentage of CSCs.LICENSESWhitehead Institute for Biomedical ResearchDrug discovery platform license agreement In October 2010, we entered into an exclusive license agreement with the Whitehead Institute, both on its own behalf and as sole and exclusiveagent of Harvard and MIT, or the drug discovery platform license agreement, which we amended and restated in January 2012. Under the drugdiscovery platform license agreement, we acquired an exclusive, royalty-bearing, worldwide license under patent rights owned by the WhiteheadInstitute, Harvard and MIT to develop, make, use and sell products covered by the licensed patent rights for use in treating cancer, and to develop andperform licensed processes, in each case, for all human therapeutic, prognostic and diagnostic uses.25Table of Contents We are required to use commercially reasonable efforts to develop and commercialize licensed products under the agreement. In particular, we arerequired to fulfill specific development and regulatory milestones by particular dates and, during each calendar year, either spend a specified amount forresearch and development, actively conduct one or more clinical trials for a licensed product or a product identified using a licensed process that doesnot constitute a licensed product, which we refer to as an identified product, prepare, file or pursue a filed application for regulatory approval of alicensed product or an identified product, or launch or sell a licensed product or identified product. Under the agreement, we paid the Whitehead Institute an upfront license fee and reimbursed patent related fees and costs incurred by the WhiteheadInstitute, Harvard and MIT totaling $104,000 in the aggregate and issued 166,664 shares of our common stock to the Whitehead Institute and entitiesand individuals affiliated with the Whitehead Institute. We also agreed to pay the Whitehead Institute annual license maintenance fees, milestone payments, royalties as a percentage of net sales and apercentage of sublicense income that we receive. Annual license maintenance fees are creditable against royalties, which are described below, earnedduring the same calendar year. Milestone payments are triggered upon the achievement of specified development, regulatory and commercializationmilestones and are not creditable against the royalties described below. For each licensed product, we agreed to make milestone payments of up to anaggregate of $1,560,000 plus an additional amount for each subsequent approval of additional indications for a maximum number of licensed products.For each identified product that is not a licensed product, we agreed to make milestone payments of up to an aggregate of $815,000 plus an additionalamount for each subsequent approval of additional indications for a maximum number of identified products. Each type of specified milestone paymentis payable only for each of the maximum number of licensed products and the maximum number of identified products, as the case may be, to achievethe applicable milestone. In addition, a separate milestone payment is due upon the first commercial sale of each licensed product or identified productthat is a diagnostic or prognostic test. A single additional milestone payment is due for the first issuance of licensed patent rights in the United States,the United Kingdom, France, Germany, Spain or Italy. The royalty rate is in the low single digits as a percentage of net sales for licensed products thatare therapeutics, the mid single digits for licensed products that are diagnostics or prognostics and less than one percent for identified products. The Whitehead Institute, Harvard and MIT retain the right to, and may grant licenses to other academic and non-profit institutions for the right to,practice the licensed patent rights for research, teaching and educational purposes. The Whitehead Institute, Harvard, MIT or any such other institutioncould seek to license to third parties any intellectual property rights that it discovers using the licensed patent rights while pursuing these purposes.Under the agreement, we have a right, subject to the Whitehead Institute's obligations under third party research funding agreements, to negotiate alicense for any compounds identified prior to a specified date in the Whitehead Institute's laboratory run by Dr. Weinberg that selectively target CSCsgenerated by induction through the Epithelial to Mesenchymal Transition, or EMT. After a specified period of time, if a third party requests to sublicense the patent rights for a product or process that is not directly competitive withour products or processes, we must enter into good-faith negotiations to grant a sublicense for such proposed product or process. If we do not grant asublicense within a specified period of time after receiving a written request, the Whitehead Institute may grant a license to the third party and our rightsin the field of use of such sublicense will terminate. Additionally, after a specified period of time, if we are not actively conducting high-throughputscreening using the licensed patent rights to identify product candidates, then, except for any rights directed to uses that we are actively developing, theWhitehead Institute may convert our license to the licensed patent rights from exclusive to non-exclusive.26Table of Contents We have the right to terminate the agreement for any reason upon at least 90 days' prior written notice. The Whitehead Institute has the right toterminate the agreement if we and all of our sublicensees cease to carry on business related to the agreement for a specified period of time, we fail to payany amounts due and payable under the agreement to the Whitehead Institute, subject to a grace period, we materially breach the agreement and fail tocure such breach within a specified grace period or we or a sublicensee challenge the licensed patent rights in a legal or administrative proceeding. Theagreement otherwise terminates upon the expiration or abandonment of all licensed patents and patent applications.Cancer diagnostic license agreement In October 2010, we entered into a separate license agreement with the Whitehead Institute, or the cancer diagnostic license agreement, underwhich we acquired a non-exclusive, worldwide license to patent rights owned by the Whitehead Institute for research purposes. In December 2011, weamended and restated this agreement with the Whitehead Institute. Under the amended and restated cancer diagnostic license agreement, we acquired anexclusive, royalty-bearing, worldwide license under these patent rights to develop, make, use and sell products covered by the licensed patent rights andto develop and perform services using a licensed product or the practice of the licensed patent rights for or on behalf of a third party, in each case, forcancer diagnostics and companion clinical uses. Under the agreement, we paid the Whitehead Institute upfront license fees and reimbursed patent related fees and costs incurred by the WhiteheadInstitute totaling $70,000 in the aggregate. We also agreed to pay the Whitehead Institute annual license maintenance fees, milestone payments, royaltiesas a percentage of net sales and a percentage of sublicense income that we receive. Annual license maintenance fees are creditable against royalties,which are described below, earned during the same calendar year. Milestone payments of up to an aggregate of $825,000 are triggered upon theachievement of specified regulatory and commercialization milestones and are not creditable against the royalties described below. The royalty rate is inthe mid-single digits as a percentage of net sales. If we are required to pay royalties to a third party in consideration of a license or similar right in order to make, use or sell a licensed product orlicensed service, then we may deduct up to 50% of the amounts paid to such third party, subject to specified limitations, from the payments that we oweto the Whitehead Institute for such licensed product or licensed service. We are required to use commercially reasonable efforts to develop and commercialize licensed products or licensed services under the agreement.In particular, we are required to fulfill specific development, regulatory and commercialization milestones by particular dates and to commit a specifiednumber of full time staff equivalents toward the development of a licensed product or licensed service until the first commercial sale of a licensedproduct or performance of a licensed service. The Whitehead Institute retains the right to, and may grant licenses to other academic and non-profit institutions for the right to, practice thelicensed patent rights for research, teaching and educational purposes. The Whitehead Institute or any such other institution could seek to license to thirdparties any intellectual property rights that it discovers using the licensed patent rights while pursuing these purposes. After a specified period of time, if a third party requests to sublicense the patent rights for a product or service that is not directly competitive withour products or services, we must enter into good-faith negotiations to grant a sublicense for such proposed product or service. If we do not grant sucha sublicense within a specified period of time after receiving a written request, the Whitehead Institute may grant a license to the third party and ourrights in the field of use of such sublicense will terminate. Additionally, after a specified period of time, if the market is not being reasonably served byus, as determined by the Whitehead Institute, and a third party requests to sublicense the patent rights27Table of Contentsfor a product or service that is directly competitive with our products or services, we must enter into good-faith negotiations to grant a sublicense forsuch proposed product or service. If we do not grant such a sublicense within a specified period of time after receiving a written request, we and theWhitehead Institute have agreed to mutually select a qualified independent third party to set commercially reasonable terms and conditions consistentwith similar technology in the industry under which we would sublicense our rights for such proposed product or service to the third party.Additionally, after a specified period of time, if we are not actively conducting efforts to validate, use or commercialize a license product or licensedservice, then the Whitehead Institute may convert our license to the licensed patent rights from exclusive to nonexclusive. We have the right to terminate the agreement for any reason upon at least 90 days' prior written notice. The Whitehead Institute has the right toterminate the agreement if we and all of our sublicensees cease to carry on business related to the agreement for a specified period of time, we fail to payany amounts due and payable under the agreement to the Whitehead Institute, subject to a grace period, we materially breach the agreement and fail tocure such breach within a specified grace period or we or a sublicensee challenge the licensed patent rights in a legal or administrative proceeding. Theagreement otherwise terminates upon the expiration or abandonment of all licensed patents and patent applications.The Scripps Research Institute In November 2011, we entered into a license agreement with Poniard Pharmaceuticals, Inc., or Poniard, under which we acquired an exclusive,worldwide license under patent rights and know-how owned or controlled by Poniard to develop, make, use and sell compounds and products coveredby the licensed patent rights for the diagnosis, treatment, prevention or control of all human diseases and conditions. These licensed patent rights includepatent rights owned by The Scripps Research Institute, or Scripps, and licensed to Poniard. Under the agreement, we paid Poniard an upfront license feeand agreed to pay Poniard milestone payments upon the achievement of specified development and regulatory milestones. On August 2, 2013, patents and other rights which were the subject of our license agreement with Poniard were sold to Encarta, Inc., or Encarta.We purchased these assets from Encarta in an asset purchase agreement dated February 21, 2014 and also entered into a securities issuance agreement.Under the terms of these agreements, we issued 97,500 shares of common stock, issued a warrant to purchase 142,857 shares of common stock with anexercise price equal to $17.16 per share and paid Encarta $25,000. All existing obligations under the license agreement, including an achieveddevelopment milestone and an obligation to issue a warrant, were settled as part of this transaction. In connection with the asset purchase agreement, we also assumed the rights and obligations under the license agreement by and between Scrippsand Poniard, dated May 5, 2008, or the Scripps License Agreement. Pursuant to the Scripps License Agreement, we acquired an exclusive, worldwidelicense under patent rights owned or controlled by Scripps to make and have made, to use and have used, to offer to sell, to sell and have sold, andimport products covered by the licensed patent rights for the diagnosis, treatment or prevention of human diseases or conditions. The licensed patentrights include patents covering our product candidate VS-4718. Under the Scripps License Agreement, Scripps retains the right to grant non-exclusivelicenses to nonprofit or academic institutions, without the right to sublicense, to use any of the licensed patent rights for any noncommercial research oreducation purposes. Pursuant to the Scripps License Agreement, we are obligated to pay Scripps potential product development milestone payments of up to anaggregate of $3,000,000 upon the achievement of specified development and regulatory milestones. In addition, we are obligated to pay Scripps lowsingle-digit royalties as a percentage of net sales of licensed products. Our obligation to pay royalties28Table of Contentson net sales is on a country by country basis. In the event that we challenge a patent or patent application covered by the Scripps License Agreement,our royalties will increase by fifty percent during the pendency of the challenge (and increase by one hundred percent in the event the challenge is notsuccessful). We also forfeit the right to recoup any royalties, sublicense payments, milestone payments, patent costs or other payments during the periodof any challenge to the patents covered under the Scripps License Agreement. If we license or acquire technology from a third party in order to commercialize a licensed product and to pay such third party royalties or otheramounts, then we may deduct up to 50% of the amount paid to such third party from the payments owed to Scripps for such licensed product. Thisdeduction is subject to specified limitations, including that in no event will any such deduction reduce a payment that we owe to Scripps to less than50% of the otherwise applicable amount. We are required to use reasonable and diligent efforts to commercialize (directly or through sublicense arrangements) licensed products (includingour product candidate VS-4718) in either the United States, the United Kingdom, France, Germany or Japan. The Scripps License Agreement expires upon the last expiration of any of the licensed patent rights. We have the right to terminate the ScrippsLicense Agreement or any portion of our licensed rights under the Scripps License Agreement for any reason upon at least 90 days prior written noticeand payment of a low five-figure termination fee. We are not responsible for the termination fee if Scripps defaults in the performance of its materialobligations and fails to cure. Scripps can terminate the Scripps License Agreement for certain material breaches by us or defaults in our performance ofmaterial obligations.Pfizer Inc. On July 11, 2012, we entered into a license agreement with Pfizer Inc., or Pfizer, under which Pfizer granted us worldwide, exclusive rights toresearch, develop, manufacture and commercialize products containing certain of Pfizer's inhibitors of focal adhesion kinase for all therapeutic,diagnostic and prophylactic uses in humans. We have the right to grant sublicenses under the foregoing licensed rights, subject to certain restrictions.We are solely responsible, at our own expense, for the clinical development of these products, which is to be conducted in accordance with an agreed-upon development plan. We are also responsible for all manufacturing and commercialization activities at our own expense. Pfizer provided us with aninitial quantity of clinical supplies of one of the products for an agreed upon price. Upon entering into the license agreement, we made a one-time cash payment to Pfizer in the amount of $1.5 million and issued 192,012 shares ofour common stock. Pfizer is also eligible to receive up to $2 million in developmental milestones and up to an additional $125 million based on thesuccessful attainment of regulatory and commercial sales milestones. Pfizer is also eligible to receive high single to mid double digit royalties on futurenet sales of the products. Our royalty obligations with respect to each product in each country begin on the date of first commercial sale of the product inthat country, and end on the later of 10 years after the date of first commercial sale of the product in that country or the date of expiration orabandonment of the last claim contained in any issued patent or patent application licensed by Pfizer to us that covers the product in that country. The license agreement will remain in effect until the expiration of all of our royalty obligations to Pfizer, determined on a product-by-product andcountry-by-country basis. So long as we are not in breach of the license agreement, we have the right to terminate the license agreement at will on aproduct-by-product and country-by-country basis, or in its entirety, upon 90 days written notice to Pfizer. Either party has the right to terminate thelicense agreement in connection with an insolvency event involving the other party or a material breach of the license agreement by the other party thatremains uncured for a specified period of time. If the license agreement is terminated by either party29Table of Contentsfor any reason, worldwide rights to the research, development, manufacture and commercialization of the products revert back to Pfizer.COMPETITION The biotechnology and pharmaceutical industries are characterized by rapidly advancing technologies, intense competition and a strong emphasison proprietary products. While we believe that our technology, development experience and scientific knowledge provide us with competitiveadvantages, we face potential competition from many different sources, including major pharmaceutical, specialty pharmaceutical and biotechnologycompanies, academic institutions and governmental agencies and public and private research institutions. Any product candidates that we successfullydevelop and commercialize will compete with existing therapies and new therapies that may become available in the future. There are other companies working to develop therapies that target CSCs. These companies include divisions of large pharmaceutical companiesincluding Astellas Pharma Inc., Celgene, Inc., Sanofi-Aventis U.S. LLC, GlaxoSmithKline plc, Boehringer Ingelheim GmbH, Pfizer Inc. and others.There are also biotechnology companies of various sizes that are developing therapies against CSCs, including OncoMed Pharmaceuticals, Inc., BostonBiomedical Inc. (a division of Dainippon Sumitomo Corp) and Stemline Therapeutics, Inc. and others. Many of our competitors may have significantly greater financial resources and expertise in research and development, manufacturing, preclinicaltesting, conducting clinical trials, obtaining regulatory approvals and marketing approved products than we do. Mergers and acquisitions in thepharmaceutical, biotechnology and diagnostic industries may result in even more resources being concentrated among a smaller number of ourcompetitors. These competitors also compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinicaltrial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs. Smaller orearly stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and establishedcompanies. The key competitive factors affecting the success of all of our product candidates, if approved, are likely to be their efficacy, safety, convenience,price, the effectiveness of companion diagnostics in guiding the use of related therapeutics, the level of generic competition and the availability ofreimbursement from government and other third-party payors. Our commercial opportunity could be reduced or eliminated if our competitors develop and commercialize products that are safer, more effective,have fewer or less severe side effects, are more convenient or are less expensive than any products that we may develop. Our competitors also mayobtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitorsestablishing a strong market position before we are able to enter the market. In addition, our ability to compete may be affected in many cases byinsurers or other third-party payors seeking to encourage the use of generic products. There are many generic products currently on the market for theindications that we are pursuing, and additional products are expected to become available on a generic basis over the coming years. If our therapeuticproduct candidates are approved, we expect that they will be priced at a significant premium over competitive generic products. The most common methods of treating patients with cancer are surgery, radiation and drug therapy, including chemotherapy, hormone therapy andtargeted drug therapy. There are a variety of available drug therapies marketed for cancer. In many cases, these drugs are administered in combination toenhance efficacy. While our product candidates may compete with many existing drug and other therapies, to the extent they are ultimately used incombination with or as an adjunct to these therapies, our product candidates will not be competitive with them. Some of the currently30Table of Contentsapproved drug therapies are branded and subject to patent protection, and others are available on a generic basis. Many of these approved drugs are wellestablished therapies and are widely accepted by physicians, patients and third-party payors. In general, although there has been considerable progressover the past few decades in the treatment of cancer and the currently marketed therapies provide benefits to many patients, these therapies all are limitedto some extent in their efficacy and frequency of adverse events, and none of them are successful in treating all patients. As a result, the level ofmorbidity and mortality from cancer remains high. In addition to currently marketed therapies, there are also a number of products in late stage clinical development to treat cancer. These products indevelopment may provide efficacy, safety, convenience and other benefits that are not provided by currently marketed therapies. As a result, they mayprovide significant competition for any of our product candidates for which we obtain market approval.MANUFACTURING We do not own or operate, and currently have no plans to establish, any manufacturing facilities. We currently rely, and expect to continue to rely,on third parties for the manufacture of our product candidates and any products that we may develop, other than small amounts of compounds that wemay synthesize ourselves for preclinical testing. To date, we have obtained starting materials for our supply of the bulk drug substance for our productcandidates from third-party manufacturers. We obtain our supplies from these manufacturers on a purchase order basis and do not have a long-termsupply arrangement in place. We do not currently have arrangements in place for redundant supply or a second source for bulk drug substance. If ourcurrent third-party manufacturers should become unavailable to us for any reason, we believe that there are several potential replacements, although wemight incur some delay in identifying and qualifying such replacements. All of our drug candidates are organic compounds of low molecular weight, generally called small molecules. We select compounds not only onthe basis of their potential efficacy and safety, but also for their ease of synthesis and reasonable cost of their starting materials. We expect to continue todevelop drug candidates that can be produced cost-effectively at third-party manufacturing facilities.GOVERNMENT REGULATION Government authorities in the United States, at the federal, state and local level, and in other countries extensively regulate, among other things, theresearch, development, testing, manufacture, including any manufacturing changes, packaging, storage, recordkeeping, labeling, advertising, promotion,distribution, marketing, post-approval monitoring and reporting, import and export of pharmaceutical products, such as those we are developing.United States drug approval process In the United States, the FDA regulates drugs under the Federal Food, Drug, and Cosmetic Act, or FDCA, the Public Health Service Act andimplementing regulations. The process of obtaining regulatory approvals and the subsequent compliance with appropriate federal, state, local andforeign statutes and regulations requires the expenditure of substantial time and financial resources. Failure to comply with the applicable United Statesrequirements at any time during the product development process, approval process or after approval, may subject an applicant to a variety ofadministrative or judicial sanctions, such as the FDA's refusal to approve pending applications, withdrawal of an approval, imposition of a clinical hold,issuance of warning letters, product recalls, product seizures, total or partial suspension of production or distribution injunctions, fines, refusals ofgovernment contracts, restitution, disgorgement of profits or civil or criminal penalties.31Table of Contents The process required by the FDA before a drug may be marketed in the United States generally involves the following:•completion of preclinical laboratory tests, animal studies and formulation studies in compliance with the FDA's good laboratory practice,or GLP, regulations; •submission to the FDA of an IND, which must become effective before human clinical trials may begin; •approval by an independent institutional review board, or IRB, at each clinical site before each trial may be initiated; •performance of adequate and well-controlled human clinical trials in accordance with good clinical practices, or GCP, to establish thesafety and efficacy of the proposed drug for each indication; •submission to the FDA of a new drug application, or NDA; •satisfactory completion of an FDA advisory committee review, if applicable; •satisfactory completion of an FDA inspection of the manufacturing facility or facilities at which the product is produced to assesscompliance with current good manufacturing practices, or cGMP, requirements and to assure that the facilities, methods and controls areadequate to preserve the drug's identity, strength, quality and purity; and •FDA review and approval of the NDA.Preclinical studies Preclinical studies include laboratory evaluation of product chemistry and formulation, as well as in vitro and animal studies to assess the potentialfor adverse events and in some cases to establish a rationale for therapeutic use. The conduct of preclinical studies is subject to federal regulations andrequirements, including GLP regulations. An IND sponsor must submit the results of the preclinical tests, together with manufacturing information,analytical data, any available clinical data or literature and plans for clinical studies, among other things, to the FDA as part of an IND. Some long-termpreclinical testing, such as animal tests of reproductive adverse events and carcinogenicity, may continue after the IND is submitted. An INDautomatically becomes effective 30 days after receipt by the FDA, unless before that time the FDA raises concerns or questions related to one or moreproposed clinical trials and places the trial on clinical hold. In such a case, the IND sponsor and the FDA must resolve any outstanding concerns beforethe clinical trial can begin. As a result, submission of an IND may not result in the FDA allowing clinical trials to commence.Clinical trials Clinical trials involve the administration of the investigational new drug to human subjects under the supervision of qualified investigators inaccordance with GCP requirements, which include, among other things, the requirement that all research subjects provide their informed consent inwriting before their participation in any clinical trial. Clinical trials are conducted under written study protocols detailing, among other things, theobjectives of the study, the parameters to be used in monitoring safety and the effectiveness criteria to be evaluated. A protocol for each clinical trial andany subsequent protocol amendments must be submitted to the FDA as part of the IND. In addition, an IRB at each institution participating in theclinical trial must review and approve the plan for any clinical trial before it commences at that institution, and the IRB must conduct continuing review.The IRB must review and approve, among other things, the study protocol and informed consent information to be provided to study subjects. An IRBmust operate in compliance with FDA32Table of Contentsregulations. Information about certain clinical trials must be submitted within specific timeframes to the National Institutes of Health for publicdissemination on their ClinicalTrials.gov website. Human clinical trials are typically conducted in three sequential phases, which may overlap or be combined:•Phase 1: The drug is initially introduced into healthy human subjects or patients with the target disease or condition and tested forsafety, dosage tolerance, absorption, metabolism, distribution, excretion and, if possible, to gain an early indication of its effectiveness. •Phase 2: The drug is administered to a limited patient population to identify possible adverse effects and safety risks, to preliminarilyevaluate the efficacy of the product for specific targeted diseases and to determine dosage tolerance and optimal dosage. •Phase 3: The drug is administered to an expanded patient population in adequate and well-controlled clinical trials to generate sufficientdata to statistically confirm the efficacy and safety of the product for approval, to establish the overall risk-benefit profile of the productand to provide adequate information for the labeling of the product. Progress reports detailing the results of the clinical trials must be submitted at least annually to the FDA and more frequently if serious adverseevents occur. Phase 1, Phase 2 and Phase 3 clinical trials may not be completed successfully within any specified period, or at all. Furthermore, theFDA or the sponsor may suspend or terminate a clinical trial at any time on various grounds, including a finding that the research subjects are beingexposed to an unacceptable health risk. Similarly, an IRB can suspend or terminate approval of a clinical trial at its institution if the clinical trial is notbeing conducted in accordance with the IRB's requirements or if the drug has been associated with unexpected serious harm to patients.Marketing approval Assuming successful completion of the required clinical testing, the results of the preclinical and clinical studies, together with detailed informationrelating to the product's chemistry, manufacture, controls and proposed labeling, among other things, are submitted to the FDA as part of an NDArequesting approval to market the product for one or more indications. Under federal law, the submission of most NDAs is additionally subject to asubstantial application user fee, currently exceeding $1.8 million, and the sponsor of an approved NDA is also subject to annual product andestablishment user fees, currently exceeding $98,000 per product and $520,000 per establishment. These fees are typically increased annually. The FDA conducts a preliminary review of all NDAs within the first 60 days after submission before accepting them for filing to determinewhether they are sufficiently complete to permit substantive review. The FDA may request additional information rather than accept an NDA for filing.In this event, the application must be resubmitted with the additional information. The resubmitted application is also subject to review before the FDAaccepts it for filing. Once the submission is accepted for filing, the FDA begins an in-depth substantive review. The FDA has agreed to specifiedperformance goals in the review of NDAs. Under these goals, the FDA has committed to review most such applications for non-priority productswithin 10 months, and most applications for priority review products, that is, drugs that the FDA determines represent a significant improvement overexisting therapy, within six months. The review process may be extended by the FDA for three additional months to consider certain information orclarification regarding information already provided in the submission. The FDA may also refer applications for novel drugs or products that presentdifficult questions of safety or efficacy to an advisory committee, typically a panel that includes clinicians and other experts, for review, evaluation and arecommendation as to whether the application should be33Table of Contentsapproved. The FDA is not bound by the recommendations of an advisory committee, but it considers such recommendations carefully when makingdecisions. Before approving an NDA, the FDA typically will inspect the facility or facilities where the product is manufactured. The FDA will not approvean application unless it determines that the manufacturing processes and facilities are in compliance with cGMP requirements and adequate to assureconsistent production of the product within required specifications. In addition, before approving an NDA, the FDA will typically inspect one or moreclinical sites to assure compliance with GCP and integrity of the clinical data submitted. The testing and approval process requires substantial time, effort and financial resources, and each may take many years to complete. Data obtainedfrom clinical activities are not always conclusive and may be susceptible to varying interpretations, which could delay, limit or prevent regulatoryapproval. The FDA may not grant approval on a timely basis, or at all. We may encounter difficulties or unanticipated costs in our efforts to develop ourproduct candidates and secure necessary governmental approvals, which could delay or preclude us from marketing our products. After the FDA's evaluation of the NDA and inspection of the manufacturing facilities, the FDA may issue an approval letter or a completeresponse letter. An approval letter authorizes commercial marketing of the drug with specific prescribing information for specific indications. Acomplete response letter generally outlines the deficiencies in the submission and may require substantial additional testing or information in order forthe FDA to reconsider the application. If and when those deficiencies have been addressed to the FDA's satisfaction in a resubmission of the NDA, theFDA will issue an approval letter. The FDA has committed to reviewing such resubmissions in two or six months depending on the type of informationincluded. Even with submission of this additional information, the FDA ultimately may decide that the application does not satisfy the regulatory criteriafor approval and refuse to approve the NDA. Even if the FDA approves a product, it may limit the approved indications for use for the product, require that contraindications, warnings orprecautions be included in the product labeling, require that post-approval studies, including Phase 4 clinical trials, be conducted to further assess adrug's safety after approval, require testing and surveillance programs to monitor the product after commercialization, or impose other conditions,including distribution restrictions or other risk management mechanisms, which can materially affect the potential market and profitability of the product.The FDA may prevent or limit further marketing of a product based on the results of post-market studies or surveillance programs. After approval,some types of changes to the approved product, such as adding new indications, manufacturing changes and additional labeling claims, are subject tofurther testing requirements and FDA review and approval.Fast track designation The FDA is required to facilitate the development and expedite the review of drugs that are intended for the treatment of a serious or life-threatening condition for which there is no effective treatment and which demonstrate the potential to address unmet medical needs for the condition.Under the fast track program, the sponsor of a new drug candidate may request the FDA to designate the product for a specific indication as a fast trackproduct concurrent with or after the filing of the IND for the product candidate. The FDA must determine if the product candidate qualifies for fast trackdesignation within 60 days after receipt of the sponsor's request. In addition to other benefits, such as the ability to use surrogate endpoints and have greater interactions with the FDA, the FDA may initiate reviewof sections of a fast track product's NDA before the application is complete. This rolling review is available if the applicant provides and the FDAapproves a schedule for the submission of the remaining information and the applicant pays applicable user fees. However, the FDA's time period goalfor reviewing a fast track application does not begin until the last section of the NDA is submitted. In addition, the fast track designation may bewithdrawn by the FDA if the FDA believes that the designation is no longer supported by data emerging in the clinical trial process.34Table of ContentsPriority review Under FDA policies, a product candidate may be eligible for priority review, or review within a six-month time frame from the time a completeapplication is received. Products regulated by the FDA's Center for Drug Evaluation and Research, or CDER, are eligible for priority review if theyprovide a significant improvement compared to marketed products in the treatment, diagnosis or prevention of a disease. A fast track designated productcandidate would ordinarily meet the FDA's criteria for priority review.Accelerated approval Under the FDA's accelerated approval regulations, the FDA may approve a drug for a serious or life-threatening illness that provides meaningfultherapeutic benefit to patients over existing treatments based upon a surrogate endpoint that is reasonably likely to predict clinical benefit. In clinicaltrials, a surrogate endpoint is a measurement of laboratory or clinical signs of a disease or condition that substitutes for a direct measurement of how apatient feels, functions or survives. Surrogate endpoints can often be measured more easily or more rapidly than clinical endpoints. A product candidateapproved on this basis is subject to rigorous post-marketing compliance requirements, including the completion of Phase 4 or post-approval clinicaltrials to confirm the effect on the clinical endpoint. Failure to conduct required post-approval studies, or confirm a clinical benefit during post-marketingstudies, would allow the FDA to withdraw the drug from the market on an expedited basis. All promotional materials for drug candidates approvedunder accelerated regulations are subject to prior review by the FDA.Orphan drugs Under the Orphan Drug Act, the FDA may grant orphan drug designation to drugs intended to treat a rare disease or condition, which is generallydefined as a disease or condition that affects fewer than 200,000 individuals in the United States. Orphan drug designation must be requested beforesubmitting an NDA. After the FDA grants orphan drug designation, the generic identity of the drug and its potential orphan use are disclosed publiclyby the FDA. Orphan drug designation does not convey any advantage in, or shorten the duration of, the regulatory review and approval process. Thefirst NDA applicant to receive FDA approval for a particular active ingredient to treat a particular disease with FDA orphan drug designation is entitledto a seven-year exclusive marketing period in the United States for that product, for that indication. During the seven-year exclusivity period, the FDAmay not approve any other applications to market the same drug for the same orphan indication, except in limited circumstances, such as a showing ofclinical superiority to the product with orphan drug exclusivity in that it is shown to be safer, more effective or makes a major contribution to patientcare. Orphan drug exclusivity does not prevent the FDA from approving a different drug for the same disease or condition, or the same drug for adifferent disease or condition. Among the other benefits of orphan drug designation are tax credits for certain research and a waiver of the NDAapplication user fee.Pediatric information Under the Pediatric Research Equity Act of 2003, as amended and reauthorized by the Food and Drug Administration Amendments Act of 2007,or the FDAAA, an NDA or supplement to an NDA must contain data that are adequate to assess the safety and effectiveness of the drug for the claimedindications in all relevant pediatric subpopulations, and to support dosing and administration for each pediatric subpopulation for which the product issafe and effective. The FDA may, on its own initiative or at the request of the applicant, grant deferrals for submission of some or all pediatric data untilafter approval of the product for use in adults, or full or partial waivers from the pediatric data35Table of Contentsrequirements. Unless otherwise required by regulation, the pediatric data requirements do not apply to products with orphan drug designation.The Hatch-Waxman actAbbreviated new drug applications In seeking approval for a drug through an NDA, applicants are required to list with the FDA each patent with claims that cover the applicant'sproduct or a method of using the product. Upon approval of a drug, each of the patents listed in the application for the drug is then published in theFDA's Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book. Drugs listed in the Orange Bookcan, in turn, be cited by potential competitors in support of approval of an abbreviated new drug application, or ANDA. Generally, an ANDA providesfor marketing of a drug product that has the same active ingredients in the same strengths, dosage form and route of administration as the listed drug andhas been shown to be bioequivalent through in vitro or in vivo testing or otherwise to the listed drug. ANDA applicants are not required to conduct orsubmit results of preclinical or clinical tests to prove the safety or effectiveness of their drug product, other than the requirement for bioequivalencetesting. Drugs approved in this way are commonly referred to as "generic equivalents" to the listed drug, and can often be substituted by pharmacistsunder prescriptions written for the original listed drug. The ANDA applicant is required to certify to the FDA concerning any patents listed for the approved product in the FDA's Orange Book, exceptfor patents covering methods of use for which the ANDA applicant is not seeking approval. Specifically, the applicant must certify with respect to eachpatent that:•the required patent information has not been filed; •the listed patent has expired; •the listed patent has not expired, but will expire on a particular date and approval is sought after patent expiration; or •the listed patent is invalid, unenforceable or will not be infringed by the new product. A certification that the new product will not infringe the already approved product's listed patents or that such patents are invalid or unenforceableis called a Paragraph IV certification. If the applicant does not challenge the listed patents or indicate that it is not seeking approval of a patented methodof use, the ANDA application will not be approved until all the listed patents claiming the referenced product have expired. If the ANDA applicant has provided a Paragraph IV certification to the FDA, the applicant must also send notice of the Paragraph IV certificationto the NDA and patent holders once the ANDA has been accepted for filing by the FDA. The NDA and patent holders may then initiate a patentinfringement lawsuit in response to the notice of the Paragraph IV certification. The filing of a patent infringement lawsuit within 45 days after thereceipt of a Paragraph IV certification automatically prevents the FDA from approving the ANDA until the earlier of 30 months after the NDA or patentholder's receipt of the Paragraph IV certification, expiration of the patent, settlement of the lawsuit or a decision in the infringement case that is favorableto the ANDA applicant. The ANDA also will not be approved until any applicable non-patent exclusivity period, such as exclusivity for obtaining approval of a newchemical entity, for the referenced product has expired. Federal law provides a period of five years following approval of a drug containing nopreviously approved active moiety during which ANDAs for generic versions of those drugs cannot be submitted unless the submission contains aParagraph IV challenge to a listed patent, in which case the submission may be made four years following the original product approval. Federal lawprovides for a36Table of Contentsperiod of three years of exclusivity during which the FDA cannot grant effective approval of an ANDA for the conditions of use covered by theexclusivity, but FDA requires as a condition of approval new clinical trials conducted by or for the sponsor. This three-year exclusivity period oftenprotects changes to a previously approved drug product, such as a new dosage form, route of administration, combination or indication. Under the BestPharmaceuticals for Children Act, federal law also provides that periods of patent and non-patent marketing exclusivity listed in the Orange Book for adrug may be extended by six months if the NDA sponsor conducts pediatric studies identified by the FDA in a written request. For written requestsissued by the FDA after September 27, 2007, the date of enactment of the FDAAA, the FDA must grant pediatric exclusivity no later than nine monthsprior to the date of expiration of patent or non-patent exclusivity in order for the six-month pediatric extension to apply to that exclusivity period.Section 505(b)(2) new drug applications Most drug products obtain FDA marketing approval pursuant to an NDA or an ANDA. A third alternative is a special type of NDA, commonlyreferred to as a Section 505(b)(2) NDA, which enables the applicant to rely, in part, on the FDA's previous approval of a similar product, or publishedliterature, in support of its application. 505(b)(2) NDAs often provide an alternate path to FDA approval for new or improved formulations or new uses of previously approvedproducts. Section 505(b)(2) permits the filing of an NDA where at least some of the information required for approval comes from studies notconducted by or for the applicant and for which the applicant has not obtained a right of reference. If the 505(b)(2) applicant can establish that relianceon the FDA's previous approval is scientifically appropriate, it may eliminate the need to conduct certain preclinical or clinical studies of the newproduct. The FDA may also require companies to perform additional studies or measurements to support the change from the approved product. TheFDA may then approve the new product candidate for all or some of the label indications for which the referenced product has been approved, as wellas for any new indication sought by the Section 505(b)(2) applicant. To the extent that the Section 505(b)(2) applicant is relying on studies conducted for an already approved product, the applicant is required tocertify to the FDA concerning any patents listed for the approved product in the Orange Book to the same extent that an ANDA applicant would. As aresult, approval of a 505(b)(2) NDA can be stalled until all the listed patents claiming the referenced product have expired, until any non-patentexclusivity, such as exclusivity for obtaining approval of a new chemical entity, listed in the Orange Book for the referenced product has expired, and,in the case of a Paragraph IV certification and subsequent patent infringement suit, until the earlier of 30 months, settlement of the lawsuit or a decisionin the infringement case that is favorable to the Section 505(b)(2) applicant.Combination products The FDA regulates combinations of products that cross FDA centers, such as drug, biologic or medical device components that are physically,chemically or otherwise combined into a single entity, as a combination product. The FDA center with primary jurisdiction for the combination productwill take the lead in the premarket review of the product, with the other center consulting or collaborating with the lead center. The FDA's Office of Combination Products, or OCP, determines which center will have primary jurisdiction for the combination product based onthe combination product's "primary mode of action." A mode of action is the means by which a product achieves an intended therapeutic effect or action.The primary mode of action is the mode of action that provides the most important therapeutic action37Table of Contentsof the combination product, or the mode of action expected to make the greatest contribution to the overall intended therapeutic effects of thecombination product. Often it is difficult for the OCP to determine with reasonable certainty the most important therapeutic action of the combination product. In thosedifficult cases, the OCP will consider consistency with other combination products raising similar types of safety and effectiveness questions, or whichcenter has the most expertise to evaluate the most significant safety and effectiveness questions raised by the combination product. A sponsor may use a voluntary formal process, known as a Request for Designation, when the product classification is unclear or in dispute, toobtain a binding decision as to which center will regulate the combination product. If the sponsor objects to that decision, it may request that the agencyreconsider that decision.Overview of FDA regulation of companion diagnostics We are developing in vitro and in vivo companion diagnostics for use in selecting the patients that we believe will respond to our cancertherapeutics. FDA officials have issued draft guidance that, when finalized, would address issues critical to developing in vitro companion diagnostics, such asbiomarker qualification, establishing clinical validity, the use of retrospective data, the appropriate patient population and when the FDA will require thatthe device and the drug be approved simultaneously. The draft guidance issued in July 2011 states that if safe and effective use of a therapeutic productdepends on an in vitro diagnostic, then the FDA generally will require approval or clearance of the diagnostic at the same time that the FDA approvesthe therapeutic product. The FDA has yet to issue further guidance, and it is unclear whether it will do so, or what the scope would be. The FDA previously has required in vitro companion diagnostics intended to select the patients who will respond to the cancer treatment to obtainPre-Market Approval, or PMA, simultaneously with approval of the drug. Based on the draft guidance, and the FDA's past treatment of companiondiagnostics, we believe that the FDA will require one or more of our in vitro companion diagnostics to obtain PMA for our companion diagnostics toidentify patient populations suitable for our cancer therapies, such as the in vitro companion diagnostic for our product candidates. The review of thesein vitro companion diagnostics in conjunction with the review of our cancer treatments involves coordination of review by CDER and by the FDA'sCenter for Devices and Radiological Health Office of In Vitro Diagnostics Device Evaluation and Safety.PMA approval pathway A medical device, including an in vitro diagnostic, or IVD, to be commercially distributed in the United States must receive either 510(k) clearanceor PMA approval from the FDA prior to marketing. Devices deemed by the FDA to pose the greatest risk, such as life-sustaining, life supporting orimplantable devices, or devices deemed not substantially equivalent to a previously 510(k) cleared device or a preamendment class III device for whichPMA applications have not been called, are placed in Class III requiring PMA approval. The PMA approval pathway requires proof of the safety andeffectiveness of the device to the FDA's satisfaction. The PMA approval pathway generally takes from one to three years or even longer from submission of the application. A PMA application for an IVD must provide extensive preclinical and clinical trial data. Preclinical data for an IVD includes many different tests,including how reproducible the results are when the same sample is tested multiple times by multiple users at multiple laboratories. The clinical dataneed to establish that the test is sufficiently safe, effective and reliable in the intended use population. In38Table of Contentsaddition, the FDA must be convinced that a device has clinical utility, meaning that an IVD provides information that is clinically meaningful. Abiomarker's clinical significance may be obvious, or the applicant may be able to rely upon published literature or submit data to show clinical utility. A PMA application also must provide information about the device and its components regarding, among other things, device design,manufacturing and labeling. The sponsor must pay an application fee. As part of the PMA review, the FDA will typically inspect the manufacturer's facilities for compliance with Quality System Regulation, or QSR,requirements, which impose elaborate testing, control, documentation and other quality assurance procedures. Upon submission, the FDA determines if the PMA application is sufficiently complete to permit a substantive review, and, if so, the FDA acceptsthe application for filing. The FDA then commences an in-depth review of the PMA application. The entire process typically takes one to three years,but may take longer. The review time is often significantly extended as a result of the FDA asking for more information or clarification of informationalready provided. The FDA also may respond with a not approvable determination based on deficiencies in the application and require additional clinicaltrials that are often expensive and time-consuming and can substantially delay approval. During the review period, an FDA advisory committee, typically a panel of clinicians, may be convened to review the application and recommendto the FDA whether, or upon what conditions, the device should be approved. Although the FDA is not bound by the advisory panel decision, thepanel's recommendation is important to the FDA's overall decision making process. If the FDA's evaluation of the PMA application is favorable, the FDA typically issues an approvable letter requiring the applicant's agreement tospecific conditions, such as changes in labeling, or specific additional information, such as submission of final labeling, in order to secure final approvalof the PMA. If the FDA concludes that the applicable criteria have been met, the FDA will issue a PMA for the approved indications, which can bemore limited than those originally sought by the manufacturer. The PMA can include post-approval conditions that the FDA believes necessary toensure the safety and effectiveness of the device, including, among other things, restrictions on labeling, promotion, sale and distribution. Failure tocomply with the conditions of approval can result in material adverse enforcement action, including the loss or withdrawal of the approval. Even after approval of a PMA, a new PMA or PMA supplement may be required in the event of a modification to the device, its labeling or itsmanufacturing process. Supplements to a PMA often require the submission of the same type of information required for an original PMA, except thatthe supplement is generally limited to the information needed to support the proposed change from the product covered by the original PMA.Clinical trials A clinical trial is almost always required to support a PMA application. In some cases, one or more smaller Investigational Device Exemption, orIDE, studies may precede a pivotal clinical trial intended to demonstrate the safety and efficacy of the investigational device. All clinical studies of investigational devices must be conducted in compliance with the FDA's requirements. If an investigational device couldpose a significant risk to patients pursuant to FDA regulations, the FDA must approve an IDE application prior to initiation of investigational use. IVDtrials usually do not require an IDE, as the FDA does not judge them to be a significant risk because the results do not affect the patients in the study.However, for a trial where the IVD result directs the therapeutic care of patients with cancer, we believe that the FDA would consider the investigationto present significant risk.39Table of Contents An IDE application must be supported by appropriate data, such as laboratory test results, showing that it is safe to test the device in humans andthat the testing protocol is scientifically sound. The FDA typically grants IDE approval for a specified number of patients. A nonsignificant risk devicedoes not require FDA approval of an IDE. Both significant risk and nonsignificant risk investigational devices require approval from IRBs at the studycenters where the device will be used. During the trial, the sponsor must comply with the FDA's IDE requirements for investigator selection, trial monitoring, reporting and recordkeeping. The investigators must obtain patient informed consent, rigorously follow the investigational plan and study protocol, control the disposition ofinvestigational devices and comply with all reporting and record keeping requirements. Prior to granting PMA approval, the FDA typically inspects therecords relating to the conduct of the study and the clinical data supporting the PMA application for compliance with applicable requirements. Although the QSR does not fully apply to investigational devices, the requirement for controls on design and development does apply. Thesponsor also must manufacture the investigational device in conformity with the quality controls described in the IDE application and any conditions ofIDE approval that the FDA may impose with respect to manufacturing.Post-market After a device is on the market, numerous regulatory requirements apply. These requirements include: the QSR, labeling regulations, the FDA'sgeneral prohibition against promoting products for unapproved or "off label" uses, the Medical Device Reporting regulation, which requires thatmanufacturers report to the FDA if their device may have caused or contributed to a death or serious injury or malfunctioned in a way that would likelycause or contribute to a death or serious injury if it were to recur, and the regulations, requiring manufacturers to report recalls and field actions to theFDA if initiated to reduce a risk to health posed by the device or to remedy a violation of the FDCA. The FDA enforces these requirements by inspection and market surveillance. If the FDA finds a violation, it can institute a wide variety ofenforcement actions, ranging from a public warning letter to more severe sanctions such as: fines, injunctions and civil penalties; recall or seizure ofproducts; operating restrictions, partial suspension or total shutdown of production; refusing requests for PMA approval of new products; withdrawingPMA approvals already granted; and criminal prosecution.Other regulatory requirements Any drug manufactured or distributed by us pursuant to FDA approvals are subject to pervasive and continuing regulation by the FDA, including,among other things, requirements relating to recordkeeping, periodic reporting, product sampling and distribution, advertising and promotion andreporting of adverse experiences with the product. After approval, most changes to the approved product, such as adding new indications or otherlabeling claims are subject to prior FDA review and approval. The FDA may impose a number of post-approval requirements as a condition of approval of an NDA. For example, the FDA may require post-marketing testing, including Phase 4 clinical trials, and surveillance to further assess and monitor the product's safety and effectiveness aftercommercialization. Regulatory approval of oncology products often requires that patients in clinical trials be followed for long periods to determine theoverall survival benefit of the drug. In addition, drug manufacturers and other entities involved in the manufacture and distribution of approved drugs are required to register theirestablishments with the FDA and state agencies, and are subject to periodic unannounced inspections by the FDA and these state agencies forcompliance with cGMP requirements. Changes to the manufacturing process are strictly regulated and often require prior FDA approval before beingimplemented. FDA regulations also require investigation and40Table of Contentscorrection of any deviations from cGMP and impose reporting and documentation requirements upon us and any third-party manufacturers that we maydecide to use. Accordingly, manufacturers must continue to expend time, money and effort in the areas of production and quality control to maintaincGMP compliance. Once an approval is granted, the FDA may withdraw the approval if compliance with regulatory requirements and standards is not maintained or ifproblems occur after the product reaches the market. Later discovery of previously unknown problems with a product, including adverse events ofunanticipated severity or frequency, or with manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to theapproved labeling to add new safety information, imposition of post-market studies or clinical trials to assess new safety risks or imposition ofdistribution or other restrictions under a Risk Evaluation and Mitigation Strategy program. Other potential consequences include, among other things:•restrictions on the marketing or manufacturing of the product, complete withdrawal of the product from the market or product recalls; •fines, warning letters or holds on post-approval clinical trials; •refusal of the FDA to approve pending applications or supplements to approved applications, or suspension or revocation of productlicense approvals; •product seizure or detention, or refusal to permit the import or export of products; or •consent decrees, injunctions or the imposition of civil or criminal penalties. The FDA strictly regulates marketing, labeling, advertising and promotion of products that are placed on the market. Drugs may be promoted onlyfor the approved indications and in accordance with the provisions of the approved label. The FDA and other agencies actively enforce the laws andregulations prohibiting the promotion of off label uses, and a company that is found to have improperly promoted off label uses may be subject tosignificant liability.Additional provisionsAnti-kickback and false claims laws In addition to FDA restrictions on marketing of pharmaceutical products, several other types of state and federal laws have been applied to restrictcertain marketing practices in the pharmaceutical industry in recent years. These laws include anti-kickback statutes and false claims statutes. The federalhealthcare program anti-kickback statute prohibits, among other things, knowingly and willfully offering, paying, soliciting or receiving remuneration toinduce or in return for purchasing, leasing, ordering or arranging for the purchase, lease or order of any healthcare item or service reimbursable underMedicare, Medicaid or other federally financed healthcare programs. This statute has been interpreted to apply to arrangements between pharmaceuticalmanufacturers on the one hand and prescribers, purchasers and formulary managers on the other. Violations of the anti-kickback statute are punishableby imprisonment, criminal fines, civil monetary penalties and exclusion from participation in federal healthcare programs. Although there are a numberof statutory exemptions and regulatory safe harbors protecting certain common activities from prosecution or other regulatory sanctions, the exemptionsand safe harbors are drawn narrowly, and practices that involve remuneration intended to induce prescribing, purchases or recommendations may besubject to scrutiny if they do not qualify for an exemption or safe harbor. Federal false claims laws prohibit any person from knowingly presenting, or causing to be presented, a false claim for payment to the federalgovernment, or knowingly making, or causing to be made, a false statement to have a false claim paid. Recently, several pharmaceutical and otherhealthcare companies have been prosecuted under these laws for allegedly inflating drug prices they41Table of Contentsreport to pricing services, which in turn were used by the government to set Medicare and Medicaid reimbursement rates, and for allegedly providingfree product to customers with the expectation that the customers would bill federal programs for the product. In addition, certain marketing practices,including off-label promotion, may also violate false claims laws. The majority of states also have statutes or regulations similar to the federal anti-kickback law and false claims laws, which apply to items and services reimbursed under Medicaid and other state programs, or, in several states, applyregardless of the payor.Physician drug samples As part of the sales and marketing process, pharmaceutical companies frequently provide samples of approved drugs to physicians. ThePrescription Drug Marketing Act, or the PDMA, imposes requirements and limitations upon the provision of drug samples to physicians, as well asprohibits states from licensing distributors of prescription drugs unless the state licensing program meets certain federal guidelines that includeminimum standards for storage, handling and record keeping. In addition, the PDMA sets forth civil and criminal penalties for violations.Foreign regulation In order to market any product outside of the United States, we would need to comply with numerous and varying regulatory requirements of othercountries regarding safety and efficacy and governing, among other things, clinical trials, marketing authorization, commercial sales and distribution ofour products. Whether or not we obtain FDA approval for a product, we would need to obtain the necessary approvals by the comparable regulatoryauthorities of foreign countries before we can commence clinical trials or marketing of the product in those countries. The approval process varies fromcountry to country and can involve additional product testing and additional administrative review periods. The time required to obtain approval in othercountries might differ from and be longer than that required to obtain FDA approval. Regulatory approval in one country does not ensure regulatoryapproval in another, but a failure or delay in obtaining regulatory approval in one country may negatively impact the regulatory process in others. To date, we have not initiated any discussions with the European Medicines Agency or any other foreign regulatory authorities with respect toseeking regulatory approval for any of our products in Europe or in any other country outside the United States.New legislation and regulations From time to time, legislation is drafted, introduced and passed in Congress that could significantly change the statutory provisions governing thetesting, approval, manufacturing and marketing of products regulated by the FDA. For example, the FDAAA discussed above was enacted in 2007. Inaddition to new legislation, FDA regulations and policies are often revised or interpreted by the agency in ways that may significantly affect ourbusiness and our products. It is impossible to predict whether further legislative changes will be enacted or whether FDA regulations, guidance, policiesor interpretations changed or what the effect of such changes, if any, may be.Pharmaceutical coverage, pricing and reimbursement Significant uncertainty exists as to the coverage and reimbursement status of any drug products for which we obtain regulatory approval. Sales ofany of our product candidates, if approved, will depend, in part, on the extent to which the costs of the products will be covered by third-party payors,including government health programs such as Medicare and Medicaid, commercial health insurers and managed care organizations. The process fordetermining whether a payor will provide coverage for a drug product may be separate from the process for setting the price or reimbursement rate thatthe payor42Table of Contentswill pay for the drug product once coverage is approved. Third-party payors may limit coverage to specific drug products on an approved list, orformulary, which might not include all of the approved drugs for a particular indication. In order to secure coverage and reimbursement for any product that might be approved for sale, we may need to conduct expensivepharmacoeconomic studies in order to demonstrate the medical necessity and cost-effectiveness of the product, in addition to the costs required to obtainFDA or other comparable regulatory approvals. Our product candidates may not be considered medically necessary or cost-effective. A payor's decisionto provide coverage for a drug product does not imply that an adequate reimbursement rate will be approved. Third-party reimbursement may not besufficient to enable us to maintain price levels high enough to realize an appropriate return on our investment in product development. The containment of healthcare costs has become a priority of federal, state and foreign governments, and the prices of drugs have been a focus inthis effort. Third-party payors are increasingly challenging the prices charged for medical products and services and examining the medical necessityand cost-effectiveness of medical products and services, in addition to their safety and efficacy. If these third-party payors do not consider our productsto be cost-effective compared to other available therapies, they may not cover our products after approval as a benefit under their plans or, if they do, thelevel of payment may not be sufficient to allow us to sell our products at a profit. The U.S. government, state legislatures and foreign governments haveshown significant interest in implementing cost containment programs to limit the growth of government-paid health care costs, including price controls,restrictions on reimbursement and requirements for substitution of generic products for branded prescription drugs. Adoption of such controls andmeasures, and tightening of restrictive policies in jurisdictions with existing controls and measures, could limit payments for pharmaceuticals such as thedrug candidates that we are developing and could adversely affect our net revenue and results. Pricing and reimbursement schemes vary widely from country to country. Some countries provide that drug products may be marketed only after areimbursement price has been agreed. Some countries may require the completion of additional studies that compare the cost-effectiveness of a particularproduct candidate to currently available therapies. For example, the European Union provides options for its member states to restrict the range of drugproducts for which their national health insurance systems provide reimbursement and to control the prices of medicinal products for human use.European Union member states may approve a specific price for a drug product or may instead adopt a system of direct or indirect controls on theprofitability of the company placing the drug product on the market. Other member states allow companies to fix their own prices for drug products, butmonitor and control company profits. The downward pressure on health care costs in general, particularly prescription drugs, has become very intense.As a result, increasingly high barriers are being erected to the entry of new products. In addition, in some countries, cross-border imports from low-priced markets exert competitive pressure that may reduce pricing within a country. There can be no assurance that any country that has price controls orreimbursement limitations for drug products will allow favorable reimbursement and pricing arrangements for any of our products. The marketability of any products for which we receive regulatory approval for commercial sale may suffer if the government and third-partypayors fail to provide adequate coverage and reimbursement. In addition, an increasing emphasis on managed care in the United States has increasedand we expect will continue to increase the pressure on drug pricing. Coverage policies, third-party reimbursement rates and drug pricing regulationmay change at any time. In particular, the Patient Protection and Affordable Care Act was enacted in the United States in March 2010 and containprovisions that may reduce the profitability of drug products, including, for example, increased rebates for drugs sold to Medicaid programs, extensionof Medicaid rebates to Medicaid managed care plans, mandatory discounts for certain Medicare Part D beneficiaries and annual fees based on43Table of Contentspharmaceutical companies' share of sales to federal health care programs. Even if favorable coverage and reimbursement status is attained for one ormore products for which we receive regulatory approval, less favorable coverage policies and reimbursement rates may be implemented in the future.EMPLOYEES As of March 3, 2014, we had 30 full-time employees, including a total of 12 employees with M.D. or Ph.D. degrees. Of these full-timeemployees, 20 employees are engaged in research and development activities. None of our employees is represented by a labor union or covered by acollective bargaining agreement. We consider our relationship with our employees to be good.BUSINESS—EXECUTIVE OFFICERS OF THE REGISTRANT The following table sets forth the name, age and position of each of our executive officers as of March 3, 2014. Christoph Westphal, M.D., Ph.D. has served as our Executive Chairman since July 2013, as our Chief Executive Officer from September 2011until July 2013 and our President from September 2011 until January 2013. He has served on our board of directors since August 2010 and as theChairman of our board of directors since March 2011. Dr. Westphal has served as a partner of Longwood Fund, LP, a venture capital investment fund,since 2010. He served as the President of SR One, the corporate venture capital arm of GlaxoSmithKline, from 2010 until 2011. Dr. Westphal haspreviously been involved in founding a number of biotechnology companies as chief executive officer. Dr. Westphal co-founded SirtrisPharmaceuticals, Inc., which was acquired by GlaxoSmithKline plc in 2008, and served as its Chief Executive Officer from 2004 to 2010. He also co-founded Alnara Pharmaceuticals, Inc., Acceleron Pharma, Inc., serving as its Chief Executive Officer in 2003, Alnylam Pharmaceuticals, Inc., servingas its Chief Executive Officer in 2002, and Momenta Pharmaceuticals, Inc., serving as its Chief Executive Officer in 2001. Dr. Westphal serves on theBoard of Fellows of Harvard Medical School and the Board of Overseers for the Boston Symphony Orchestra and is a member of the ResearchAdvisory Council at the Massachusetts General Hospital. He earned his M.D. from Harvard Medical School, his Ph.D. in genetics from HarvardUniversity and his B.A. from Columbia University. Robert Forrester has served has served as our Chief Executive Officer since July 2013, as our Chief Operating Officer from March 2011 untilJuly 2013 and our President since January 2013. Mr. Forrester has previously held executive level positions at both private and public life sciencescompanies. Prior to joining us, Mr. Forrester served as Chief Operating Officer of Forma Therapeutics, Inc. from 2010 until 2011. Previously heserved as Interim President and Chief Executive Officer of CombinatoRx, Inc., now Zalicus Inc., from 2009 until 2010 and as its Executive VicePresident and Chief Financial Officer from 2004 to 2009. Mr. Forrester served as Senior Vice President, Finance and Corporate Development at ColeyPharmaceuticals Group, Inc. from 2000 to 2003. He earned his LL.B. from Bristol University in England.44Name Age PositionChristoph Westphal, M.D., Ph.D. 45 Executive ChairmanRobert Forrester 50 President, Chief Executive OfficerJohn Green 59 Chief Financial OfficerJoanna Horobin, M.B., Ch.B. 59 Chief Medical OfficerDaniel Paterson 52 Chief Business OfficerJonathan Pachter, Ph.D. 56 Vice President, Head of ResearchMonica Singh 33 Vice President and General CounselTable of Contents John (Jack) Green has served as our Chief Financial Officer since May 2013. Prior to joining the Company, from March 2011 until November2012, Mr. Green was Vice President, Finance and a key member of the senior management team for On-Q-ity and oversaw the financial activities,planning, budgeting and management of commercial and banking relationships. Previously, from May 2002 until June 2010, Mr. Green was the SeniorVice President and Chief Financial Officer of GTC Biotherapeutics (formerly Genzyme Transgenics Corporation) which was spun out from GenzymeCorporation as a stand-alone public company. Mr. Green is a Certified Public Accountant (CPA) with over 30 years of financial experience, including20 within the biotechnology industry. Mr. Green received a Master's degree in Business Administration from Boston University Graduate School ofManagement and a Bachelor's degree from the College of the Holy Cross. Joanna Horobin, M.B., Ch.B. has served as our Chief Medical Officer since October 2012. Dr. Horobin has 30 years of pharmaceutical drugdevelopment experience. Prior to joining us, Dr. Horobin served as President of Syndax Pharmaceuticals from April 2012 to October 2012 andPresident and CEO from May 2006 to April 2012. At Syndax, Dr. Horobin designed and implemented the phase 2 clinical development of Entinostat inmetastatic breast cancer. Entinostat has been designated breakthrough designation by the FDA. Previously, she served as VP, Oncology at Rhone-Poulenc Rorer (now Sanofi), COO of CombinatoRx (now Zalicus) and EVP at EntreMed with experience spanning clinical development, marketing,and general management in both public and private companies. At Rhone-Poulenc Rorer (now Sanofi) Dr. Horobin led the global oncology business,including the successful global launch of Taxotere® (docetaxel) in breast cancer and Campto/Camptosar® (CPT11) for colorectal cancer. Dr. Horobinalso led a successful joint venture with Chugai to launch Granocyte® (lenograstim). Previously, Dr. Horobin played significant leadership roles in theapprovals of Lovenox®, Celectol®, Augmentin®, Timentin®, temocillin, Bactroban® and Relafen®/Reliflex®. Dr. Horobin received her medicaldegree from the University of Manchester, England. While in clinical practice she gained membership to the Royal College of General Practitioners inthe United Kingdom and holds a diploma of pharmaceutical medicine from the Royal College of Physicians in the United Kingdom. Daniel Paterson has served as our Chief Business Officer since July 2013 and as our Vice President, Head of Corporate Development andDiagnostics from March 2012 until July 2013. Prior to joining us, Mr. Paterson was a consultant in 2011 until joining us in 2012. From 2009 through2010, Mr. Paterson was the COO of On-Q-ity. Mr. Paterson was the President and CEO of The DNA Repair Company from 2006 until 2009, when itwas acquired by On-Q-ity. Previously, he held senior level positions at IMS Health, CareTools, OnCare and Axion. Jonathan Pachter, Ph.D. has served as our Vice President, Head of Research since July 2011. Prior to joining us, Dr. Pachter served as the SeniorDirector of Cancer Biology at OSI Pharmaceuticals, Inc., which was acquired by Astellas Pharma Inc. in 2010, from 2005 to 2011. He earned hisPh.D. in Neuroscience and his M.S. in Pharmacology from Baylor College of Medicine. Monica Singh has served as our General Counsel since March 2014. Prior to joining the Company, Ms. Singh was a member of the securities andpublic company group at Ropes & Gray LLP where she advised on corporate transactions involving public companies, investment banks and privateequity funds and provided counsel on securities law compliance and corporate governance matters. Ms. Singh holds a B.A. from Amherst College anda J.D. from Harvard Law School.OUR CORPORATE INFORMATION We were incorporated under the laws of the State of Delaware in August 2010. Our principal executive offices are located at 215 First Street,Suite 440, Cambridge, Massachusetts 02142 and our telephone number is (617) 252-9300.45Table of ContentsADDITIONAL INFORMATION We maintain a website at www.verastem.com. We make available, free of charge on our website, our annual report on Form 10-K, quarterlyreports on Form 10-Q, current reports on Form 8-K and all amendments to those reports filed or furnished pursuant to Section 13(a) or 15(d) of theSecurities Exchange Act of 1934, as amended, or the Exchange Act, as soon as reasonably practicable after we electronically file those reports with, orfurnish them to, the Securities and Exchange Commission, or SEC. We also make available, free of charge on our website, the reports filed with theSEC by our executive officers, directors and 10% stockholders pursuant to Section 16 under the Exchange Act as soon as reasonably practicable aftercopies of those filings are provided to us by those persons. The information contained on, or that can be accessed through, our website is not a part ofor incorporated by reference in this annual report on Form 10-K.ITEM 1A. Risk Factors. RISKS RELATED TO OUR FINANCIAL POSITION AND NEED FOR ADDITIONAL CAPITALWe have incurred significant losses since our inception. We expect to incur losses for the foreseeable future and may never achieve or maintainprofitability. Since inception, we have incurred significant operating losses. As of December 31, 2013, we had a deficit accumulated during the developmentstage of $87.7 million. To date, we have not generated any revenues and have financed our operations through private placements of our preferred stockand public offerings completed in February 2012 and July 2013. We have devoted substantially all of our efforts to research and development. Our leadproduct candidate, VS-6063, is currently in a registration-directed trial in mesothelioma. Our other product candidates and VS-6063 in indicationsoutside of mesothelioma are in earlier stage clinical trials. As a result we expect that it will be many years, if ever, before we have a product candidateready for commercialization. We expect to continue to incur significant expenses and increasing operating losses for the foreseeable future. The netlosses we incur may fluctuate significantly from quarter to quarter. We anticipate that our expenses will increase substantially if and as we:•continue our research, preclinical and clinical development of our product candidates, including the registration-directed trial of VS-6063in mesothelioma. •initiate additional clinical trials for our product candidates; •seek marketing approvals for any of our product candidates that successfully complete clinical trials; •ultimately establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketingapproval; •acquire or in-license other products and technologies; •maintain, expand and protect our intellectual property portfolio; •hire additional clinical, development and scientific personnel; and •add operational, financial and management information systems and personnel, including personnel to support our product developmentand planned future commercialization efforts. To become and remain profitable, we must develop and eventually commercialize a product or products with significant market potential. This willrequire us to be successful in a range of challenging activities, including completing preclinical testing and clinical trials of our product candidates,obtaining marketing approval for these product candidates and manufacturing, marketing and selling those products for which we may obtain marketingapproval. We may never succeed in these46Table of Contentsactivities and, even if we do, may never generate revenues that are significant or large enough to achieve profitability. If we do achieve profitability, wemay not be able to sustain or increase profitability on a quarterly or annual basis. Our failure to become and remain profitable would decrease the valueof the company and could impair our ability to raise capital, maintain our research and development efforts, expand our business or continue ouroperations. A decline in the value of our company could also cause you to lose all or part of your investment.Our short operating history may make it difficult for you to evaluate the success of our business to date and to assess our future viability. We are an early stage company. We commenced active operations in the second half of 2010. Our operations to date have been limited toorganizing and staffing our company, business planning, raising capital, acquiring and developing our technology, identifying potential productcandidates and more recently undertaking preclinical and early clinical studies of our product candidates. We completed our initial public offering inFebruary 2012. We have not yet demonstrated our ability to successfully complete any clinical trials, including large-scale, pivotal clinical trials, obtainmarketing approvals, manufacture a commercial scale product, or arrange for a third party to do so on our behalf, or conduct sales and marketingactivities necessary for successful product commercialization. It takes about ten to 15 years to develop one new medicine from the time it is discoveredto when it is available for treating patients. Given our short operating history compared to the timeline required to fully develop a new drug, you arecautioned about making any predictions on our future success or viability based on our activities or results to date. In addition, as a new business, we may encounter unforeseen expenses, difficulties, complications, delays and other known and unknown factors.We will need to transition from a company with a research focus to a company capable of supporting commercial activities. We may not be successful insuch a transition.We will continue to need substantial additional funding. If we are unable to raise capital when needed, we would be forced to delay, reduce oreliminate our product development programs or commercialization efforts. We expect our expenses to increase in connection with our ongoing activities, particularly as we continue the research and development and initiateadditional clinical trials of, and seek marketing approval for, our product candidates. In addition, if we obtain marketing approval for any of our productcandidates, we expect to incur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. Accordingly,we will need to obtain substantial additional funding in connection with our continuing operations. If we are unable to raise capital when needed or onattractive terms, we would be forced to delay, reduce or eliminate our research and development programs or any future commercialization efforts. We expect our existing cash, cash equivalents and investments, will enable us to fund our current operating plan and capital expenditurerequirements into the first half of 2016. Our future capital requirements will depend on many factors, including:•the rate of progress, results and cost of completing of registration-directed trial of VS-6063 in mesothelioma; •assuming favorable clinical results, the cost, timing and outcome of our efforts to seek approval in mesothelioma in the United States andelsewhere in the world, including to fund the preparation and filing of regulatory submissions with the FDA and other regulatoryagencies worldwide; •the scope, progress and, results of our other ongoing and potential future clinical trials; •the extent to which we acquire or in-license other products and technologies;47Table of Contents•the costs, timing and outcome of regulatory review of our product candidates; •the costs of future commercialization activities, including product sales, marketing, manufacturing and distribution, for any of ourproduct candidates for which we receive marketing approval; •revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketingapproval; •the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defendingintellectual property-related claims; and •our ability to establish collaborations on favorable terms, if at all. Identifying potential product candidates and conducting preclinical testing and clinical trials is a time-consuming, expensive and uncertain processthat takes years to complete, and we may never generate the necessary data or results required to obtain marketing approval and achieve product sales. Inaddition, our product candidates, if approved, may not achieve commercial success. Our commercial revenues, if any, will be derived from sales ofproducts that we do not expect to be commercially available for many years, if at all. Accordingly, we will need to continue to rely on additionalfinancing to achieve our business objectives. Adequate additional financing may not be available to us on acceptable terms, or at all.Raising additional capital may cause dilution to our stockholders, restrict our operations or require us to relinquish rights to our technologies orproduct candidates. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equityofferings, debt financings, collaborations, strategic alliances and licensing arrangements. We do not have any committed external source of funds. To theextent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will bediluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debtfinancing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurringadditional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquishvaluable rights to our technologies, future revenue streams, research programs or product candidates or to grant licenses on terms that may not befavorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce orterminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwiseprefer to develop and market ourselves.RISKS RELATED TO THE DISCOVERY, DEVELOPMENT AND COMMERCIALIZATION OF OUR PRODUCT CANDIDATESOur approach to the discovery and development of product candidates that target CSCs is unproven, and we do not know whether we will be ableto develop any products of commercial value. Our scientific approach focuses on using proprietary technology to create a stable population of CSCs in the laboratory that we then use to screenfor and identify product candidates targeting these CSCs. Research on CSCs is an emerging field and, consequently, there is ongoing debate regardingthe existence of CSCs, whether the appropriate nomenclature to refer to these cells is cancer stem cells,48Table of Contentstumor-initiating cells or another term and the importance of these cells as an underlying cause of tumor recurrence and metastasis. Although there is general consensus that some cancer cells have tumor- initiating capacity, there also is some debate in the scientific communityregarding the defining characteristics of these cells, which we call CSCs, and the origin of these cells. Some believe that normal adult stem cells mutateand transform into CSCs. Others believe that all cancer cells have tumor-initiating capabilities, these capabilities cannot be attributed to a factor intrinsicto a particular cell and, therefore, a definitive CSC cannot be isolated or targeted. We believe that the discovery by our scientific co-founders of the linkbetween the epithelial-to-mesenchymal transition, or EMT, and the emergence of cancer stem cells is one way a cancer cell can transition to a CSC, butthis view is not universally accepted. Even if our beliefs regarding the existence, characteristics and function of CSCs are correct, any drugs that we develop may not effectively targetCSCs. We do not believe that any drugs that target CSCs have been successfully developed to date for the treatment of cancer. While we are currentlyconducting clinical trials for product candidates that we believe target CSCs, we may not ultimately be successful in demonstrating their safety andefficacy.We may not be successful in our efforts to identify or discover additional potential product candidates. A key element of our strategy is to identify and test additional compounds that target CSCs in a variety of different types of cancer. A significantportion of the research that we are conducting involves new compounds, new uses of existing compounds and new and unproven drug discoverymethods, including our proprietary technology. The drug discovery that we are conducting using our EMT technology may not be successful inidentifying compounds that are useful in treating cancer. Our research programs may initially show promise in identifying potential product candidates,yet fail to yield product candidates for clinical development for a number of reasons, including:•the research methodology used may not be successful in identifying potential product candidates; or •potential product candidates may, on further study, be shown to have harmful side effects or other characteristics that indicate that theyare unlikely to be products that will receive marketing approval and achieve market acceptance. In particular, because our EMT technology induces the EMT process to create a stable population of CSCs, it is possible that these stable CSCsmay not react in precisely the same manner as naturally occurring CSCs when treated with a particular product candidate. As a result, a productcandidate that shows initial promise in targeting our stable population of CSCs may not have the same effect on tumors with naturally occurring CSCs. Research programs to identify new product candidates require substantial technical, financial and human resources. We may choose to focus ourefforts and resources on a potential product candidate that ultimately proves to be unsuccessful. If we are unable to identify suitable compounds for preclinical and clinical development, we will not be able to obtain product revenues in futureperiods, which likely would result in significant harm to our financial position and adversely impact our stock price.We may not be successful in obtaining necessary rights to compounds and product candidates for our development pipeline through acquisitionsand in-licenses. Because we are screening a range of compounds, including compounds with proprietary rights held by third parties, for their activity againstCSCs, the growth of our business will depend in significant part on our ability to acquire or in-license rights to these compounds. However, we may beunable to49Table of Contentsacquire or in-license any compounds or product candidates from third parties that we identify using our proprietary EMT technology or otherwise. Thelicensing and acquisition of proprietary compounds is a competitive area, and a number of more established companies are also pursuing strategies tolicense or acquire compounds and product candidates that we may consider attractive. These established companies may have a competitive advantageover us due to their size, cash resources and greater clinical development and commercialization capabilities. The Whitehead Institute and affiliated parties have retained the right to use the EMT technology that we license from it for research, teaching andeducational purposes and could seek to license to third parties any intellectual property rights that it discovers using the EMT technology while pursuingthese purposes. Pursuant to our drug discovery platform license agreement with the Whitehead Institute, we will have an opportunity, subject to theWhitehead Institute's obligations under any third-party research funding agreements, to negotiate a license to any such intellectual property under thedrug discovery platform license agreement that is developed or conceived on or prior to a specified date in Robert Weinberg's laboratory at theWhitehead Institute. Our failure to reach an agreement with either the Broad Institute or the Whitehead Institute for any applicable intellectual propertycould result in a third party acquiring the related rights. In addition, companies that perceive us to be a competitor may be unwilling to assign or license rights to us. We also may be unable to license oracquire the relevant compound or product candidate on terms that would allow us to make an appropriate return on our investment. In addition, weexpect competition for acquisition and in-licensing product candidates that are attractive to us may increase in the future, especially if our approach oftargeting CSCs gains greater scientific acceptance, which may mean fewer suitable opportunities for us as well as higher acquisition or licensing prices.If we are unable to successfully obtain rights to suitable compounds or product candidates, our business, financial condition and prospects for growthcould suffer.Preclinical testing and clinical trials of our product candidates may not be successful. If we are unable to commercialize our product candidates orexperience significant delays in doing so, our business will be materially harmed. We have invested a significant portion of our efforts and financial resources in the identification, preclinical and early clinical development of drugsthat target CSCs. We are conducting clinical trials of our lead product candidate, VS-6063, in multiple indications, including a registration-directed trialin mesothelioma. Our other product candidates are in earlier stage clinical trials. Our ability to generate product revenues, which we do not expect willoccur for many years, if ever, will depend heavily on the successful development and eventual commercialization of our product candidates, particularlyVS-6063. The success of our product candidates will depend on several factors, including the following:•successful completion of these clinical trials; •receipt of marketing approvals from applicable regulatory authorities, including pricing approvals where required; •establishing commercial manufacturing capabilities or making arrangements with third-party manufacturers; •obtaining and maintaining patent and trade secret protection and regulatory exclusivity for our product candidates; •establishing commercial capabilities, including hiring and training a sales force, and launching commercial sales of the products, if andwhen approved, whether alone or in collaboration with others;50Table of Contents•acceptance of the products, if and when approved, by patients, the medical community and third-party payors; •effectively competing with other therapies; and •a continued acceptable safety profile of the products following approval. If we do not achieve one or more of these factors in a timely manner or at all, we could experience significant delays or an inability to successfullycommercialize our product candidates, which would materially harm our business.If clinical trials of our product candidates fail to demonstrate safety and efficacy to the satisfaction of regulatory authorities or do not otherwiseproduce positive results, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the developmentand commercialization of our product candidates. Before obtaining marketing approval from regulatory authorities for the sale of our product candidates, we must complete extensive clinical trials todemonstrate the safety and efficacy of our product candidates in humans. Clinical testing is expensive, difficult to design and implement, can take manyyears to complete and is uncertain as to outcome. A failure of one or more clinical trials can occur at any stage of testing. The outcome of preclinicaltesting and early clinical trials may not be predictive of the success of later clinical trials, and interim results of a clinical trial do not necessarily predictfinal results. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believedtheir product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of theirproducts. For example, standard measures of clinical activity with respect to solid tumors, such as Response Criteria in Solid Tumors, or RECIST,measurement guidelines, which are based on gross changes in the size of tumor lesions, may not be sufficient to detect the targeting of CSCs by ourproduct candidates. We may experience numerous unforeseen events during, or as a result of, clinical trials that could delay or prevent our ability to receive marketingapproval or commercialize our product candidates, including:•regulators or institutional review boards may not authorize us or our investigators to commence a clinical trial or conduct a clinical trial ata prospective trial site; •we may have delays in reaching or fail to reach agreement on acceptable clinical trial contracts or clinical trial protocols with prospectivetrial sites; •clinical trials of our product candidates may produce negative or inconclusive results, and we may decide, or regulators may require us,to conduct additional clinical trials or abandon product development programs; •the number of patients required for clinical trials of our product candidates may be larger than we anticipate, enrollment in these clinicaltrials may be slower than we anticipate or participants may drop out of these clinical trials at a higher rate than we anticipate; •our third-party contractors may fail to comply with regulatory requirements or meet their contractual obligations to us in a timely manner,or at all; •we might have to suspend or terminate clinical trials of our product candidates for various reasons, including a finding that theparticipants are being exposed to unacceptable health risks; •regulators or institutional review boards may require that we or our investigators suspend or terminate clinical research for variousreasons, including noncompliance with regulatory requirements or a finding that the participants are being exposed to unacceptable healthrisks;51Table of Contents•the cost of clinical trials of our product candidates may be greater than we anticipate; •the supply or quality of our product candidates or other materials necessary to conduct clinical trials of our product candidates may beinsufficient or inadequate; and •our product candidates may have undesirable side effects or other unexpected characteristics, causing us or our investigators, regulatorsor institutional review boards to suspend or terminate the trials. If we are required to conduct additional clinical trials or other testing of our product candidates beyond those that we currently contemplate, if weare unable to successfully complete clinical trials of our product candidates or other testing, if the results of these trials or tests are not positive or areonly modestly positive or if there are safety concerns, we may:•be delayed in obtaining marketing approval for our product candidates; •not obtain marketing approval at all; •obtain approval for indications or patient populations that are not as broad as intended or desired; •obtain approval with labeling that includes significant use or distribution restrictions or safety warnings, including boxed warnings; •be subject to additional post-marketing testing requirements; or •have the product removed from the market after obtaining marketing approval. We are currently conducting a registration-directed trial of VS-6063 in mesothelioma. We have interacted with regulatory agencies in the US, UKand ten other countries to discuss this trial. Based on these discussions, we believe that positive results from this trial, if obtained, may enable us to seekregulatory approval for VS-6063. However, the FDA and foreign regulatory authorities may determine that the results from this trial do not supportregulatory approval and may require us to conduct an additional clinical trial or trials. If these agencies take such a position, the costs of development ofVS-6063 in mesothelioma could increase materially and its potential market introduction could be delayed. The regulatory agencies could also requirethat we conduct additional clinical, nonclinical or manufacturing validation studies and submit that data before it will consider an NDA application. Our product development costs will also increase if we experience delays in testing or marketing approvals. We do not know whether any clinicaltrials will begin as planned, will need to be restructured or will be completed on schedule, or at all. Significant clinical trial delays also could shorten anyperiods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to marketbefore we do and impair our ability to successfully commercialize our product candidates and may harm our business and results of operations.If we experience delays or difficulties in the enrollment of patients in clinical trials, our receipt of necessary regulatory approvals could be delayedor prevented. We may not be able to initiate or continue clinical trials for our product candidates if we are unable to locate and enroll a sufficient number ofeligible patients to participate in these trials as required by the U.S. Food and Drug Administration, or FDA, or similar regulatory authorities outside theUnited States. In addition, there are a number of ongoing clinical trials for product candidates treating cancer. Patients who would otherwise be eligiblefor our clinical trials may instead enroll in clinical trials of our competitors' product candidates, particularly if they view such treatments to be moreconventional and established.52Table of Contents Patient enrollment is affected by other factors including:•severity of the disease under investigation; •eligibility criteria for the study in question; •perceived risks and benefits of the product candidate under study; •efforts to facilitate timely enrollment in clinical trials; •patient referral practices of physicians; •the ability to monitor patients adequately during and after treatment; and •proximity and availability of clinical trial sites for prospective patients. For example, we may have difficulty enrolling a sufficient number of patients in our registration-directed trial of VS-6063 in mesothelioma due tothe relatively small patient population and the rapid progression of the disease. Our inability to enroll a sufficient number of patients for our clinical trials would result in significant delays or may require us to abandon one ormore clinical trials altogether. Enrollment delays in our clinical trials may result in increased development costs for our product candidates, which wouldcause the value of our company to decline and limit our ability to obtain additional financing.If serious adverse or unexpected side effects are identified during the development of our product candidates, we may need to abandon or limit ourdevelopment of some of our product candidates. All of our product candidates are in preclinical development or early stages of clinical development and their risk of failure is high. It is impossibleto predict when or if any of our product candidates will prove effective or safe in humans or will receive marketing approval. If our product candidatesare associated with undesirable side effects or have characteristics that are unexpected, we may need to abandon their development or limit developmentto certain uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe or more acceptable from arisk-benefit perspective. Many compounds that initially showed promise in early stage testing for treating cancer have later been found to cause sideeffects that prevented further development of the compound.If we fail to obtain regulatory approval in jurisdictions outside the United States, we will not be able to market our products in those jurisdictions. We intend to seek regulatory approval for our product candidates in a number of countries outside of the United States and expect that thesecountries will be important markets for our products, if approved. Marketing our products in these countries will require separate regulatory approvalsin each market and compliance with numerous and varying regulatory requirements. The regulations that apply to the conduct of clinical trials andapproval procedures vary from country to country and may require additional testing. Moreover, the time required to obtain approval may differ fromthat required to obtain FDA approval. In addition, in many countries outside the United States, drugs must be approved for reimbursement before it canbe approved for sale in that country. Approval by the FDA does not ensure approval by regulatory authorities in other countries or jurisdictions, andapproval by one foreign regulatory authority does not ensure approval by regulatory authorities in other foreign countries or by the FDA. The foreignregulatory approval process may include all of the risks associated with obtaining FDA approval. We may not obtain foreign regulatory approvals on atimely basis, if at all. We may not be able to file for regulatory approvals and may not receive necessary approvals to commercialize our products in anyforeign market.53Table of ContentsWe may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates orindications that may be more profitable or for which there is a greater likelihood of success. Because we have limited financial and managerial resources, we focus on research programs and product candidates that we identify for specificindications. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to havegreater commercial potential. Our resource allocation decisions may cause us to fail to capitalize on viable commercial products or profitable marketopportunities. Our spending on current and future research and development programs and product candidates for specific indications may not yield anycommercially viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we mayrelinquish valuable rights to that product candidate through collaboration, licensing or other royalty arrangements in cases in which it would have beenmore advantageous for us to retain sole development and commercialization rights to such product candidate.If we are unable to successfully develop companion diagnostics for our therapeutic product candidates, or experience significant delays in doingso, we may not realize the full commercial potential of our therapeutics. We plan to develop companion diagnostics for our therapeutic product candidates. There has been limited success to date industry wide indeveloping these types of companion diagnostics. To be successful, we would need to address a number of scientific, technical and logistical challenges.We have only recently initiated development of companion diagnostics. We have limited experience in the development of diagnostics and may not besuccessful in developing appropriate diagnostics to pair with any of our therapeutic product candidates that receive marketing approval. Companiondiagnostics are subject to regulation by the FDA and similar regulatory authorities outside the United States as medical devices and require separateregulatory approval prior to commercialization. Given our limited experience in developing diagnostics, we expect to rely in part on third parties for theirdesign and manufacture. For example, we have entered into agreements with the Laboratory Corporation of America Holdings to assist us in validatingbiomarkers for VS-6063, and establishing, validating and performing a test for merlin protein being used for stratification in the COMMAND study,and in the development of an applicable companion diagnostic, if necessary. If we or any third parties that we engage to assist us, are unable tosuccessfully develop companion diagnostics for our therapeutic product candidates, or experience delays in doing so:•the development of our therapeutic product candidates may be adversely affected if we are unable to appropriately select patients forenrollment in our clinical trials; •our therapeutic product candidates may not receive marketing approval if safe and effective use of a therapeutic product candidatedepends on an in vitro diagnostic; and •we may not realize the full commercial potential of any therapeutics that receive marketing approval if, among other reasons, we areunable to appropriately select patients who are likely to benefit from therapy with our drugs. As a result, our business would be harmed, possibly materially.Even if any of our product candidates receive marketing approval, they may fail to achieve the degree of market acceptance by physicians, patients,healthcare payors and others in the medical community necessary for commercial success. If any of our product candidates receive marketing approval, they may nonetheless fail to gain sufficient market acceptance by physicians, patients,healthcare payors and others in the medical community. For example, current cancer treatments like chemotherapy and radiation therapy are well54Table of Contentsestablished in the medical community, and doctors may continue to rely on these treatments. If our product candidates do not achieve an adequate levelof acceptance, we may not generate significant product revenues and we may not become profitable. The degree of market acceptance of our productcandidates, if approved for commercial sale, will depend on a number of factors, including:•efficacy and potential advantages compared to alternative treatments; •the ability to offer our products for sale at competitive prices; •convenience and ease of administration compared to alternative treatments; •the willingness of the target patient population to try new therapies and of physicians to prescribe these therapies; •the strength of marketing and distribution support; •sufficient third-party coverage or reimbursement; and •the prevalence and severity of any side effects.If, in the future, we are unable to establish sales and marketing capabilities or enter into agreements with third parties to sell and market ourproduct candidates, we may not be successful in commercializing our product candidates if and when they are approved. We do not have a sales or marketing infrastructure and have no experience in the sale, marketing or distribution of pharmaceutical products. Toachieve commercial success for any approved product, we must either develop a sales and marketing organization or outsource these functions to thirdparties. In the future, we may choose to build a focused sales and marketing infrastructure to market or co-promote some of our product candidates ifand when they are approved. There are risks involved with both establishing our own sales and marketing capabilities and entering into arrangements with third parties toperform these services. For example, recruiting and training a sales force is expensive and time consuming and could delay any product launch. If thecommercial launch of a product candidate for which we recruit a sales force and establish marketing capabilities is delayed or does not occur for anyreason, we would have prematurely or unnecessarily incurred these commercialization expenses. This may be costly, and our investment would be lostif we cannot retain or reposition our sales and marketing personnel. Factors that may inhibit our efforts to commercialize our products on our own include:•our inability to recruit and retain adequate numbers of effective sales and marketing personnel; •the inability of sales personnel to obtain access to physicians or persuade adequate numbers of physicians to prescribe any futureproducts; •the lack of complementary products to be offered by sales personnel, which may put us at a competitive disadvantage relative tocompanies with more extensive product lines; and •unforeseen costs and expenses associated with creating an independent sales and marketing organization. If we enter into arrangements with third parties to perform sales, marketing and distribution services, our product revenues or the profitability ofthese product revenues to us are likely to be lower than if we were to market and sell any products that we develop ourselves. In addition, we may notbe successful in entering into arrangements with third parties to sell and market our product candidates or may be unable to do so on terms that arefavorable to us. We likely will have little control over such third parties, and any of them may fail to devote the necessary resources and attention to selland market our products effectively. If we do not establish sales and marketing capabilities successfully,55Table of Contentseither on our own or in collaboration with third parties, we will not be successful in commercializing our product candidates.We face substantial competition, which may result in others discovering, developing or commercializing products before or more successfully thanwe do. The development and commercialization of new drug products is highly competitive. We face competition with respect to our current productcandidates, and will face competition with respect to any product candidates that we may seek to develop or commercialize in the future, from majorpharmaceutical companies, specialty pharmaceutical companies and biotechnology companies worldwide. There are a number of large pharmaceuticaland biotechnology companies that currently market and sell products or are pursuing the development of products for the treatment of the diseaseindications for which we are developing our product candidates. Some of these competitive products and therapies are based on scientific approachesthat are the same as or similar to our approach, and others are based on entirely different approaches. Potential competitors also include academicinstitutions, government agencies and other public and private research organizations that conduct research, seek patent protection and establishcollaborative arrangements for research, development, manufacturing and commercialization. We are developing our product candidates for the treatment of cancer. There are a variety of available therapies marketed for cancer. In many cases,these drugs are administered in combination to enhance efficacy. Some of these drugs are branded and subject to patent protection, and others areavailable on a generic basis. Many of these approved drugs are well established therapies and are widely accepted by physicians, patients and third-partypayors. Insurers and other third-party payors may also encourage the use of generic products. We expect that if our product candidates are approved,they will be priced at a significant premium over competitive generic products. This may make it difficult for us to achieve our business strategy ofusing our product candidates in combination with existing therapies or replacing existing therapies with our product candidates. There are also a number of products in clinical development by third parties to treat cancer by targeting CSCs. These companies include divisionsof large pharmaceutical companies, including Astellas Pharma US, Inc., Sanofi-Aventis US LLC, GlaxoSmithKline plc, Boehringer Ingelheim GmbH,Pfizer Inc, Celgene, Inc. and others. There are also biotechnology companies of various sizes that are developing therapies against CSCs, includingOncoMed Pharmaceuticals, Inc., Boston Biomedical, Inc (a division of Dainippon Sumitomo Corp.)., Stemline Therapeutics, Inc. and others. Ourcompetitors may develop products that are more effective, safer, more convenient or less costly than any that we are developing or that would render ourproduct candidates obsolete or non-competitive. In addition, our competitors may discover biomarkers that more efficiently measure CSCs than ourmethods, which may give them a competitive advantage in developing potential products. Our competitors may also obtain marketing approval from theFDA or other regulatory authorities for their products more rapidly than we may obtain approval for ours, which could result in our competitorsestablishing a strong market position before we are able to enter the market. Many of our competitors have significantly greater financial resources and expertise in research and development, manufacturing, preclinicaltesting, conducting clinical trials, obtaining regulatory approvals and marketing approved products than we do. Mergers and acquisitions in thepharmaceutical and biotechnology industries may result in even more resources being concentrated among a smaller number of our competitors. Smallerand other early stage companies may also prove to be significant competitors, particularly through collaborative arrangements with large and establishedcompanies. These third parties compete with us in recruiting and retaining qualified scientific and management personnel, establishing clinical trial sitesand patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.56Table of Contents In addition, to the extent that product or product candidates of our competitors demonstrate serious adverse side effects or are determined to beineffective in clinical trials, the development of our product candidates could be negatively impacted.Even if we are able to commercialize any product candidates, the products may become subject to unfavorable pricing regulations, third-partyreimbursement practices or healthcare reform initiatives, which would harm our business. The regulations that govern marketing approvals, pricing and reimbursement for new drug products vary widely from country to country. In theUnited States, recently passed legislation may significantly change the approval requirements in ways that could involve additional costs and causedelays in obtaining approvals. Some countries require approval of the sale price of a drug before it can be marketed. In many countries, the pricingreview period begins after marketing or product licensing approval is granted. In some foreign markets, prescription pharmaceutical pricing remainssubject to continuing governmental control even after initial approval is granted. As a result, we might obtain marketing approval for a product in aparticular country, but then be subject to price regulations that delay our commercial launch of the product, possibly for lengthy time periods, andnegatively impact the revenues we are able to generate from the sale of the product in that country. Adverse pricing limitations may hinder our ability torecoup our investment in one or more product candidates, even if our product candidates obtain marketing approval. Our ability to commercialize any products successfully also will depend in part on the extent to which reimbursement for these products and relatedtreatments will be available from government health administration authorities, private health insurers and other organizations. Government authoritiesand third-party payors, such as private health insurers and health maintenance organizations, decide which medications they will pay for and establishreimbursement levels. A primary trend in the U.S. healthcare industry and elsewhere is cost containment. Government authorities and third-party payorshave attempted to control costs by limiting coverage and the amount of reimbursement for particular medications. Increasingly, third-party payors arerequiring that drug companies provide them with predetermined discounts from list prices and are challenging the prices charged for medical products.We cannot be sure that reimbursement will be available for any product that we commercialize and, if reimbursement is available, the level ofreimbursement. Reimbursement may impact the demand for, or the price of, any product candidate for which we obtain marketing approval. Obtainingreimbursement for our products may be particularly difficult because of the higher prices often associated with drugs administered under the supervisionof a physician. If reimbursement is not available or is available only to limited levels, we may not be able to successfully commercialize any productcandidate for which we obtain marketing approval. There may be significant delays in obtaining reimbursement for newly approved drugs, and coverage may be more limited than the purposes forwhich the drug is approved by the FDA or similar regulatory authorities outside the United States. Moreover, eligibility for reimbursement does notimply that any drug will be paid for in all cases or at a rate that covers our costs, including research, development, manufacture, sale and distribution.Interim reimbursement levels for new drugs, if applicable, may also not be sufficient to cover our costs and may not be made permanent.Reimbursement rates may vary according to the use of the drug and the clinical setting in which it is used, may be based on reimbursement levelsalready set for lower cost drugs and may be incorporated into existing payments for other services. Net prices for drugs may be reduced by mandatorydiscounts or rebates required by government healthcare programs or private payors and by any future relaxation of laws that presently restrict imports ofdrugs from countries where they may be sold at lower prices than in the United States. Third-party payors often rely upon Medicare coverage policyand payment limitations in setting their own reimbursement policies. Our inability to promptly obtain coverage and profitable payment rates from bothgovernment-funded and private payors for any approved products57Table of Contentsthat we develop could have a material adverse effect on our operating results, our ability to raise capital needed to commercialize products and ouroverall financial condition.Product liability lawsuits against us could cause us to incur substantial liabilities and to limit commercialization of any products that we maydevelop. We face an inherent risk of product liability exposure related to the testing of our product candidates in human clinical trials and will face an evengreater risk if we commercially sell any products that we may develop. If we cannot successfully defend ourselves against claims that our productcandidates or products caused injuries, we will incur substantial liabilities. Regardless of merit or eventual outcome, liability claims may result in:•decreased demand for any product candidates or products that we may develop; •injury to our reputation and significant negative media attention; •withdrawal of clinical trial participants; •significant costs to defend the related litigation; •substantial monetary awards to trial participants or patients; •loss of revenue; and •the inability to commercialize any products that we may develop. We currently hold $5.0 million in product liability insurance coverage in the aggregate, with a per incident limit of $5.0 million, which may not beadequate to cover all liabilities that we may incur. We may need to increase our insurance coverage as we initiate additional clinical trials or upon thecommercialization of our product candidates, if ever. Insurance coverage is increasingly expensive. We may not be able to maintain insurance coverageat a reasonable cost or in an amount adequate to satisfy any liability that may arise.If we fail to comply with environmental, health and safety laws and regulations, we could become subject to fines or penalties or incur costs thatcould have a material adverse effect on the success of our business. We are subject to numerous environmental, health and safety laws and regulations, including those governing laboratory procedures and thehandling, use, storage, treatment and disposal of hazardous materials and wastes. Our operations involve the use of hazardous and flammable materials,including chemicals and biological materials. Our operations also produce hazardous waste products. We generally contract with third parties for thedisposal of these materials and wastes. We cannot eliminate the risk of contamination or injury from these materials. In the event of contamination orinjury resulting from our use of hazardous materials, we could be held liable for any resulting damages, and any liability could exceed our resources.We also could incur significant costs associated with civil or criminal fines and penalties. Although we maintain workers' compensation insurance to cover us for costs and expenses we may incur due to injuries to our employeesresulting from the use of hazardous materials, this insurance may not provide adequate coverage against potential liabilities. We do not maintaininsurance for environmental liability or toxic tort claims that may be asserted against us in connection with our storage or disposal of biological,hazardous or radioactive materials. In addition, we may incur substantial costs in order to comply with current or future environmental, health and safety laws and regulations. Thesecurrent or future laws and regulations may impair our research, development or production efforts. Failure to comply with these laws and regulationsalso may result in substantial fines, penalties or other sanctions.58Table of ContentsRISKS RELATED TO OUR DEPENDENCE ON THIRD PARTIESWe may depend on collaborations with third parties for the development and commercialization of our product candidates. If those collaborationsare not successful, we may not be able to capitalize on the market potential of these product candidates. We may seek third-party collaborators for the development and commercialization of our product candidates. Our likely collaborators for anycollaboration arrangements include large and mid-size pharmaceutical companies, regional and national pharmaceutical companies and biotechnologycompanies. If we do enter into any such arrangements with any third parties, we will likely have limited control over the amount and timing of resourcesthat our collaborators dedicate to the development or commercialization of our product candidates. Our ability to generate revenues from thesearrangements will depend on our collaborators' abilities to successfully perform the functions assigned to them in these arrangements. Collaborations involving our product candidates would pose the following risks to us:•collaborators have significant discretion in determining the efforts and resources that they will apply to these collaborations; •collaborators may not pursue development and commercialization of our product candidates or may elect not to continue or renewdevelopment or commercialization programs based on clinical trial results, changes in the collaborator's strategic focus or availablefunding or external factors such as an acquisition that diverts resources or creates competing priorities; •collaborators may delay clinical trials, provide insufficient funding for a clinical trial program, stop a clinical trial or abandon a productcandidate, repeat or conduct new clinical trials or require a new formulation of a product candidate for clinical testing; •collaborators could independently develop, or develop with third parties, products that compete directly or indirectly with our products orproduct candidates if the collaborators believe that competitive products are more likely to be successfully developed or can becommercialized under terms that are more economically attractive than ours; •a collaborator with marketing and distribution rights to one or more products may not commit sufficient resources to the marketing anddistribution of such product or products; •collaborators may not properly maintain or defend our intellectual property rights or may use our proprietary information in such a wayas to invite litigation that could jeopardize or invalidate our proprietary information or expose us to potential litigation; •disputes may arise between the collaborators and us that result in the delay or termination of the research, development orcommercialization of our products or product candidates or that result in costly litigation or arbitration that diverts management attentionand resources; and •collaborations may be terminated and, if terminated, may result in a need for additional capital to pursue further development orcommercialization of the applicable product candidates. Collaboration agreements may not lead to development or commercialization of product candidates in the most efficient manner or at all. If apresent or future collaborator of ours were to be involved in a business combination, the continued pursuit and emphasis on our product development orcommercialization program could be delayed, diminished or terminated.59Table of ContentsIf we are not able to establish collaborations, we may have to alter our development and commercialization plans. Our drug development programs and the potential commercialization of our product candidates will require substantial additional cash to fundexpenses. For some of our product candidates, we may decide to collaborate with pharmaceutical and biotechnology companies for the development andpotential commercialization of those product candidates. We face significant competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a collaboration will depend,among other things, upon our assessment of the collaborator's resources and expertise, the terms and conditions of the proposed collaboration and theproposed collaborator's evaluation of a number of factors. Those factors may include the design or results of clinical trials, the likelihood of approval bythe FDA or similar regulatory authorities outside the United States, the potential market for the subject product candidate, the costs and complexities ofmanufacturing and delivering such product candidate to patients, the potential of competing products, the existence of uncertainty with respect to ourownership of technology, which can exist if there is a challenge to such ownership without regard to the merits of the challenge and industry and marketconditions generally. The collaborator may also consider alternative product candidates or technologies for similar indications that may be available tocollaborate on and whether such a collaboration could be more attractive than the one with us for our product candidate. We may also be restricted underexisting license agreements from entering into agreements on certain terms with potential collaborators. Collaborations are complex and time-consumingto negotiate and document. In addition, there have been a significant number of recent business combinations among large pharmaceutical companiesthat have resulted in a reduced number of potential future collaborators. We may not be able to negotiate collaborations on a timely basis, on acceptable terms, or at all. If we are unable to do so, we may have to curtail thedevelopment of such product candidate, reduce or delay its development program or one or more of our other development programs, delay its potentialcommercialization or reduce the scope of any sales or marketing activities, or increase our expenditures and undertake development or commercializationactivities at our own expense. If we elect to increase our expenditures to fund development or commercialization activities on our own, we may need toobtain additional capital, which may not be available to us on acceptable terms or at all. If we do not have sufficient funds, we may not be able to furtherdevelop our product candidates or bring them to market and generate product revenue.We rely in part on third parties to conduct our clinical trials and preclinical testing, and if they do not properly and successfully perform theirobligations to us, we may not be able to obtain regulatory approvals for our product candidates.. We rely on third parties, such as contract research organizations, clinical data management organizations, medical institutions and clinicalinvestigators, to conduct our clinical trials. For example, we currently rely on third party CROs to conduct our registration-directed trial of VS-6063 inmesothelioma, our Phase 1b trial of VS-6063 in combination with weekly paclitaxel for patients with ovarian cancer, our Phase 2 study of VS-6063 inpatients with non-small cell lung cancer, our Phase 1 trial of VS-6063 in Japan in patients with solid tumors and our Phase 1 trials of VS-4718 and VS-5584 in patients with advanced cancers. We compete with many other companies for the resources of these third parties. Any of these third parties mayterminate their engagements with us at any time. If we need to enter into alternative arrangements, it would delay our product development activities andultimately the commercialization of our product candidates. Our reliance on these third parties for research and development activities will reduce our control over these activities but will not relieve us of ourresponsibilities. For example, we will remain responsible for ensuring that each of our clinical trials is conducted in accordance with the general60Table of Contentsinvestigational plan and protocols for the trial. Moreover, the FDA requires us to comply with standards, commonly referred to as Good ClinicalPractices, or GCP, for conducting, recording and reporting the results of clinical trials to assure that data and reported results are credible and accurateand that the rights, integrity and confidentiality of trial participants are protected. Regulatory authorities enforce these GCP requirements throughperiodic inspections of trial sponsors, principal investigators and trial sites. If we or any of our CROs fail to comply with applicable GCP requirements,the clinical data generated in our clinical trials may be deemed unreliable and the FDA or other regulatory authorities may require us to performadditional clinical trials before approving our marketing applications. We cannot assure you that upon inspection by a given regulatory authority, suchregulatory authority will determine that any of our clinical trials comply with GCP requirements. We also are required to register ongoing clinical trialsand post the results of completed clinical trials on a government-sponsored database, ClinicalTrials.gov, within certain timeframes. Failure to do so canresult in fines, adverse publicity and civil and criminal sanctions. If these third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our clinical trials in accordance withregulatory requirements or our stated protocols, we will not be able to obtain, or may be delayed in obtaining, marketing approvals for our productcandidates and will not be able to, or may be delayed in our efforts to, successfully commercialize our product candidates.We contract with third parties for the manufacture of our product candidates and for compound formulation research, and these third partiesmay not perform satisfactorily. We do not have any manufacturing facilities or personnel. We currently obtain all of our supply of our product candidates for clinical developmentfrom third-party manufacturers or third-party collaborators, and we expect to continue to rely on third parties for the manufacture of clinical and, ifnecessary, commercial quantities of our product candidates. In addition, we current rely on third parties for the development of various formulations ofour product candidates. We obtain our supplies from these manufacturers on a purchase order basis, and we do not have any long term supplyagreements in place. This reliance on third parties increases the risk that we will not have sufficient quantities of our product candidates or suchquantities at an acceptable cost or quality, which could delay, prevent or impair our development or commercialization efforts. Any of these third parties may terminate their engagement with us at any time. We do not currently have arrangements in place for redundantsupply or a second source for bulk drug substance. Even if we are able to establish agreements with third-party manufacturers, reliance on third-partymanufacturers entails additional risks, including:•reliance on the third party for regulatory compliance and quality assurance; •the possible breach of the manufacturing agreement by the third party, including the misappropriation of our proprietary information,trade secrets and know-how; •the possible termination or nonrenewal of the agreement by the third party at a time that is costly or inconvenient for us; and •disruptions to the operations of our manufacturers or suppliers caused by conditions unrelated to our business or operations, includingthe bankruptcy of the manufacturer or supplier or a catastrophic event affecting our manufacturers or suppliers. Third-party manufacturers may not be able to comply with current good manufacturing practices, or cGMP, regulations or similar regulatoryrequirements outside the United States. Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result insanctions being imposed on us, including fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, license revocation, seizuresor recalls of product candidates or products, operating61Table of Contentsrestrictions and criminal prosecutions, any of which could significantly and adversely affect supplies of our products and harm our business and resultsof operations. Any products that we may develop may compete with other product candidates and products for access to manufacturing facilities. There are alimited number of manufacturers that operate under cGMP regulations and that might be capable of manufacturing for us. If our current contract manufacturers cannot perform as agreed, we may be required to replace that manufacturer. Although we believe that thereare several potential alternative manufacturers who could manufacture our product candidates, we may incur added costs and delays in identifying andqualifying any such replacement, as well as producing the drug product. In addition, we have to enter into technical transfer agreements and share ourknow-how with the third-party manufacturers, which can be time-consuming and may result in delays. Our current and anticipated future dependence upon others for the manufacture of our product candidates or products may adversely affect ourfuture profit margins and our ability to commercialize any products that receive marketing approval on a timely and competitive basis.RISKS RELATED TO OUR INTELLECTUAL PROPERTYIf we fail to comply with our obligations under our intellectual property licenses with third parties, we could lose license rights that are importantto our business. We are a party to a number of intellectual property license agreements with third parties, including the Whitehead Institute, The Scripps ResearchInsitute, or Scripps, and Pfizer, Inc., or Pfizer, and expect to enter into additional license agreements in the future. In particular, we have exclusivelylicensed rights to our lead product candidate, VS-6063, from Pfizer. Our existing license agreements impose, and we expect that future licenseagreements will impose, various diligence, milestone payment, royalty, insurance and other obligations on us. For example, under our licenseagreements with the Whitehead Institute, Scripps, and Pfizer, we are required to use commercially reasonable efforts to develop and commercializelicensed products under the agreement and to satisfy other specified obligations. If we fail to comply with our obligations under these licenses, ourlicensors may have the right to terminate these license agreements, in which event we might not be able to market any product that is covered by theseagreements, or to convert the exclusive licenses to non-exclusive licenses, which could materially adversely affect the value of the product candidatebeing developed under these license agreements. Termination of these license agreements or reduction or elimination of our licensed rights may result inour having to negotiate new or reinstated licenses with less favorable terms. If the Whitehead Institute were to terminate its drug discovery platformlicense agreement with us for any reason, we would lose access to the EMT technology and the ability to use the stable population of CSCs for high-throughput screening. If Scripps were to terminate its license agreement with us for any reason, we would lose our rights to VS-4718. If Pfizer were toterminate its license agreement with us for any reason, we would lose our rights to VS-6063, which is our lead product candidate.If we are unable to obtain and maintain patent protection for our technology and products, or if our licensors are unable to obtain and maintainpatent protection for the technology or products that we license from them, or if the scope of the patent protection obtained is not sufficientlybroad, our competitors could develop and commercialize technology and products similar or identical to ours, and our ability to successfullycommercialize our technology and products may be adversely affected. Our success depends in large part on our and our licensors' ability to obtain and maintain patent protection in the United States and other countrieswith respect to our proprietary technology and products. We and our licensors seek to protect our proprietary position by filing patent applications in theUnited States and abroad related to our novel technologies and products that are important to our business. We cannot be certain that any patents willissue with claims that cover our proprietary technology or product candidates.62Table of Contents The patent prosecution process is expensive and time-consuming, and we may not be able to file and prosecute all necessary or desirable patentapplications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research and developmentoutput before it is too late to obtain patent protection. Moreover, in some circumstances, we do not have the right to control the preparation, filing andprosecution of patent applications, or to maintain the patents, covering technology or products that we license from third parties and are reliant on ourlicensors. For example, we do not control the prosecution of the patent applications licensed to us under our agreements with the Whitehead Institute orthose patent applications owned by Scripps .. Therefore, we cannot be certain that these patents and applications will be prosecuted and enforced in amanner consistent with the best interests of our business. If such licensors fail to maintain such patents, or lose rights to those patents, the rights wehave licensed may be reduced or eliminated. The patent position of biotechnology and pharmaceutical companies generally is highly uncertain, involves complex legal and factual questions andhas in recent years been the subject of much litigation. As a result, the issuance, scope, validity, enforceability and commercial value of our and ourlicensors' patent rights are highly uncertain. Our and our licensors' pending and future patent applications may not result in patents being issued whichprotect our technology or products or which effectively prevent others from commercializing competitive technologies and products. Changes in eitherthe patent laws or interpretation of the patent laws in the United States and other countries may diminish the value of our patents or narrow the scope ofour patent protection. The laws of foreign countries may not protect our rights to the same extent as the laws of the United States. Publications of discoveries in thescientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not publisheduntil 18 months after filing, or in some cases not at all. Therefore we cannot be certain that we or our licensors were the first to make the inventionsclaimed in our owned or licensed patents or pending patent applications, or that we or our licensors were the first to file for patent protection of suchinventions. Assuming the other requirements for patentability are met, in the United States, for patents that have an effective filing date prior to March 15,2013, the first to make the claimed invention is entitled to the patent, while outside the United States, the first to file a patent application is entitled to thepatent. In March 2013, the United States transitioned to a first inventor to file system in which, assuming the other requirements for patentability aremet, the first inventor to file a patent application will be entitled to the patent. We may be subject to a third party preissuance submission of prior art tothe U.S. Patent and Trademark Office, or become involved in opposition, derivation, reexamination, inter parties review or interference proceedingschallenging our patent rights or the patent rights of others. An adverse determination in any such submission, proceeding or litigation could reduce thescope of, or invalidate, our patent rights, allow third parties to commercialize our technology or products and compete directly with us, without paymentto us, or result in our inability to manufacture or commercialize products without infringing third-party patent rights. Even if our owned and licensed patent applications issue as patents, they may not issue in a form that will provide us with any meaningfulprotection, prevent competitors from competing with us or otherwise provide us with any competitive advantage. Our competitors may be able tocircumvent our owned or licensed patents by developing similar or alternative technologies or products in a non-infringing manner. The issuance of a patent is not conclusive as to its inventorship, scope, validity or enforceability, and our owned and licensed patents may bechallenged in the courts or patent offices in the United States and abroad. Such challenges may result in loss of exclusivity or freedom to operate or inpatent claims being narrowed, invalidated or held unenforceable, which could limit our ability to stop others from using or commercializing similar oridentical technology and products, or limit the duration of the63Table of Contentspatent protection of our technology and products. Given the amount of time required for the development, testing and regulatory review of new productcandidates, patents protecting such candidates might expire before or shortly after such candidates are commercialized. As a result, our owned andlicensed patent portfolio may not provide us with sufficient rights to exclude others from commercializing products similar or identical to ours.We may become involved in lawsuits to protect or enforce our patents, which could be expensive, time consuming and unsuccessful. Competitors may infringe our patents. To counter infringement or unauthorized use, we may be required to file infringement claims, which can beexpensive and time consuming. In addition, in an infringement proceeding, a court may decide that a patent of ours is invalid or unenforceable, or mayrefuse to stop the other party from using the technology at issue on the grounds that our patents do not cover the technology in question. An adverseresult in any litigation proceeding could put one or more of our patents at risk of being invalidated or interpreted narrowly. Furthermore, because of thesubstantial amount of discovery required in connection with intellectual property litigation, there is a risk that some of our confidential information couldbe compromised by disclosure during this type of litigation. In addition, our licensors may have rights to file and prosecute such claims and we arereliant on them.Third parties may initiate legal proceedings alleging that we are infringing their intellectual property rights, the outcome of which would beuncertain and could have a material adverse effect on the success of our business. Our commercial success depends upon our ability and the ability of our collaborators to develop, manufacture, market and sell our productcandidates and use our proprietary technologies without infringing the proprietary rights of third parties. We have yet to conduct comprehensivefreedom-to-operate searches to determine whether our use of certain of the patent rights owned by or licensed to us would infringe patents issued tothird parties. We may become party to, or threatened with, future adversarial proceedings or litigation regarding intellectual property rights with respectto our products and technology, including interference proceedings before the U.S. Patent and Trademark Office. Third parties may assert infringementclaims against us based on existing patents or patents that may be granted in the future. If we are found to infringe a third party's intellectual propertyrights, we could be required to obtain a license from such third party to continue developing and marketing our products and technology. However, wemay not be able to obtain any required license on commercially reasonable terms or at all. Even if we were able to obtain a license, it could be non-exclusive, thereby giving our competitors access to the same technologies licensed to us. We could be forced, including by court order, to ceasecommercializing the infringing technology or product. In addition, we could be found liable for monetary damages. A finding of infringement couldprevent us from commercializing our product candidates or force us to cease some of our business operations, which could materially harm ourbusiness. Claims that we have misappropriated the confidential information or trade secrets of third parties could have a similar negative impact on ourbusiness.We may be subject to claims that our employees have wrongfully used or disclosed alleged trade secrets of their former employers. Many of our employees were previously employed at universities or other biotechnology or pharmaceutical companies, including our competitorsor potential competitors. Although we try to ensure that our employees do not use the proprietary information or know-how of others in their work forus, we may be subject to claims that we or these employees have used or disclosed intellectual property, including trade secrets or other proprietaryinformation, of any such employee's former employer. Litigation may be necessary to defend against these claims. If we fail in defending any such64Table of Contentsclaims, in addition to paying monetary damages, we may lose valuable intellectual property rights or personnel. Even if we are successful in defendingagainst such claims, litigation could result in substantial costs and be a distraction to management.Intellectual property litigation could cause us to spend substantial resources and distract our personnel from their normal responsibilities. Even if resolved in our favor, litigation or other legal proceedings relating to intellectual property claims may cause us to incur significant expenses,and could distract our technical and management personnel from their normal responsibilities. In addition, there could be public announcements of theresults of hearings, motions or other interim proceedings or developments and if securities analysts or investors perceive these results to be negative, itcould have a substantial adverse effect on the price of our common stock. Such litigation or proceedings could substantially increase our operatinglosses and reduce the resources available for development activities or any future sales, marketing or distribution activities. We may not have sufficientfinancial or other resources to adequately conduct such litigation or proceedings. Some of our competitors may be able to sustain the costs of suchlitigation or proceedings more effectively than we can because of their greater financial resources. Uncertainties resulting from the initiation andcontinuation of patent litigation or other proceedings could have a material adverse effect on our ability to compete in the marketplace.If we are unable to protect the confidentiality of our trade secrets, our business and competitive position would be harmed. In addition to seeking patents for some of our technology and products, we also rely on trade secrets, including unpatented know-how, technologyand other proprietary information, to maintain our competitive position. We seek to protect these trade secrets, in part, by entering into non-disclosureand confidentiality agreements with parties who have access to them, such as our employees, corporate collaborators, outside scientific collaborators,contract manufacturers, consultants, advisors and other third parties. We also enter into confidentiality and invention or patent assignment agreementswith our employees and consultants. Despite these efforts, any of these parties may breach the agreements and disclose our proprietary information,including our trade secrets, and we may not be able to obtain adequate remedies for such breaches. Enforcing a claim that a party illegally disclosed ormisappropriated a trade secret is difficult, expensive and time-consuming, and the outcome is unpredictable. In addition, some courts inside and outsidethe United States are less willing or unwilling to protect trade secrets. If any of our trade secrets were to be lawfully obtained or independentlydeveloped by a competitor, we would have no right to prevent them from using that technology or information to compete with us. If any of our tradesecrets were to be disclosed to or independently developed by a competitor, our competitive position would be harmed.RISKS RELATED TO REGULATORY APPROVAL OF OUR PRODUCT CANDIDATES AND OTHER LEGAL COMPLIANCEMATTERSIf we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize our productcandidates, and our ability to generate revenue will be materially impaired. Our product candidates and the activities associated with their development and commercialization, including their design, testing, manufacture,safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by theFDA and other regulatory agencies in the United States and by comparable authorities in other countries. Failure to obtain marketing approval for aproduct candidate will prevent us from commercializing the product candidate. We have not received approval to market any of our product candidatesfrom regulatory authorities in any jurisdiction. We have only limited experience in filing and supporting the applications necessary to gain marketingapprovals and expect to rely on third-party65Table of Contentscontract research organizations to assist us in this process. Securing FDA approval requires the submission of extensive preclinical and clinical data andsupporting information to the FDA for each therapeutic indication to establish the product candidate's safety and efficacy. Securing FDA approval alsorequires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the FDA. Our productcandidates may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities or othercharacteristics that may preclude our obtaining marketing approval or prevent or limit commercial use. The process of obtaining marketing approvals, both in the United States and abroad, is expensive, may take many years if additional clinical trialsare required, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity and novelty of theproduct candidates involved. Changes in marketing approval policies during the development period, changes in or the enactment of additional statutesor regulations, or changes in regulatory review for each submitted product application, may cause delays in the approval or rejection of an application.The FDA has substantial discretion in the approval process and may refuse to accept any application or may decide that our data is insufficient forapproval and require additional preclinical, clinical or other studies. In addition, varying interpretations of the data obtained from preclinical and clinicaltesting could delay, limit or prevent marketing approval of a product candidate. Any marketing approval we ultimately obtain may be limited or subjectto restrictions or post-approval commitments that render the approved product not commercially viable. If we experience delays in obtaining approval or if we fail to obtain approval of our product candidates, the commercial prospects for our productcandidates may be harmed and our ability to generate revenues will be materially impaired.Failure to obtain marketing approval in international jurisdictions would prevent our product candidates from being marketed abroad. We intend to seek regulatory approval for our product candidates in a number of countries outside of the United States and expect that thesecountries will be important markets for our products, if approved .In order to market and sell our products in the European Union and many otherjurisdictions, we or our third-party collaborators must obtain separate marketing approvals and comply with numerous and varying regulatoryrequirements. The approval procedure varies among countries and can involve additional testing. The time required to obtain approval may differsubstantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of the risksassociated with obtaining FDA approval. In addition, in many countries outside the United States, it is required that the product be approved forreimbursement before the product can be approved for sale in that country. We or these third parties may not obtain approvals from regulatoryauthorities outside the United States on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory authorities in othercountries or jurisdictions, and approval by one regulatory authority outside the United States does not ensure approval by regulatory authorities in othercountries or jurisdictions or by the FDA. We may not be able to file for marketing approvals and may not receive necessary approvals to commercializeour products in any market.We have received for orphan disease status for VS-6063 for mesothelioma in the United States and the European Union, but there can be noassurance that we will be able to prevent third parties from developing and commercializing products that are competitive to VS-6063. We received orphan drug designation in the U.S. and the EU for the use of VS-6063 in mesothelioma. If VS-6063 obtains marketingauthorization, it will receive orphan drug exclusivity. Orphan drug exclusivity grants seven years of marketing exclusivity under the Federal Food, Drugand Cosmetic Act and up to 10 years of marketing exclusivity in Europe. A competitor may receive orphan drug marketing authorization prior to us forthe same indication for which we are developing VS-6063.66Table of ContentsOther companies have received orphan drug designations for compounds other than VS-6063 for mesothelioma in the U.S. and EU. While the orphandrug exclusivity for VS-6063 would provide market exclusivity in the U.S., Europe, and other countries, we would not be able to exclude othercompanies from manufacturing and/or selling drugs using the same active ingredient for the same indication beyond that timeframe on the basis oforphan drug designation. Furthermore, the marketing exclusivity in Europe can be reduced from 10 years to six years if the initial designation criteriahave significantly changed since the market authorization of the orphan medicinal product. We cannot guarantee that another company also with orphandrug designation will not receive marketing authorization for the same indication before we do. If that were to happen, our applications for thatindication may not be approved until the competing company's period of exclusivity has expired. Even if we are the first to obtain marketingauthorization for an orphan drug indication, there are circumstances under which a competing product may be approved for the same indication duringthe seven-year period of marketing exclusivity, such as if the later product is shown to be clinically superior to our product, or if the later product is adifferent drug than VS-6063. Further, the seven-year marketing exclusivity would not prevent competitors from obtaining approval of the samecompound for other indications or the use of other types of drugs for the same use as the orphan drug.Any product candidate for which we obtain marketing approval could be subject to restrictions or withdrawal from the market and we may besubject to penalties if we fail to comply with regulatory requirements or if we experience unanticipated problems with our products, when and ifany of them are approved. Any product candidate for which we obtain marketing approval, along with the manufacturing processes, post-approval clinical data, labeling,advertising and promotional activities for such product, will be subject to continual requirements of and review by the FDA and other regulatoryauthorities. These requirements include submissions of safety and other post-marketing information and reports, registration and listing requirements,cGMP requirements relating to quality control, quality assurance and corresponding maintenance of records and documents, requirements regarding thedistribution of samples to physicians and recordkeeping. Even if marketing approval of a product candidate is granted, the approval may be subject tolimitations on the indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for costly post-marketing testing and surveillance to monitor the safety or efficacy of the product. The FDA closely regulates the post-approval marketing andpromotion of drugs to ensure drugs are marketed only for the approved indications and in accordance with the provisions of the approved labeling. TheFDA imposes stringent restrictions on manufacturers' communications regarding off-label use and if we do not market our products for their approvedindications, we may be subject to enforcement action for off-label marketing. In addition, later discovery of previously unknown problems with our products, manufacturers or manufacturing processes, or failure to complywith regulatory requirements, may yield various results, including:•restrictions on such products, manufacturers or manufacturing processes; •restrictions on the labeling or marketing of a product; •restrictions on product distribution or use; •requirements to conduct post-marketing clinical trials; •warning or untitled letters; •withdrawal of the products from the market; •refusal to approve pending applications or supplements to approved applications that we submit; •recall of products;67Table of Contents•fines, restitution or disgorgement of profits or revenue; •suspension or withdrawal of marketing approvals; •refusal to permit the import or export of our products; •product seizure; or •injunctions or the imposition of civil or criminal penalties.Our relationships with customers and third-party payors will be subject to applicable anti-kickback, fraud and abuse and other healthcare lawsand regulations, which could expose us to criminal sanctions, civil penalties, contractual damages, reputational harm and diminished profits andfuture earnings. Healthcare providers, physicians and third-party payors play a primary role in the recommendation and prescription of any product candidates forwhich we obtain marketing approval. Our future arrangements with third-party payors and customers may expose us to broadly applicable fraud andabuse and other healthcare laws and regulations that may constrain the business or financial arrangements and relationships through which we market,sell and distribute our products for which we obtain marketing approval. Restrictions under applicable federal and state healthcare laws and regulations,include the following:•the federal healthcare anti-kickback statute prohibits, among other things, persons from knowingly and willfully soliciting, offering,receiving or providing remuneration, directly or indirectly, in cash or in kind, to induce or reward either the referral of an individual for,or the purchase, order or recommendation of, any good or service, for which payment may be made under federal and state healthcareprograms such as Medicare and Medicaid; •the federal False Claims Act imposes criminal and civil penalties, including civil whistleblower or qui tam actions, against individuals orentities for knowingly presenting, or causing to be presented, to the federal government, claims for payment that are false or fraudulentor making a false statement to avoid, decrease or conceal an obligation to pay money to the federal government; •the federal Health Insurance Portability and Accountability Act of 1996, as amended by the Health Information Technology forEconomic and Clinical Health Act, imposes criminal and civil liability for executing a scheme to defraud any healthcare benefit programand also imposes obligations, including mandatory contractual terms, with respect to safeguarding the privacy, security and transmissionof individually identifiable health information; •the federal false statements statute prohibits knowingly and willfully falsifying, concealing or covering up a material fact or making anymaterially false statement in connection with the delivery of or payment for healthcare benefits, items or services; •the federal transparency requirements under the Health Care Reform Law requires manufacturers of drugs, devices, biologics andmedical supplies to report to the Department of Health and Human Services information related to physician payments and othertransfers of value and physician ownership and investment interests; and •analogous state laws and regulations, such as state anti-kickback and false claims laws, may apply to sales or marketing arrangementsand claims involving healthcare items or services reimbursed by non-governmental third-party payors, including private insurers, andsome state laws require pharmaceutical companies to comply with the pharmaceutical industry's voluntary compliance guidelines and therelevant compliance guidance promulgated by the federal government in addition to requiring drug manufacturers to report informationrelated to payments to physicians and other health care providers or marketing expenditures.68Table of Contents Efforts to ensure that our business arrangements with third parties will comply with applicable healthcare laws and regulations will involvesubstantial costs. It is possible that governmental authorities will conclude that our business practices may not comply with current or future statutes,regulations or case law involving applicable fraud and abuse or other healthcare laws and regulations. If our operations are found to be in violation ofany of these laws or any other governmental regulations that may apply to us, we may be subject to significant civil, criminal and administrativepenalties, damages, fines, exclusion from government funded healthcare programs, such as Medicare and Medicaid, and the curtailment or restructuringof our operations. If any of the physicians or other providers or entities with whom we expect to do business are found to be not in compliance withapplicable laws, they may be subject to criminal, civil or administrative sanctions, including exclusions from government funded healthcare programs.Recently enacted and future legislation may increase the difficulty and cost for us to obtain marketing approval of and commercialize our productcandidates and affect the prices we may obtain. In the United States and some foreign jurisdictions, there have been a number of legislative and regulatory changes and proposed changesregarding the healthcare system that could prevent or delay marketing approval of our product candidates, restrict or regulate post-approval activities andaffect our ability to profitably sell any product candidates for which we obtain marketing approval. In the United States, the Medicare Prescription Drug, Improvement, and Modernization Act of 2003, or Medicare Modernization Act, changed theway Medicare covers and pays for pharmaceutical products. The legislation expanded Medicare coverage for drug purchases by the elderly andintroduced a new reimbursement methodology based on average sales prices for physician administered drugs. In addition, this legislation providedauthority for limiting the number of drugs that will be covered in any therapeutic class. Cost reduction initiatives and other provisions of this legislationcould decrease the coverage and price that we receive for any approved products. While the Medicare Modernization Act applies only to drug benefitsfor Medicare beneficiaries, private payors often follow Medicare coverage policy and payment limitations in setting their own reimbursement rates.Therefore, any reduction in reimbursement that results from the Medicare Modernization Act may result in a similar reduction in payments from privatepayors. More recently, in March 2010, President Obama signed into law the Health Care Reform Law, a sweeping law intended to broaden access tohealth insurance, reduce or constrain the growth of healthcare spending, enhance remedies against fraud and abuse, add new transparency requirementsfor health care and health insurance industries, impose new taxes and fees on the health industry and impose additional health policy reforms. EffectiveOctober 1, 2010, the Health Care Reform Law revises the definition of "average manufacturer price" for reporting purposes, which could increase theamount of Medicaid drug rebates to states. Further, the new law imposes a significant annual fee on companies that manufacture or import brandedprescription drug products. Substantial new provisions affecting compliance have also been enacted, which may affect our business practices withhealth care practitioners. We will not know the full effects of the Health Care Reform Law until applicable federal and state agencies issue regulations orguidance under the new law. Although it is too early to determine the effect of the Health Care Reform Law, the new law appears likely to continue thepressure on pharmaceutical pricing, especially under the Medicare program, and may also increase our regulatory burdens and operating costs. Legislative and regulatory proposals have been made to expand post-approval requirements and restrict sales and promotional activities forpharmaceutical products. We cannot be sure whether additional legislative changes will be enacted, or whether the FDA regulations, guidance orinterpretations will be changed, or what the impact of such changes on the marketing approvals of our product candidates, if any, may be. In addition,increased scrutiny by the U.S. Congress of the FDA's69Table of Contentsapproval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketingtesting and other requirements.RISKS RELATED TO EMPLOYEE MATTERS AND MANAGING GROWTHOur future success depends on our ability to retain our chief executive officer and other key executives and to attract, retain and motivate qualifiedpersonnel. We are highly dependent on Christoph Westphal, our Executive Chairman, Robert Forrester, our President and Chief Executive Officer, andJoanna Horobin, our Chief Medical Officer, as well as the other principal members of our management and scientific teams, including our scientific co-founders, Robert Weinberg and Eric Lander. Although we have formal employment agreements with Christoph Westphal, Robert Forrester and JoannaHorobin, these agreements do not prevent them from terminating their employment with us at any time. We do not maintain "key person" insurance forany of our executives or other employees. The loss of the services of any of these persons could impede the achievement of our research, developmentand commercialization objectives. Recruiting and retaining qualified scientific, clinical, manufacturing and sales and marketing personnel will also be critical to our success. We maynot be able to attract and retain these personnel on acceptable terms given the competition among numerous pharmaceutical and biotechnologycompanies for similar personnel. We also experience competition for the hiring of scientific and clinical personnel from universities and researchinstitutions. In addition, we rely on consultants and advisors, including scientific and clinical advisors, to assist us in formulating our research anddevelopment and commercialization strategy. Our consultants and advisors, including our scientific co-founders, may be employed by employers otherthan us and may have commitments under consulting or advisory contracts with other entities that may limit their availability to us.We expect to expand our development, regulatory and future sales and marketing capabilities, and as a result, we may encounter difficulties inmanaging our growth, which could disrupt our operations. We expect to experience significant growth in the number of our employees and the scope of our operations, particularly in the areas of drugdevelopment, regulatory affairs and sales and marketing. To manage our anticipated future growth, we must continue to implement and improve ourmanagerial, operational and financial systems, expand our facilities and continue to recruit and train additional qualified personnel. Due to our limitedfinancial resources and the limited experience of our management team in managing a company with such anticipated growth, we may not be able toeffectively manage the expansion of our operations or recruit and train additional qualified personnel. The physical expansion of our operations maylead to significant costs and may divert our management and business development resources. Any inability to manage growth could delay the executionof our business plans or disrupt our operations.RISKS RELATED TO OUR COMMON STOCKOur executive officers, directors and principal stockholders maintain the ability to control all matters submitted to stockholders for approval. As of December 31, 2013, our executive officers, directors and stockholders who owned more than 5% of our outstanding common stock, in theaggregate, beneficially owned shares representing approximately 40% of our capital stock. As a result, if these stockholders chose to act together, theywould be able to control all matters submitted to our stockholders for approval, as well as our management and affairs. For example, these persons, ifthey chose to act together, would control the election of directors and approval of any merger, consolidation or sale of all or substantially all of ourassets. This concentration of voting power could delay or prevent an acquisition of our company on terms that other stockholders may desire.70Table of ContentsProvisions in our corporate charter documents and under Delaware law could make an acquisition of us, which may be beneficial to ourstockholders, more difficult and may prevent attempts by our stockholders to replace or remove our current management. Provisions in our corporate charter and our bylaws may discourage, delay or prevent a merger, acquisition or other change in control of us thatstockholders may consider favorable, including transactions in which you might otherwise receive a premium for your shares. These provisions couldalso limit the price that investors might be willing to pay in the future for shares of our common stock, thereby depressing the market price of ourcommon stock. In addition, because our board of directors is responsible for appointing the members of our management team, these provisions mayfrustrate or prevent any attempts by our stockholders to replace or remove our current management by making it more difficult for stockholders toreplace members of our board of directors. Among other things, these provisions:•establish a classified board of directors such that not all members of the board are elected at one time; •allow the authorized number of our directors to be changed only by resolution of our board of directors; •limit the manner in which stockholders can remove directors from the board; •establish advance notice requirements for stockholder proposals that can be acted on at stockholder meetings and nominations to ourboard of directors; •require that stockholder actions must be effected at a duly called stockholder meeting and prohibit actions by our stockholders by writtenconsent; •limit who may call stockholder meetings; •authorize our board of directors to issue preferred stock without stockholder approval, which could be used to institute a "poison pill"that would work to dilute the stock ownership of a potential hostile acquirer, effectively preventing acquisitions that have not beenapproved by our board of directors; and •require the approval of the holders of at least 75% of the votes that all our stockholders would be entitled to cast to amend or repealcertain provisions of our charter or bylaws. Moreover, because we are incorporated in Delaware, we are governed by the provisions of Section 203 of the Delaware General Corporation Law,which prohibits a person who owns in excess of 15% of our outstanding voting stock from merging or combining with us for a period of three yearsafter the date of the transaction in which the person acquired in excess of 15% of our outstanding voting stock, unless the merger or combination isapproved in a prescribed manner.The market price of our common stock has been, and may continue to be, highly volatile. Our stock price has been volatile. Since January 27, 2012, when we became a public company, the price for one share of our common stock hasreached a high of $18.82 and a low of $6.25. We cannot predict whether the price of our common stock will rise or fall. The market price for ourcommon stock may be influenced by many factors, including:•the success of competitive products or technologies; •results of clinical trials of our product candidates or those of our competitors; •regulatory or legal developments in the United States and other countries;71Table of Contents•developments or disputes concerning patent applications, issued patents or other proprietary rights; •the recruitment or departure of key personnel; •the level of expenses related to any of our product candidates or clinical development programs; •the results of our efforts to discover, develop, acquire or in-license additional product candidates or products; •actual or anticipated changes in estimates as to financial results, development timelines or recommendations by securities analysts; •variations in our financial results or those of companies that are perceived to be similar to us; •changes in the structure of healthcare payment systems; •market conditions in the pharmaceutical and biotechnology sectors; •general economic, industry and market conditions; and •the other factors described in this "Risk Factors" section. In addition, the stock market in general and the market for small pharmaceutical companies and biotechnology companies in particular haveexperienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of particularcompanies. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operatingperformance. In the past, following periods of volatility in the market, securities class-action litigation has often been instituted against companies. Suchlitigation, if instituted against us, could result in substantial costs and diversion of management's attention and resources, which could materially andadversely affect our business and financial condition.Because we do not anticipate paying any cash dividends on our capital stock in the foreseeable future, capital appreciation, if any, will be thesource of gain for our stockholders. We have never declared or paid cash dividends on our capital stock. We currently intend to retain all of our future earnings, if any, to finance thegrowth and development of our business. In addition, the terms of any future debt agreements may preclude us from paying dividends. As a result,capital appreciation, if any, of our common stock will be the sole source of gain for our stockholders for the foreseeable future.We are an "emerging growth company," and our election to delay adoption of new or revised accounting standards applicable to publiccompanies may result in our financial statements not being comparable to those of other public companies. As a result of this and other reduceddisclosure requirements applicable to emerging growth companies, our common stock may be less attractive to investors. We are an "emerging growth company," as defined in the Jumpstart Our Business Startups Act, or JOBS Act, and may remain an emerginggrowth company for up to five years, until December 31, 2017, although if the market value of our common stock that is held by non-affiliates exceeds$700 million as of any June 30 before that time or if we have annual gross revenues of $1 billion or more in any fiscal year, we would cease to be anemerging growth company as of December 31 of the applicable year. For so long as we remain an emerging growth company, we are permitted andintend to rely on exemptions from certain reporting requirements that are applicable to other public companies that are not emerging growth companies.These exemptions include not being required to comply with the auditor attestation requirements of Section 404 of the Sarbanes-Oxley Act of 2002, notbeing required to comply with any requirement that may be adopted by the Public Company Accounting Oversight Board regarding mandatory auditfirm rotation or a supplement to the auditor's report providing additional72Table of Contentsinformation about the audit and the financial statements, reduced disclosure obligations regarding executive compensation in our periodic reports andproxy statements, and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval ofany golden parachute payments not previously approved. Among other provisions, the JOBS Act provides that an emerging growth company can take advantage of the extended transition period providedin Section 7(a)(2)(B) of the Securities Act of 1933, as amended, or the Securities Act, for complying with new or revised accounting standards. Thisallows an emerging growth company to delay the adoption of certain accounting standards until those standards would otherwise apply to privatecompanies. We are electing to delay such adoption of new or revised accounting standards, and as a result, we may not comply with new or revisedaccounting standards on the relevant dates on which adoption of such standards is required for public companies that are not emerging growthcompanies. As a result of such election, our financial statements may not be comparable to the financial statements of other public companies. We cannot predict whether investors will find our common stock less attractive because we will rely on these exemptions. If some investors findour common stock less attractive as a result, there may be a less active trading market for our common stock and our stock price may be more volatile.Item 1B. Unresolved Staff Comments None.Item 2. Properties We occupy approximately 7,484 square feet of office and laboratory space in Cambridge, Massachusetts under a lease that expires in October2014. We believe that our facility is sufficient to meet our current needs and that suitable additional space will be available as and when needed.Item 3. Legal Proceedings None.Item 4. Mine Safety Disclosures Not applicable.73Table of ContentsPART II Item 5. Market for Registrant's Common Equity, Related Stockholder Matters and Issuers Purchases of Equity Securities MARKET INFORMATION Our common stock is publicly traded on the NASDAQ Global Market under the symbol "VSTM." Trading of our common stock commenced onJanuary 27, 2012, following the completion of our initial public offering. The following table sets forth, for the periods indicated, the high and low salesprices for our common stock as reported on the NASDAQ Global Market. HOLDERS As of February 28, 2014, there were 40 holders of record of our common stock and the closing price of our common stock on the NASDAQGlobal Market as of that date was $13.37. The number of holders of record does not include beneficial owners whose shares are held by nominees instreet name.DIVIDENDS We have never declared or paid cash dividends on our common stock, and we do not expect to pay any cash dividends on our common stock in theforeseeable future.SECURITIES AUTHORIZED FOR ISSUANCE UNDER EQUITY COMPENSATION PLANS The following table contains information about our equity compensation plans as of December 31, 2013. There are no equity compensation plansthat have not been approved by our security holders.74Year ended December 31, 2013 High Low First quarter $11.75 $8.78 Second quarter $14.09 $8.27 Third quarter $18.82 $12.38 Fourth quarter $13.47 $8.60 Year ended December 31, 2012 High Low First quarter (beginning January 27, 2012) $12.24 $10.05 Second quarter $11.58 $8.89 Third quarter $12.00 $7.56 Fourth quarter $9.67 $6.25 Plan category Number of securitiesto be issued uponexercise ofoutstanding stockoptions, warrants andrights Weighted-average exerciseprice ofoutstanding options,warrants andrights Number of securitiesremaining availablefor future issuanceunder equitycompensation plans Equity compensation plans approved by securityholders(1) 2,388,062 $8.66 1,372,376 (1)Includes information regarding our 2010 Equity Incentive Plan and 2012 Incentive Plan.Table of ContentsPERFORMANCE GRAPH The following performance graph and related information shall not be deemed to be "soliciting material" or to be "filed" with the SEC, nor shallsuch information be incorporated by reference into any future filing under the Securities Act, except to the extent that we specifically incorporate it byreference into such filing. The following graph compares the performance of our common stock to the NASDAQ Composite Index and to the NASDAQ BiotechnologyIndex from January 27, 2012 (the first date that shares of our common stock were publicly traded) through December 31, 2013. The comparisonassumes $100 was invested after the market closed on January 27, 2012 in our common stock and in each of the foregoing indices, and it assumesreinvestment of dividends, if any.COMPARISON OF 2 YEAR CUMULATIVE TOTAL RETURN*Among Verastem, Inc., the NASDAQ Composite Index, and the NASDAQ Biotechnology Index *$100 invested on 1/27/12 in stock or 12/31/11 in index, including reinvestment of dividends. Fiscal year ending December 31, 2013.Cumulative Total Return ComparisonPURCHASE OF EQUITY SECURITIES We did not purchase any of our equity securities during the period covered by this Annual Report on Form 10-K.USE OF PROCEEDS FROM REGISTERED SECURITIES In February 2012, we completed an initial public offering of 6,325,000 shares of our common stock at a price of $10.00 per share for an aggregateoffering price of $63.3 million. The offer and sale of all of the shares in the offering were registered under the Securities Act pursuant to a registration75 1/27/12 3/12 6/12 9/12 12/12 3/13 6/13 9/13 12/13 Verastem, Inc. 100.00 98.56 87.38 84.67 79.26 86.74 125.16 112.26 102.80 NASDAQComposite 100.00 118.32 112.69 120.24 116.75 127.31 133.33 149.00 165.43 NASDAQBiotechnology 100.00 115.66 120.05 134.02 133.42 161.32 172.32 207.74 226.99 Table of Contentsstatement on Form S-1 (File No. 333-177677), which was declared effective by the SEC on January 26, 2012, and a registration statement on Form S-1(File No. 333-179910) filed pursuant to Rule 462(b) of the Securities Act. As of February 28, 2014, we have used approximately $56.8 million of the net proceeds primarily to fund the preclinical and clinical developmentof our lead product candidates, to advance and expand the research and preclinical development of additional product candidates and companiondiagnostics and for working capital, capital expenditures and other general corporate purposes.Item 6. Selected Financial Data You should read the following selected financial data together with our consolidated financial statements and the related notes appearing elsewherein this Annual Report on Form 10-K and the "Management's discussion and analysis of financial condition and results of operations" section of thisAnnual Report on Form 10-K. The selected historical financial information in this section is not intended to replace our financial statements and therelated notes therein. Our historical results for any prior period are not necessarily indicative of results to be expected in any future period. Period fromAugust 4, 2010(inception) toDecember 31,2010 Years Ended December 31, Period fromAugust 4, 2010(inception) toDecember 31,2013 Statement of operations data: 2011 2012 2013 (in thousands, except per share amounts) Operating expenses: Research and development $400 $9,883 $21,712 $25,930 $57,925 General and administrative 384 3,815 10,518 15,472 30,189 Total operating expenses 784 13,698 32,230 41,402 88,020 Loss from operations (784) (13,698) (32,230) (41,402) (88,114)Interest income — 15 246 200 461 Net loss (784) (13,683) (31,984) (41,202) (87,653)Accretion of preferredstock (2) (32) (6) — (40) Net loss applicable tocommon stockholders $(786)$(13,715)$(31,990)$(41,202)$(87,693) Net loss per shareapplicable to commonstockholders—basicand diluted $(0.91)$(10.59)$(1.70)$(1.82)$(6.90) Weighted-average numberof common shares usedin net loss per shareapplicable to commonstockholders—basicand diluted 850 1,295 18,765 22,680 12,707 As of December 31, Balance sheet data: 2010 2011 2012 2013 (in thousands) Cash, cash equivalents and investments $3,584 $56,805 $91,520 $123,656 Working capital 3,228 44,795 54,683 94,151 Total assets 3,604 59,037 92,923 125,261 Redeemable convertible preferred stock 3,923 68,141 — — Deficit accumulated during the development stage (784) (14,467) (46,451) (87,653)76Total stockholders' (deficit) equity (687) (12,766) 90,466 117,446 Table of ContentsItem 7. Management's Discussion and Analysis of Financial Condition and Results of Operations You should read the following discussion and analysis of our financial condition and results of operations together with our financial statementsand related notes appearing elsewhere in this Annual Report on Form 10-K. Some of the information contained in this discussion and analysis or setforth elsewhere in this Annual Report on Form 10-K, includes forward-looking statements that involve risks and uncertainties. As a result of manyfactors, such as those set forth under "Risk Factors" and elsewhere in this Form 10-K, our actual results may differ materially from those described inor implied by these forward-looking statements. Please also refer to the section under heading "Special Note Regarding Forward-Looking Statement."OVERVIEW We are a clinical-stage biopharmaceutical company focused on discovering and developing proprietary small molecule drugs targeting cancer stemcells along with proprietary companion diagnostics. A cancer stem cell is a particularly aggressive type of tumor cell, resistant to conventional cancertherapy, that we believe is an underlying cause of tumor recurrence and metastasis. Our scientific co-founders have made discoveries that link theepithelial-to-mesenchymal transition, or EMT, to the emergence of cancer stem cells. This transition involves the transformation of one type of cancercell into a more aggressive and drug resistant type of cancer cell. Building on these discoveries, our scientific co-founders developed proprietarytechnology to create a stable population of cancer stem cells that we use to screen for and identify small molecule compounds that target cancer stemcells. We have initiated multiple clinical trials with our product candidates VS-6063, VS-4718 and VS-5584, including a potentially pivotal trial of VS-6063 in mesothelioma. We commenced active operations in the second half of 2010. Our operations to date have been limited to organizing and staffing our company,business planning, raising capital, acquiring and developing our technology, identifying potential product candidates, undertaking preclinical studies ofour most advanced product candidates and, recently, initiating clinical trials for our product candidates. To date, we have not generated any revenues andhave financed our operations with net proceeds from the private placement of our preferred stock, our initial public offering in February 2012 and ourfollow-on offering in July 2013. As of December 31, 2013, we had a deficit accumulated during the development stage of $87.7 million. Our net loss was $41.2 million,$32.0 million, $13.7 million and $87.7 million for the years ended December 31, 2013, 2012 and 2011 and for the period from August 4, 2010(inception) to December 31, 2013, respectively. We expect to incur significant expenses and increasing operating losses for the foreseeable future. Weexpect our expenses to increase in connection with our ongoing activities, particularly as we continue the research and development and clinical trials of,and seek marketing approval for, our product candidates. In addition, if we obtain marketing approval for any of our product candidates, we expect toincur significant commercialization expenses related to product sales, marketing, manufacturing and distribution. Accordingly, we will need to obtainsubstantial additional funding in connection with our continuing operations. Adequate additional financing may not be available to us on acceptableterms, or at all. If we are unable to raise capital when needed or on attractive terms, we would be forced to delay, reduce or eliminate our research anddevelopment programs or any future commercialization efforts. We will need to generate significant revenues to achieve profitability, and we may neverdo so.77Table of ContentsFINANCIAL OPERATIONS OVERVIEWRevenue To date, we have not generated any revenues. Our ability to generate product revenues, which we do not expect will occur for many years, if ever,will depend heavily on the successful development and eventual commercialization of our product candidates.Research and development expenses Research and development expenses consist of costs associated with our research activities, including our drug discovery efforts, and thedevelopment of our therapeutic product candidates and companion diagnostics. Our research and development expenses consist of:•employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; •external research and development expenses incurred under arrangements with third parties, such as contract research organizations, orCROs, clinical sites, manufacturing organizations and consultants, including our scientific advisory board; •license fees; and •facilities, depreciation and other allocated expenses, which include direct and allocated expenses for rent and maintenance of facilities,depreciation of leasehold improvements and equipment, and laboratory and other supplies. We expense research and development costs to operations as incurred. We account for nonrefundable advance payments for goods and servicesthat will be used in future research and development activities as expenses when the service has been performed or when the goods have been received,rather than when the payment is made. We use our employee and infrastructure resources across multiple research and development projects. We do not allocate employee-relatedexpenses or depreciation to any particular project and do not track research and development costs by project. The components of our research anddevelopment costs are described in more detail in "—Results of operations." We anticipate that our research and development expenses will increase significantly in future periods as we continue costlier developmentactivities, including clinical trials for our product candidates. The successful development of our product candidates is highly uncertain. At this time, we cannot reasonably estimate or know the nature, timingand estimated costs of the efforts that will be necessary to complete development of our product candidates or the period, if any, in which material netcash inflows from our product candidates may commence. This is due to the numerous risks and uncertainties associated with developing drugs,including the uncertainty of:•the scope, rate of progress and expense of our drug discovery efforts and other research and development activities; •the potential benefits of our product candidates over other therapies; •our ability to market, commercialize and achieve market acceptance for any of our product candidates that we receive regulatory approvalfor; •clinical trial results; •the terms and timing of regulatory approvals; and78Table of Contents•the expense of filing, prosecuting, defending and enforcing patent claims and other intellectual property rights. A change in the outcome of any of these variables with respect to the development of a product candidate could mean a significant change in thecosts and timing associated with the development of that product candidate. For example, if the FDA or other regulatory authority were to require us toconduct clinical trials beyond those which we currently anticipate will be required for the completion of clinical development of a product candidate or ifwe experience significant delays in enrollment in any clinical trials, we could be required to expend significant additional financial resources and time onthe completion of clinical development.General and administrative expenses General and administrative expenses consist primarily of salaries and related costs for personnel, including stock-based compensation expense, inour executive, finance and business development functions. Other general and administrative expenses include allocated facility costs and professionalfees for legal, patent, investor and public relations, consulting, insurance premiums, and accounting services.Interest income Interest income includes interest earned on our cash equivalent and investment accounts. Prior to September 30, 2011, our cash and cashequivalents were invested in non-interest-bearing accounts. As a result, we did not earn interest income until the last three months of 2011.Accretion of preferred stock Prior to the conversion of our preferred stock into 11,740,794 shares of common stock upon the closing of our initial public offering in February2012, our preferred stock was redeemable beginning in 2016 at its original issue price plus any declared but unpaid dividends upon a specified vote ofthe preferred stockholders. Accretion of preferred stock reflects the periodic accretion of issuance costs on our preferred stock to its redemption value.CRITICAL ACCOUNTING POLICIES AND SIGNIFICANT JUDGMENTS AND ESTIMATES Our management's discussion and analysis of our financial condition and results of operations is based on our financial statements, which we haveprepared in accordance with U.S. generally accepted accounting principles. The preparation of these financial statements requires us to make estimatesand judgments that affect the reported amounts of assets, liabilities and expenses and the disclosure of contingent assets and liabilities in our financialstatements. On an ongoing basis, we evaluate our estimates and judgments, including those related to accrued expenses and stock-based compensationdescribed in greater detail below. We base our estimates on our limited historical experience, known trends and events and various other factors that webelieve are reasonable under the circumstances, the results of which form the basis for making judgments about the carrying values of assets andliabilities that are not readily apparent from other sources. Actual results may differ from these estimates under different assumptions or conditions. Our significant accounting policies are described in more detail in the notes to our consolidated financial statements appearing elsewhere in thisAnnual Report on Form 10-K. However, we believe that the following accounting policies are the most critical to aid you in fully understanding andevaluating our financial condition and results of operations.79Table of ContentsAccrued research and development expenses As part of the process of preparing our financial statements, we are required to estimate our accrued expenses. This process involves reviewingquotations and contracts, identifying services that have been performed on our behalf and estimating the level of service performed and the associatedcost incurred when we have not yet been invoiced or otherwise notified of the actual cost. The majority of our service providers invoice us monthly inarrears for services performed or when contractual milestones are met. We make estimates of our accrued expenses as of each balance sheet date in ourfinancial statements based on facts and circumstances known to us at that time. We periodically confirm the accuracy of our estimates with the serviceproviders and make adjustments if necessary. The significant estimates in our accrued research and development expenses include fees paid to contractresearch organizations, or CROs, in connection with research and development activities for which we have not yet been invoiced. We base our expenses related to CROs on our estimates of the services received and efforts expended pursuant to quotes and contracts with CROsthat conduct research and development on our behalf. The financial terms of these agreements are subject to negotiation, vary from contract to contractand may result in uneven payment flows. There may be instances in which payments made to our vendors will exceed the level of services provided andresult in a prepayment of the research and development expense. In accruing service fees, we estimate the time period over which services will beperformed and the level of effort to be expended in each period. If the actual timing of the performance of services or the level of effort varies from ourestimate, we adjust the accrual or prepaid accordingly. Although we do not expect our estimates to be materially different from amounts actuallyincurred, our understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary andcould result in us reporting amounts that are too high or too low in any particular period.Stock-based compensation Prior to becoming a public company, we utilized significant estimates and assumptions in determining the fair value of our common stock. Wegranted stock options at exercise prices not less than the fair market value of our common stock as determined by the board of directors, with input frommanagement. The board of directors determined the estimated fair value of our common stock based on a number of objective and subjective factors,including external market conditions affecting the biotechnology industry sector and the prices at which we sold shares of redeemable convertiblepreferred stock, the superior rights and preferences of securities senior to our common stock at the time and the likelihood of achieving a liquidity event,such as an initial public offering or sale of our company. We utilized various valuation methodologies in accordance with the framework of the 2004 American Institute of Certified Public AccountantsTechnical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation, to estimate the fair value of our commonstock. The methodologies included an asset-based approach and the current value method for our initial common stock valuation as of November 30,2010, the option pricing method utilizing the reverse backsolve method to estimate our underlying equity value as of July 31, 2011 and a methodologythat determined an estimated value under an initial public offering scenario and a sale scenario based upon an assessment of the probability ofoccurrence of each scenario as of September 30, 2011, November 17, 2011, and December 31, 2011. Each valuation methodology included estimatesand assumptions that required our judgment. These estimates included assumptions regarding future performance, including the successful completionof preclinical studies and clinical trials and the time to completing an initial public offering or sale. Significant changes to the key assumptions used inthe valuations could have resulted in different fair values of common stock at each valuation date.80Table of Contents We recognize stock-based compensation expense for stock options issued to employees based on the grant date fair value of the awards on astraight-line basis over the requisite service period. We record stock-based compensation expense for stock options issued to non-employees based onthe estimated fair value of the services received or of the equity instruments issued, whichever is more reliably measured, based on the vesting date fairvalue of the awards on a straight-line basis over the vesting period. We estimate the fair value of stock option awards using the Black-Scholes option-pricing model. Determining the fair value of share-based awardsrequires the use of subjective assumptions, including the expected term of the award and expected stock price volatility. The assumptions used indetermining the fair value of share-based awards represent management's best estimates, which involve inherent uncertainties and the application ofmanagement judgment. As a result, if factors change, and we use different assumptions, our share-based compensation could be materially different inthe future. The risk-free interest rate used for each grant is based on a U.S. Treasury instrument whose term is consistent with the expected term of thestock option. Because we do not have a sufficient history to estimate the expected term, we use the simplified method as described in SAB Topic 14.D.2for estimating the expected term. The simplified method is based on the average of the vesting tranches and the contractual life of each grant. Becausethere was no public market for our common stock prior to our initial public offering, we lacked company-specific historical and implied volatilityinformation. Therefore, we used the historical volatility of a representative group of public biotechnology and life sciences companies with similarcharacteristics to us. In 2012, subsequent to our initial public offering, we began to consider including our own historical volatility, based on futureexpectations. We have not paid and do not anticipate paying cash dividends on our shares of common stock; therefore, the expected dividend yield isassumed to be zero. We also recognize compensation expense for only the portion of options that are expected to vest. Accordingly, we have estimatedexpected forfeitures of stock options based on our historical forfeiture rate, adjusted for known trends, and used these rates in developing a futureforfeiture rate. We have also granted performance-based restricted stock units (RSUs) with terms that allow the recipients to vest in a specific number of sharesbased upon the achievement of performance-based milestones as specified in the grants. Share-based compensation expense associated with theseperformance-based RSUs is recognized if the performance condition is considered probable of achievement using management's best estimates of thetime to vesting for the achievement of the performance-based milestones. If the actual achievement of the performance-based milestones varies from ourestimates, share-based compensation expense could be materially different than what is recorded in the period. The cumulative effect on current andprior periods of a change in the estimated time to vesting for performance-based RSUs will be recognized as compensation cost in the period of therevision, and recorded as a change in estimate. While the assumptions used to calculate and account for share-based compensation awards represents management's best estimates, these estimatesinvolve inherent uncertainties and the application of management's judgment. As a result, if revisions are made to our underlying assumptions andestimates, our share-based compensation expense could vary significantly from period to period. As of December 31, 2013, there was approximately $8.2 million, $4.3 million and $2.0 million of unrecognized share-based compensation, net ofestimated forfeitures, related to stock options, restricted stock units and restricted stock awards, respectively, which are expected to be recognized overweighted-average periods of 2.7 years, 2.1 and 0.75 years, respectively. The total unrecognized share-based compensation cost will be adjusted forfuture changes in estimated forfeitures. See Notes 2 and 8 to our consolidated financial statements located in this Annual Report on Form 10-K forfurther discussion of share-based compensation.81Table of ContentsRESULTS OF OPERATIONS The information reported within our consolidated financial statements from August 4, 2010 to December 31, 2011 was based solely on theaccounts of Verastem, Inc. In December 2012, Verastem Securities Company, our wholly owned subsidiary, was incorporated. All financialinformation presented after December 31, 2011 has been consolidated and includes the accounts of our wholly owned subsidiary. All significantintercompany balances and transactions have been eliminated in consolidation.Comparison of the Year Ended December 31, 2013 to the Year Ended December 31, 2012 Research and development expense. Research and development expense for the year ended December 31, 2013 (2013 Period) was $25.9 millioncompared to $21.7 million for the year ended December 31, 2012 (2012 Period). The $4.2 million increase from the 2012 Period to the 2013 Periodwas primarily related to an increase of $4.9 million in contract research organization expense for outsourced biology, chemistry and developmentservices, which includes our clinical trial costs, a $1.8 million increase in personnel costs primarily due to increased average headcount, an approximate$619,000 increase in stock-based compensation expense and an increase of approximately $368,000 in travel fees primarily due to increased travelassociated with our clinical trials. These increases were partially offset by a decrease of $3.5 million in license fee expense related to our agreement withPfizer, Inc. General and administrative expense. General and administrative expense for the 2013 Period was $15.5 million compared to $10.5 million forthe 2012 Period. The $5.0 million increase from the 2012 Period to the 2013 Period primarily resulted from an increase of $2.4 million in stock-basedcompensation expense associated with restricted stock units, an increase in professional fees and other costs of $1.5 million, an increase in consultingfees of approximately $514,000, an approximate $274,000 increase in corporate franchise taxes and an approximate $339,000 increase in personnelcosts primarily due to increase in salaries and headcount. Interest income. Interest income decreased to approximately $200,000 for the 2013 Period from approximately $246,000 for the 2012 Period.This decrease was primarily due to lower coupon rates on investments for the 2013 Period compared to the 2012 Period.Comparison of the Year Ended December 31, 2012 to the Year Ended December 31, 2011 Research and development expense. Research and development expense for the year ended December 31, 2012 was $21.7 million compared to$9.9 million for the year ended December 31, 2011. The $11.8 million increase is primarily related to increased contract research organization expenseof $4.1 million, an increase of $3.6 million in license fees due to our agreement with Pfizer, Inc., including expense associated with the issuance of192,012 shares of common stock, an increase of $3.3 million for personnel costs, including stock-based compensation of $2.0 million, primarily due toa higher fair value of our common stock, an increase of an approximate $404,000 for laboratory supplies, an increase of an approximate $118,000 fordepreciation due to additional laboratory equipment and an approximate $86,000 in additional occupancy costs due to a full year of costs associated withour new facility that we occupied in May 2011. General and administrative expense. General and administrative expense for the year ended December 31, 2012 was $10.5 million compared to$3.8 million for the year ended December 31, 2011. The $6.7 million increase resulted from an increase of $4.6 million for personnel costs, includingstock-based compensation of $3.8 million, primarily due to higher fair value of our common stock, an increase of $1.1 million in professional feesprimarily related to additional legal and accounting fees for being a publicly traded company, an increase of an approximate $389,000 in insurance costsprimarily related to being a publicly traded company, an increase of an approximate $272,000 in consulting fees and an increase of travel andentertainment costs of an approximate $95,000.82Table of Contents Interest income. Interest income increased to approximately $246,000 for the year ended December 31, 2012 from approximately $15,000 for theyear ended December 31, 2011. The increase in interest income was caused by an increase in the average cash balances available for investment and anincrease in interest rates earned on investments. The increase in the average cash balances for investment was primarily due to the net proceeds from thesale of Series C redeemable convertible preferred stock in November 2011 and net proceeds from our initial public offering in February 2012. For mostof the year ended December 31, 2011, our cash was deposited in non-interest bearing accounts. Accretion of preferred stock. During 2012, we recorded $6,000 of accretion reflecting the periodic accretion of issuance costs associated with ourseries A, series B and series C preferred stock through the date of our initial public offering when all outstanding shares of preferred stock convertedinto common stock compared to $32,000 for the year ended December 31, 2011.LIQUIDITY AND CAPITAL RESOURCESSources of liquidity To date, we have not generated any revenues. We have financed our operations to date through private placements of our preferred stock, our initialpublic offering in February 2012 and our follow-on offering in July 2013. As of December 31, 2013, we had received $68.1 million in net proceedsfrom the issuance of preferred stock and $116.6 million in net proceeds from our public offerings. As of December 31, 2013, we had $123.7 million incash, cash equivalents, short-term investments and long-term investments. We primarily invest our cash, cash equivalents and investments in a U.S.Treasury money market fund, government-sponsored enterprise securities and corporate bonds.Cash flows The following table sets forth the primary sources and uses of cash for each of the periods set forth below. Operating activities. The use of cash in all periods resulted from our net losses adjusted for non-cash charges and changes in the components ofworking capital. The increase in cash used for the year ended December 31, 2013 compared to the year ended December 31, 2012 is primarily due to anincrease in research and development expenses related to our ongoing clinical trials and development of our lead product candidates. The increase incash used in operating activities for the year ended December 31, 2012 compared to the year ended December 31, 2011 is due to an increase in researchand development expenses as we increased our research and development headcount and increased spending on external research and developmentcosts. We expect cash used in operating activities to continue to increase for the foreseeable future as we fund our increased research, development andclinical activities. Investing activities. The cash used in investing activities for the year ended December 31, 2013 includes $23.3 million in net purchases ofinvestments and approximately $56,000 of property and83 Years EndedDecember 31, 2011 2012 2013 Net cash provided by (used in) Net cash used in operating activities $(9,372)$(22,601)$(26,305)Net cash used in investing activities (36,722) (45,859) (23,393)Net cash provided by financing activities 63,464 57,602 58,491 Net increase (decrease) in cash and cash equivalents $17,370 $(10,858)$8,793 Table of Contentsequipment purchases. The cash used in investing activities for the year ended December 31, 2012 reflects the net purchases of investments of$45.5 million and the purchase of approximately $310,000 of property and equipment. The cash used in investing activities for the year endedDecember 31, 2011 included $35.9 million purchase of investments, approximately $785,000 of purchases of property and equipment and an $86,000increase in restricted cash related to a standby letter of credit issued as a security deposit for our facility lease. Financing activities. The cash provided by financing activities for the year ended December 31, 2013 is primarily related the $59.8 million of netproceeds from our follow-on offering offset by $1.3 million cash used to settle our restricted stock liability. The cash provided by financing activitiesfor the year ended December 31, 2012 reflects the $57.6 million of net proceeds from our initial public offering not including costs incurred in 2011related to the transaction. The cash provided by financing activities in the year ended December 31, 2011 is primarily the result of the sale and issuanceof 12,000,000 shares of our series A preferred stock for net proceeds of $12.0 million, the sale and issuance of 16,025,000 shares of our series Bpreferred stock for net proceeds of $31.9 million, the sale and issuance of 9,067,825 shares of our series C preferred stock for net proceeds of$20.2 million. In December 2013, the Company entered into an agreement under which it may offer and sell, from time to time, shares of our common stock in"at the market" offerings up to a total aggregate market value of $35 million. The Company may use this facility at its discretion and no shares have beensold under this facility through February 28, 2014.Funding requirements We have three product candidates currently in clinical trials. We expect to continue to incur significant expenses and increasing operating losses forthe foreseeable future. We anticipate that our expenses will increase substantially if and as we:•continue our research, preclinical and clinical development of our product candidates, including the registration-directed trial of VS-6063in mesothelioma; •initiate additional clinical trials for our product candidates; •seek marketing approvals for any of our product candidates that successfully complete clinical trials; •ultimately establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketingapproval; •maintain, expand and protect our intellectual property portfolio; •acquire or in-license other products and technologies; •hire additional clinical, development and scientific personnel; and •add operational, financial and management information systems and personnel, including personnel to support our product developmentand planned future commercialization efforts. We expect that our existing cash, cash equivalents and investments will enable us to fund our current operating plan and capital expenditurerequirements into the first half of 2016. We have based this estimate on assumptions that may prove to be wrong, and we could use our available capitalresources sooner than we currently expect. Because of the numerous risks and uncertainties associated with the development and commercialization ofour product candidates, and the extent to which we may enter into collaborations with third parties for development and commercialization of ourproduct candidates, we are unable to estimate the amounts of increased capital outlays and operating expenses84Table of Contentsassociated with completing the development of our current product candidates. Our future capital requirements will depend on many factors, including:•the rate of progress, results and cost of completing of registration-directed trial of VS-6063 in mesothelioma; •assuming favorable clinical results, the cost, timing and outcome of our efforts to seek approval in mesothelioma in the United States andelsewhere in the world, including to fund the preparation and filing of regulatory submissions with the FDA and other regulatoryagencies worldwide; •the scope, progress and, results of our other ongoing and potential future clinical trials; •the extent to which we acquire or in-license other products and technologies; •the costs, timing and outcome of regulatory review of our product candidates; •the costs of future commercialization activities, including product sales, marketing, manufacturing and distribution, for any of ourproduct candidates for which we receive marketing approval; •revenue, if any, received from commercial sales of our product candidates, should any of our product candidates receive marketingapproval; •the costs of preparing, filing and prosecuting patent applications, maintaining and enforcing our intellectual property rights and defendingintellectual property-related claims; and •our ability to establish collaborations on favorable terms, if at all. Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equityofferings, debt financings, collaborations, strategic alliances and licensing arrangements. We do not have any committed external source of funds. To theextent that we raise additional capital through the sale of equity or convertible debt securities, the ownership interest of our existing stockholders will bediluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our existing stockholders. Debtfinancing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurringadditional debt, making capital expenditures or declaring dividends. If we raise additional funds through collaborations, strategic alliances or licensingarrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or productcandidates or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings whenneeded, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to developand market product candidates that we would otherwise prefer to develop and market ourselves.CONTRACTUAL OBLIGATIONS AND COMMITMENTS The following table summarizes our contractual obligations at December 31, 2013.85(in thousands) Total Less than1 year 1 - 3 years 3 - 5 years More than5 years Operating lease obligations $307 $307 — — — License agreements(1) — — — — — (1)As discussed in Note 11 to the financial statements appearing elsewhere in this Annual Report on Form 10-K, we have executedseveral agreements to license intellectual property. The license agreements require us to pay upfront license fees and ongoingannual license maintenance fees,Table of Contents In July 2012, we entered into a License Agreement with Pfizer Inc., ("Pfizer"), under which Pfizer granted us worldwide, exclusive rightsto research, develop, manufacture and commercialize products containing certain of Pfizer's inhibitors of focal adhesion kinase (the "Products")for all therapeutic, diagnostic and prophylactic uses in humans. We have the right to grant sublicenses under the foregoing licensed rights,subject to certain restrictions. We are solely responsible, at our own expense, for the clinical development of the Products, which is to beconducted in accordance with an agreed-upon development plan. We are also responsible for all manufacturing and commercialization activitiesat our own expense. Pfizer was required to provide us with an initial quantity of clinical supply of one of the Products for an agreed upon price.We made a one-time cash payment to Pfizer in the amount of $1.5 million and issued 192,012 shares of our common stock. Pfizer is alsoeligible to receive up to $2 million in developmental milestones and up to an additional $125 million based on the successful attainment ofregulatory and commercial sales milestones. Pfizer is also eligible to receive high single to mid double digit royalties on future net sales ofProducts. Our royalty obligations with respect to each Product in each country begin on the date of first commercial sale of the Product in thatcountry, and end on the later of 10 years after the date of first commercial sale of the Product in that country or the date of expiration orabandonment of the last claim contained in any issued patent or patent application licensed by Pfizer to us that covers the Product in that country. Under our drug discovery platform license agreement, which we amended and restated in January 2012, we also have agreed to makemilestone payments to the Whitehead Institute upon achieving various development, regulatory and commercialization milestones. For eachlicensed product, we agreed to make milestone payments of up to an aggregate of $1,560,000 plus an additional amount for each subsequentapproval of additional indications for a maximum number of licensed products. For each identified product that is not a licensed product, weagreed to make milestone payments of up to an aggregate of $815,000 plus an additional amount for each subsequent approval of additionalindications for a maximum number of identified products. Each type of specified milestone payment is payable only for each of the maximumnumber of licensed products and the maximum number of identified products, as the case may be, to achieve the applicable milestone. Inaddition, a separate milestone payment is due upon the first commercial sale of each licensed product or identified product that is a diagnostic orprognostic test. A single additional milestone payment is due for the first issuance of licensed patent rights in the United States, the UnitedKingdom, France, Germany, Spain or Italy. In addition, we have agreed to pay the Whitehead Institute royalties as a percentage of net sales oflicensed products. The royalty rate is in the low single digits as a percentage of net sales for licensed products that are therapeutics, the midsingle digits for licensed products that are diagnostics or prognostics and less than one percent for identified products. Under our license agreement with Poniard that we entered into in November 2011 relating to VS-4718 and certain other compounds, wepaid an upfront license fee and agreed to pay Poniard milestone payments upon the achievement of specified development and regulatorymilestones. In February 2014, we purchased the assets which were the subject of our license agreement with Poniard from Encarta, Inc.(Encarta), who had previously purchased those assets in 2013. In consideration for the assets, we issued 97,500 shares of common stock, awarrant to purchase 142,857 shares of common stock with an exercise price equal to $17.16 per share and paid $25,000. All existing obligationsunder the license agreement, including an achieved development milestone and an obligation to issue a warrant, were settled as part of thistransaction. In connection with the asset purchase agreement, we also assumed the rights and obligations under the Scripps License Agreement.Pursuant to the Scripps86totaling a minimum of $160,000 per year beginning in 2014 up to a maximum amount of $440,000 per year beginning in 2017,as well as reimburse certain patent costs incurred by the licensors, as applicable. We have not included maintenance fees in thetable above since the minimum annual payments are perpetual and the agreements are cancelable by us at any time upon priorwritten notice to the licensor.Table of ContentsLicense Agreement, we are obligated to pay Scripps potential product development milestone payments of up to an aggregate of $3,000,000upon the achievement of specified development and regulatory milestones. In addition, we are obligated to pay Scripps low single-digit royaltiesas a percentage of net sales of licensed products, subject to adjustments in certain circumstances. Our obligation to pay royalties on net sales ison a country by country basis. Under our separate exclusive license agreement with the Whitehead Institute, or the cancer diagnostic license agreement, which weamended and restated in December 2011, we paid an upfront license fee and agreed to make milestone payments of up to an aggregate of$825,000 to the Whitehead Institute upon achieving specified regulatory and commercialization milestones. In addition, we have agreed to paythe Whitehead Institute royalties as a percentage of net sales of licensed products. The royalty rate is in the mid-single digits as a percentage ofnet sales.OFF-BALANCE SHEET ARRANGEMENTS We did not have during the periods presented, and we do not currently have, any off-balance sheet arrangements, as defined underSecurities and Exchange Commission rules.TAX LOSS CARRYFORWARDS As of December 31, 2013, we had federal and state net operating loss carryforwards of $65.7 million and $65.0 million, respectively,which are available to reduce future taxable income. We also had federal tax credits of $1.5 million and state tax credits of approximately$514,000, which may be used to offset future tax liabilities. The net operating loss and tax credit carryforwards will expire at various datesthrough 2033. Net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal Revenue Service andstate tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest ofsignificant stockholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, as wellas similar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or taxliabilities. The amount of the annual limitation is determined based on the value of our company immediately prior to the ownership change.Subsequent ownership changes may further affect the limitation in future years. At December 31, 2013, we recorded a 100% valuationallowance against our net operating loss and tax credit carryforwards of approximately $29.7 million, as we believe it is more likely than not thatthe tax benefits will not be fully realized. In the future, if we determine that a portion or all of the tax benefits associated with our taxcarryforwards will be realized, net income would increase in the period of determination.RECENTLY ADOPTED ACCOUNTING STANDARDS We have not recently adopted any new accounting standards.Item 7A. Quantitative and Qualitative Disclosures About Market Risk We are exposed to market risk related to changes in interest rates. We had cash, cash equivalents and investments of $123.7 million and$91.5 million as of December 31, 2013 and 2012, respectively, consisting of cash, money market funds, United States Treasuries, government-sponsored enterprise securities and corporate bonds. Our primary exposure to market risk is interest rate sensitivity, which is affected bychanges in the general level of U.S. interest rates, particularly because most of our investments are in short-term securities. Our available for salesecurities are subject to interest rate risk and will fall in value if market interest rates increase. Due to the short-term duration most of ourinvestment portfolio and the low risk profile of our investments, an immediate 100 basis point change in interest rates would not have a materialeffect on the fair market value of our portfolio.87Table of Contents We contract with CROs and contract manufacturers globally which may be denominated in foreign currencies. We may be subject tofluctuations in foreign currency rates in connection with these agreements. Transactions denominated in currencies other than the functionalcurrency are recorded based on exchange rates at the time such transactions arise. As of December 31, 2013 and 2012, approximately $790,000and $42,000 of our total liabilities was denominated in currencies other than the functional currency, respectively.Item 8. Financial Statements and Supplementary Data Our consolidated financial statements, together with the report of our independent registered public accounting firm, appear on pages F-1through F-28 of this Annual Report on Form 10-K.Item 9. Changes in and Disagreements with Accountants on Accounting and Financial Disclosure None.Item 9A. Controls and Procedures Evaluation of Disclosure Controls and Procedures Our Chief Executive Officer and our Chief Financial Officer evaluated the effectiveness of our disclosure controls and procedures, asdefined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934, as amended, or the Exchange Act, as of the end of theperiod covered by this report. Based on that evaluation, our Chief Executive Officer and our Chief Financial Officer concluded that ourdisclosure controls and procedures as of the end of the period covered by this report were effective.Management's Report on Internal Control Over Financial Reporting Our management is responsible for establishing and maintaining adequate internal control over our financial reporting. Internal control overfinancial reporting is defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act as the process designed by, or under the supervision of,our Chief Executive Officer and our Chief Financial Officer, and effected by our board of directors, management and other personnel, to providereasonable assurance regarding the reliability of our financial reporting and the preparation of our financial statements for external purposes inaccordance with generally accepted accounting principles, and includes those policies and procedures that:(1)pertain to the maintenance of records that, in reasonable detail, accurately and fairly reflect the transactions and dispositions of assets; (2)provide reasonable assurance that transactions are recorded as necessary to permit preparation of financial statements in accordance withgenerally accepted accounting principles, and that receipts and expenditures are being made only in accordance with the authorizations ofmanagement and directors; and (3)provide reasonable assurance regarding the prevention or timely detection of unauthorized acquisition, use or disposition of assets thatcould have a material effect on our financial statements. Under the supervision and with the participation of our management, including our Chief Executive Officer and our Chief Financial Officer, weconducted an evaluation of the effectiveness of our internal control over financial reporting based on the framework provided in Internal Control—Integrated Framework issued by the Committee of Sponsoring Organizations of the Treadway Commission (1992 Framework). Based on thisevaluation, our management concluded that our internal control over financial reporting was effective as of December 31, 2013.88Table of ContentsChanges in Internal Control Over Financial Reporting There has been no change in our internal control over financial reporting occurred during the fiscal quarter ended December 31, 2013 that hasmaterially affected, or is reasonably likely to materially affect, the Company's internal control over financial reporting.Item 9B. Other Information None89Table of ContentsPART III Item 10. Directors, Executive Officers, and Corporate Governance Other than the information regarding our executive officers provided in Part I of this report under the heading "Business—Executive Officers ofthe Registrant," the information required to be furnished pursuant to this item is incorporated herein by reference to our definitive proxy statement forthe 2014 Annual Meeting of the Stockholders.Item 11. Executive Compensation The information required by this Item 11 is incorporated herein by reference from our definitive proxy statement for the 2014 Annual Meeting ofStockholders.Item 12. Security Ownership of Certain Beneficial Owners and Management and Related Stockholder Matters The information required by this Item 12 is incorporated herein by reference from our definitive proxy statement for the 2014 Annual Meeting ofStockholders.Item 13. Certain Relationships and Related Transactions, and Director Independence The information required by this Item 13 is incorporated herein by reference from our definitive proxy statement for the 2014 Annual Meeting ofStockholders.Item 14. Principal Accountant Fees and Services The information required by this Item 14 is incorporated herein by reference from our definitive proxy statement for the 2014 Annual Meeting ofStockholders.90Table of ContentsPART IV Item 15. Exhibits and Financial Statement Schedules Financial Statements See Part II, Item 8 for the Financial Statements required to be included in this Annual Report on Form 10-K.Financial Statement Schedules All financial statement schedules are omitted because they are not applicable or the required information is included in the consolidated financialstatements or notes thereto.Exhibits Those exhibits required to be filed by Item 601 of Regulation S-K are listed in the Exhibit Index immediately preceding the exhibits hereto andsuch listing is incorporated herein by reference.91Table of ContentsSIGNATURES Pursuant to the requirements of Section 13 or 15(d) of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signedon its behalf by the undersigned, thereunto duly authorized on this 6th day of March 2014. Pursuant to the requirements of the Securities Exchange Act of 1934, this report has been signed by the following persons on behalf of theregistrant in the capacities and on the dates indicated.92 VERASTEM, INC. By: /s/ ROBERT FORRESTERRobert ForresterChief Executive OfficerSignature Title Date /s/ ROBERT FORRESTERRobert Forrester Chief Executive Officer and Director(Principal executive officer) March 6, 2014/s/ JOHN B. GREEN, CPAJohn B. Green, CPA Chief Financial Officer (Principalfinancial officer and Principal accountingofficer) March 6, 2014/s/ RICHARD ALDRICHRichard Aldrich Director March 6, 2014/s/ JOHN K. CLARKEJohn K. Clarke Director March 6, 2014/s/ MICHAEL KAUFFMAN, M.D., PH.D.Michael Kauffman, M.D., Ph.D. Director March 6, 2014/s/ ALISON LAWTONAlison Lawton Director March 6, 2014/s/ S. LOUISE PHANSTIELS. Louise Phanstiel Director March 6, 2014Table of Contents93Signature Title Date /s/ STEPHEN SHERWIN, M.D.Stephen Sherwin, M.D. Director March 6, 2014/s/ HENRI TERMEERHenri Termeer Director March 6, 2014/s/ CHRISTOPH WESTPHAL, M.D., PH.D.Christoph Westphal, M.D., Ph.D. Director March 6, 2014Table of ContentsVerastem, Inc.(A development stage company) CONSOLIDATED FINANCIAL STATEMENTS The years ended December 31, 2011, 2012 and 2013, and the period from August 4, 2010 (inception) to December 31, 2013 CONTENTS F-1Report of Independent Registered Public Accounting Firm F-2Consolidated Financial Statements Consolidated Balance Sheets F-3Consolidated Statements of Operations and Comprehensive Loss F-4Consolidated Statements of Redeemable Convertible Preferred Stock and Stockholders' (Deficit) Equity F-5Consolidated Statements of Cash Flows F-6Notes to Consolidated Financial Statements F-7Table of ContentsVerastem, Inc.(A development stage company) REPORT OF INDEPENDENT REGISTERED PUBLIC ACCOUNTING FIRM The Board of Directors and Stockholders ofVerastem, Inc. We have audited the accompanying consolidated balance sheets of Verastem, Inc. (a development stage company) (the Company) as ofDecember 31, 2012 and 2013, and the related consolidated statements of operations and comprehensive loss, redeemable convertible preferred stock andstockholders' (deficit) equity and cash flows for each of the three years in the period ended December 31, 2013 and for the period from August 4, 2010(inception) to December 31, 2013. These financial statements are the responsibility of the Company's management. Our responsibility is to express anopinion on these financial statements based on our audits. We conducted our audits in accordance with the standards of the Public Company Accounting Oversight Board (United States). Those standardsrequire that we plan and perform the audits to obtain reasonable assurance about whether the financial statements are free of material misstatement. Wewere not engaged to perform an audit of the Company's internal control over financial reporting. Our audits included consideration of internal controlover financial reporting as a basis for designing audit procedures that are appropriate in the circumstances, but not for the purpose of expressing anopinion on the effectiveness of the Company's internal control over financial reporting. Accordingly, we express no such opinion. An audit alsoincludes examining, on a test basis, evidence supporting the amounts and disclosures in the financial statements, assessing the accounting principlesused and significant estimates made by management, and evaluating the overall financial statement presentation. We believe that our audits provide areasonable basis for our opinion. In our opinion, the financial statements referred to above present fairly, in all material respects, the consolidated financial position of Verastem, Inc.as of December 31, 2012 and 2013 and the consolidated results of its operations and its cash flows for each of the three years in the period endedDecember 31, 2013 and the period from August 4, 2010 (inception) to December 31, 2013, in conformity with U.S. generally accepted accountingprinciples./s/ Ernst & Young LLPBoston, MassachusettsMarch 6, 2014F-2Table of ContentsVerastem, Inc.(A development stage company) CONSOLIDATED BALANCE SHEETS (in thousands, except per share amounts) See accompanying notes.F-3 December 31, 2012 2013 Assets Current assets: Cash and cash equivalents $10,096 $18,889 Short-term investments 46,480 82,423 Restricted cash — 86 Prepaid expenses and other current assets 506 557 Total current assets 57,082 101,955 Property and equipment, net 811 631 Long-term investments 34,944 22,344 Other assets — 331 Restricted cash 86 — Total assets $92,923 $125,261 Liabilities and stockholders' equity Current liabilities: Accounts payable $1,848 $2,760 Accrued expenses 551 4,327 Liability classified stock-based compensation awards — 717 Total current liabilities 2,399 7,804 Deferred rent 38 — Liability for shares subject to repurchase 20 11 Stockholders' equity: Convertible preferred stock, $0.0001 par value; 5,000 shares authorized, no shares issuedand outstanding — — Common stock, $0.0001 par value; 100,000 shares authorized, 20,364 and 25,328 sharesissued and outstanding at December 31, 2012 and 2013, respectively 2 3 Additional paid-in capital 136,893 205,068 Accumulated other comprehensive income 22 28 Deficit accumulated during the development stage (46,451) (87,653) Total stockholders' equity 90,466 117,446 Total liabilities and stockholders' equity $92,923 $125,261 Table of ContentsVerastem, Inc.(A development stage company) CONSOLIDATED STATEMENTS OF OPERATIONS AND COMPREHENSIVE LOSS (in thousands, except per share amounts) See accompanying notes.F-4 Period fromAugust 4, 2010(inception) toDecember 31,2013 Years Ended December 31, 2011 2012 2013 Operating expenses: Research and development $9,883 $21,712 $25,930 $57,925 General and administrative 3,815 10,518 15,472 30,189 Total operating expenses 13,698 32,230 41,402 88,114 Loss from operations (13,698) (32,230) (41,402) (88,114)Interest income 15 246 200 461 Net loss (13,683) (31,984) (41,202) (87,653)Accretion of preferred stock (32) (6) — (40) Net loss applicable to common stockholders $(13,715)$(31,990)$(41,202)$(87,693) Net loss per share applicable to common stockholders $(10.59)$(1.70)$(1.82)$(6.90) Weighted-average number of common shares used in net lossper share applicable to common stockholders—basic anddiluted 1,295 18,765 22,680 12,707 Net loss $(13,683)$(31,984)$(41,202)$(87,653)Unrealized (losses) gains on available-for-sale securities (2) 24 6 28 Comprehensive loss $(13,685)$(31,960)$(41,196)$(87,625) Table of ContentsVerastem, Inc.(A development stage company) CONSOLIDATED STATEMENTS OF REDEEMABLE CONVERTIBLE PREFERRED STOCK ANDSTOCKHOLDERS' (DEFICIT) EQUITY (in thousands, except share data) Redeemableconvertiblepreferred stock Accumulatedothercomprehensive(loss)income Deficitaccumulatedduring thedevelopmentstage Common stock Totalsstockholder's(deficit)equity Additionalpaid-incapital Shares Amount Shares Amount Balance at August 4,2010 (inception) — $— — $— $— $— $— $— Sale of common stockto founders — — 714,286 1 — — — 1 Vesting of restrictedstock — — 133,926 — — — — — Issuance of commonstock in exchangefor license — — 166,664 — 46 — — 46 Issuance of Series Aredeemableconvertible preferredstock, net of offeringcosts of $79 4,000,000 3,921 — — — — — — Accretion ofredeemableconvertible preferredstock to redemptionvalue — 2 — — (2) — — (2)Stock-basedcompensationexpense — — — — 52 — — 52 Net loss — — — — — — (784) (784) Balance atDecember 31, 2010 4,000,000 $3,923 1,014,876 $1 $96 $— $(784)$(687)Net loss — — — — — — (13,683) (13,683)Unrealized loss oninvestments — — — — — (2) (2)Issuance of Series Aredeemableconvertible preferredstock 12,000,000 12,000 — — — — — — Issuance of Series Bredeemableconvertible preferredstock, net of offeringcosts of $113 16,025,000 31,937 — — — — — — Issuance of Series Credeemableconvertible preferredstock, net of offeringcosts of $153 9,067,825 20,249 — — — — — — Accretion ofredeemableconvertible preferredstock to redemptionvalue — 32 — — (32) — — (32)Vesting of restrictedstock — — 543,712 — 3 — — 3 Stock-basedcompensationexpense — — — — 1,635 — — 1,635 Balance atDecember 31, 2011 41,092,825 $68,141 1,558,588 $1 $1,702 $(2)$(14,467)$(12,766)Net loss — — — — — — (31,984) (31,984)Unrealized gain on See accompanying notes.F-5investments — — — — — 24 — 24 Accretion ofredeemableconvertible preferredstock to redemptionvalue — 6 — — (6) — — (6)Conversion ofredeemableconvertible preferredstock into commonstock (41,092,825) (68,147) 11,740,794 1 68,146 — — 68,147 Issuance of commonstock, net ofissuance costs of$1,984 — — 6,325,000 — 56,838 — — 56,838 Issuance of commonstock in exchangefor license — — 192,012 — 1,957 — — 1,957 Vesting of restrictedstock — — 542,421 — 16 — — 16 Issuance of commonstock resulting fromexercise ofstock options — — 5,395 — 3 — — 3 Obligation to issue awarrant — — — — 837 — — 837 Stock-basedcompensationexpense — — — — 7,400 — — 7,400 Balance atDecember 31, 2012 — $— 20,364,210 $2 $136,893 $22 $(46,451)$90,466 Net loss — — — — — — (41,202) (41,202)Unrealized gain oninvestments — — — — — 6 — 6 Issuance of commonstock, net ofissuance costs of$235 — — 4,255,000 1 59,759 — — 59,760 Vesting of restrictedstock — — 417,718 — 9 — — 9 Issuance of commonstock resulting fromexercise ofstock options — — 81,795 — 33 — — 33 Issuance of commonstock resulting fromvesting of restrictedstock units andpayment of taxwithholdings — — 209,727 — (1,302) — — (1,302)Stock-basedcompensationexpense — — — — 9,676 — — 9,676 Balance atDecember 31, 2013 — $— 25,328,450 $3 $205,068 $28 $(87,653)$117,446 Table of ContentsVerastem, Inc.(A development stage company) CONSOLIDATED STATEMENTS OF CASH FLOWS (in thousands) Period fromAugust 4, 2010(inception) toDecember 31,2013 Years Ended December 31, 2011 2012 2013 Net loss $(13,683)$(31,984)$(41,202)$(87,653)Adjustments to reconcile net loss to net cash used in operatingactivities: Depreciation and amortization 83 207 236 526 Stock-based compensation expense 1,635 7,400 9,676 18,763 Common stock issued in exchange for license — 1,957 — 2,003 Obligation to issue a warrant in exchange for license 439 — — 439 Change in fair value of obligation to issue a warrant (33) 431 — 398 Changing in operating assets and liabilities: Prepaid expenses and other current assets (118) (376) (51) (557)Other assets — — (331) (331)Accounts payable 1,994 (425) 912 2,760 Accrued expenses and deferred rent 311 189 3,738 4,327 Liability classified stock-based compensation awards — — 717 717 Net cash used in operating activities (9,372) (22,601) (26,305) (58,608)Investing activities Purchases of property and equipment (785) (310) (56) (1,159)Purchases of investments (35,851) (154,028) (118,190) (308,069)Maturities of investments — 108,479 94,853 203,332 Increase in restricted cash (86) — — (86) Net cash used in investing activities (36,722) (45,859) (23,393) (105,982)Financing activities Proceeds from the issuance of redeemable convertible preferredstock 64,186 — — 68,107 Proceeds from the exercise of stock options — 3 33 36 Net (costs) proceeds from the issuance of common stock andrestricted common stock (722) 57,599 59,760 116,638 Cash used to settle tax withholdings for restricted stock units — — (1,302) (1,302) Net cash provided by financing activities 63,464 57,602 58,491 183,479 Increase (decrease) in cash and cash equivalents 17,370 (10,858) 8,793 18,889 Cash and cash equivalents at beginning of period 3,584 20,954 10,096 — Cash and cash equivalents at end of period $20,954 $10,096 $18,889 $18,889 Supplemental disclosure of non-cash financial activity Accretion of redeemable convertible preferred stock toredemption value $32 $6 $— $40 Conversion of redeemable convertible preferred stock uponinitial public offering $— $68,147 $— $68,147  See accompanying notes.F-6 Reclassification of obligation to issue warrant from liabilities toequity $— $837 $— $837 Table of ContentsVerastem, Inc.(A development stage company) NOTES TO CONSOLIDATED FINANCIAL STATEMENTS 1. Nature of business Verastem, Inc. (the "Company"), incorporated on August 4, 2010 as a Delaware corporation, is a biopharmaceutical company focused ondiscovering and developing proprietary small molecule drugs targeting cancer stem cells along with proprietary companion diagnostics. The Company'soperations to date have been limited to organizing and staffing the Company, business planning, raising capital, acquiring and developing its technology,identifying potential product candidates and undertaking preclinical and clinical studies of its most advanced product candidates. The Company has notgenerated revenue from its planned principal operations. Accordingly, the Company is considered to be in the development stage as defined in FinancialAccounting Standards Board Accounting Standards Codification Topic 915, Development Stage Entities. The Company is subject to a number of risks similar to other life science companies in the development stage, including, but not limited to, theneed to obtain adequate additional funding, possible failure of preclinical testing or clinical trials, inability to obtain marketing approval of productcandidates, competitors developing new technological innovations, market acceptance of the Company's products and protection of proprietarytechnology. If the Company does not successfully commercialize any of its product candidates, it will be unable to generate product revenue or achieveprofitability. As of December 31, 2013, the Company had a deficit accumulated during the development stage of $87.7 million. The Company expects tocontinue to incur operating losses in future periods. The Company had cash, cash equivalents and investments of $123.7 million as of December 31,2013. The Company believes that its existing cash, cash equivalents and investments will be sufficient to fund its current operating plan and capitalexpenditure requirements for at least the next twelve months.2. Significant accounting policiesBasis of consolidation Effective December 2012, Verastem Securities Company was incorporated as a wholly owned subsidiary of the Company and all financialinformation presented has been consolidated and includes the accounts of the Company and its wholly owned subsidiary. All intercompany balancesand transactions have been eliminated in consolidation.Use of estimates The preparation of the Company's financial statements in conformity with GAAP requires management to make estimates and assumptions thataffect the amounts reported in the financial statements and accompanying notes. On an ongoing basis, management evaluates its estimates, includingestimates related to accruals and stock-based compensation expense. The Company bases its estimates on historical experience and other market-specificor other relevant assumptions that it believes to be reasonable. Actual results could differ from such estimates. Prior to becoming a public company in February 2012, the Company utilized significant estimates and assumptions in determining the fair value ofits common stock. The Company granted stock options at exercise prices not less than the fair market value of its common stock as determined by theboard of directors, with input from management. The board of directors determined the estimated fair value of the Company's common stock based on anumber of objective and subjective factors, including external market conditions affecting the biotechnology industry sector and the prices at which theCompany sold shares of redeemable convertible preferred stock, the superior rights and preferences ofF-7Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued)securities senior to the Company's common stock at the time and the likelihood of achieving a liquidity event, such as an IPO or sale of the Company. The Company utilized various valuation methodologies in accordance with the framework of the American Institute of Certified PublicAccountants Technical Practice Aid, Valuation of Privately-Held Company Equity Securities Issued as Compensation (Practice Aid), to estimate the fairvalue of its common stock. The methodologies included an asset-based approach and the current value method for the Company's initial common stockvaluation as of November 30, 2010, the option pricing method utilizing the reverse backsolve method to estimate the Company's underlying equity valueas of July 31, 2011 and a methodology that determined an estimated value under an IPO scenario and a sale scenario based upon an assessment of theprobability of occurrence of each scenario as of September 30, 2011, November 17, 2011, and December 31, 2011. Each valuation methodologyincluded estimates and assumptions that required the Company's judgment. These estimates included assumptions regarding future performance,including the successful completion of preclinical studies and clinical trials and the time to completing an IPO or sale. Significant changes to the keyassumptions used in the valuations could result in different fair values of common stock at each valuation date.Segment and geographic information Operating segments are defined as components of an enterprise about which separate discrete information is available for evaluation by the chiefoperating decision maker, or decision-making group, in deciding how to allocate resources and in assessing performance. The Company views itsoperations and manages its business in one operating segment, which is the business of developing drugs that target cancer stem cells. The Companyoperates in only one geographic area.Cash and cash equivalents The Company considers all highly liquid investments with an original or remaining maturity of three months or less at the date of purchase to becash equivalents. Cash equivalents consist of money market funds, government-sponsored enterprise securities and corporate bonds. Cash equivalentsare reported at fair value.Fair value of financial instruments The Company is required to disclose information on all assets and liabilities reported at fair value that enables an assessment of the inputs used indetermining the reported fair values. The fair value hierarchy prioritizes valuation inputs based on the observable nature of those inputs. The fair valuehierarchy applies only to the valuation inputs used in determining the reported fair value of theF-8Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued)investments and is not a measure of the investment credit quality. The hierarchy defines three levels of valuation inputs: The following table presents information about the Company's financial assets and liabilities that have been measured at fair value at December 31,2013 and indicates the fair value hierarchy of the valuation inputs utilized to determine fair value (in thousands). The following table presents information about the Company's financial assets that have been measured at fair value at December 31, 2012 andindicates the fair value hierarchy of the valuation inputs utilized to determine such fair value (in thousands). In connection with the license agreement with Poniard Pharmaceuticals Inc. (Poniard), the Company is obligated to issue a warrant to Poniard forthe purchase of the Company's common stockF-9Level 1 inputs Quoted prices in active markets for identical assets or liabilitiesLevel 2 inputs Inputs other than quoted prices included within Level 1 that are observable for the asset or liability, either directly or indirectlyLevel 3 inputs Unobservable inputs that reflect the Company's own assumptions about the assumptions market participants would use inpricing the asset or liabilityDescription Total Quoted pricesin activemarkets(Level 1) Significant otherobservableinputs(Level 2) Significantunobservableinputs(Level 3) Financial assets Cash equivalents $17,000 $17,000 $— $— Short-term investments 82,423 — 82,423 — Long-term investments 22,344 — 22,344 — Total financial assets $121,767 $17,000 $104,767 $— Financial liabilities Liability classified stock-based compensation awards $717 $717 $— $— Total financial liabilities $717 $717 $— $— Description Total Quoted pricesin activemarkets(Level 1) Significant otherobservableinputs(Level 2) Significantunobservableinputs(Level 3) Financial assets Cash equivalents $8,171 $8,171 $— $— Short-term investments 46,480 — 46,480 — Long-term investments 34,944 — 34,944 — Total financial assets $89,595 $8,171 $81,424 $— Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued)upon the first patient dosing using a product licensed under the agreement with Poniard; such warrant will have a three year term from the date ofissuance. Prior to the Company's initial public offering, the exercise price of the warrant would have been equal to the fair value of the common stock onthe date of the most recent preferred stock financing prior to the issuance of the warrant. Upon the completion of the Company's initial public offering inJanuary 2012, the exercise price of the warrant will be equal to the average closing price of the Company's common stock during the five trading dayspreceding the issuance of the warrant. Prior to January 2012, the obligation to issue the warrant was a level 3 liability because its value measurement was based, in part, on significantinputs not observed in the market and reflects the Company's assumptions as to the expected warrant exercise price and the expected volatility of theCompany's common stock. The obligation to issue the warrant was initially recorded at fair value and, prior to the Company's initial public offering, wasrevalued at the end of each reporting period, with the change in the fair value reported in research and development expense within the statement ofoperations. Upon the completion of the Company's initial public offering, the obligation to issue the warrant met the definition of an equity-classifiedderivative instrument since the remaining variable inputs were consistent with those in a fixed for fixed forward option agreement, and was thereforerevalued as of January 26, 2012 with the change in fair value reported in research and development expense within the statement of operations. The fairvalue of the obligation to issue the warrant was then reclassified from liabilities to additional paid-in-capital on the Company's consolidated balancesheet. The Company will reassess the equity classification of the obligation to issue the warrant upon a change in facts and circumstances in futurereporting periods. As of December 31, 2011, the most recent issuance of the Company's Preferred Stock had been the issuance of the Series C Preferred Stock inNovember 2011. The Company estimated the value of the obligation to issue the warrant using a probability-weighted scenario analysis thatincorporated the probability of the completion of an initial public offering. The analysis included estimating the stock price on each measurement dateassuming that achievement of the milestone would be 100% probable. The estimated stock price contingent upon milestone achievement was determinedby analyzing the post-announcement returns for public companies that progressed to Phase 1 clinical trials. The following inputs were used to determinethe fair value of the obligation to issue the warrant:F-10 December 31, 2011 January 26, 2012 Non-IPO IPO Exercise price $11.09 $6.86 $10.00 Estimated stock price contingent upon milestoneachievement $12.60 $3.22 $8.54 Expected term 4.0 years 4.1 years 4.1 years Volatility 75% 70% 70%Dividend yield 0.00% 0.00% 0.00%Risk-free rate 0.54% 0.60% 0.60%Probability of achieving milestone 80% 80% 80%Probability of scenario 100% 20% 80%Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued) As of December 31, 2011, the fair value of the obligation to issue the warrant was recorded at $406,000. As a result of the change in inputs to thevaluation model, the fair value of the obligation to issue the warrant increased by $431,000 to $837,000 at January 26, 2012. Reasonable changes in theassumptions used to calculate the fair value of the obligation to issue the warrant would not result in significant changes in the fair value. The obligationto issue the warrant was settled and a warrant was issued in February 2014.Investments Investments and cash equivalents consist of investments in money market accounts, government-sponsored enterprise securities and commercialpaper of publicly traded companies secured by the U.S. government that are classified as available-for-sale pursuant to Accounting StandardsCodification (ASC) 320, Investments—Debt and Equity Securities. The Company classifies investments available to fund current operations as currentassets on its balance sheets. Investments are classified as long-term assets on the balance sheets if (i) the Company has the intent and ability to hold theinvestments for a period of at least one year and (ii) the contractual maturity date of the investments is greater than one year. Investments are carried atfair value with unrealized gains and losses included as a component of accumulated other comprehensive loss, which is a separate component ofstockholders' equity (deficit), until such gains and losses are realized. The fair value of these securities is based on quoted prices for identical or similarassets. If a decline in the fair value is considered other-than-temporary, based on available evidence, the unrealized loss is transferred from othercomprehensive loss to the statement of operations. There were no other-than-temporary declines in fair value of short-term or long-term investments forthe years ended December 31, 2011, 2012 and 2013 and the period from August 4, 2010 (inception) to December 31, 2013. Realized gains and lossesare determined using the specific identification method and are included in interest income in the statement of operations. There were no realized gains orlosses recognized for the years ended December 31, 2011, 2012 and 2013 and the period from August 4, 2010 (inception) to December 31, 2013. The Company reviews investments for other-than-temporary impairment whenever the fair value of an investment is less than the amortized costand evidence indicates that an investment's carrying amount is not recoverable within a reasonable period of time. To determine whether an impairmentis other-than-temporary, the Company considers the intent to sell, or whether it is more likely than not that the Company will be required to sell, theinvestment before recovery of the investment's amortized cost basis. Evidence considered in this assessment includes reasons for the impairment,compliance with the Company's investment policy, the severity and the duration of the impairment and changes in value subsequent to year end. As ofDecember 31, 2012 and 2013, there were no investments with a fair value that was significantly lower than the amortized cost basis or any investmentsthat had been in an unrealized loss position for a significant period.F-11Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued) As of December 31, 2013, cash, cash equivalents and investments included (in thousands): As of December 31, 2012, cash, cash equivalents and investments included (in thousands):Concentrations of credit risk and off-balance sheet risk Cash and cash equivalents and investments are financial instruments that potentially subject the Company to concentrations of credit risk. As ofDecember 31, 2013, the Company's cash, cash equivalents and investments were deposited at two financial institutions. The Company maintains itscash and cash equivalents and investments with high quality, accredited financial institutions and, accordingly, the Company believes it is not exposed toany significant credit risk on these funds. The Company has no significant off-balance sheet concentrations of credit risk, such as foreign currencyexchange contracts, option contracts or other hedging arrangements.F-12 AmortizedCost GrossUnrealizedGains GrossUnrealizedLosses FairValue Cash and cash equivalents: Cash and money market accounts $18,889 $— $— $18,889 Total cash and cash equivalents $18,889 $— $— $18,889 Investments: Government-sponsored enterprise securities (due within 1 year) $30,652 $12 $— $30,664 Government-sponsored enterprise securities (due within 1 - 2 years) $4,001 $2 $— $4,003 Corporate bonds (due within 1 year) 51,735 30 (6) 51,759 Corporate bonds (due within 1 - 2 years) 18,351 2 (12) 18,341 Total investments $104,739 $46 $(18)$104,767 Total cash, cash equivalents, and investments $123,628 $46 $(18)$123,656 AmortizedCost GrossUnrealizedGains GrossUnrealizedLosses FairValue Cash and cash equivalents: Cash and money market accounts $10,096 $— $— $10,096 Total cash and cash equivalents $10,096 $— $— $10,096 Investments: Government-sponsored enterprise securities (due within 1 year) $46,470 $13 $(3)$46,480 Government-sponsored enterprise securities (due within 1 - 2 years) 34,932 13 (1) 34,944 Total investments $81,402 $26 $(4)$81,424 Total cash, cash equivalents, and investments $91,498 $26 $(4)$91,520 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued)Property and equipment Property and equipment consists of laboratory equipment, office furniture, computer equipment and leasehold improvements. Expenditures forrepairs and maintenance are recorded to expense as incurred, whereas major betterments are capitalized as additions to property and equipment.Depreciation is calculated using the straight-line method over the following estimated useful lives of the assets: Upon retirement or sale, the cost of the disposed asset and the related accumulated depreciation are removed from the accounts and any resultinggain or loss is recognized. The Company reviews its long-lived assets for impairment whenever events or changes in business circumstances indicate that the carrying valueof assets may not be recoverable. Recoverability is measured by comparison of the asset's book value to future net undiscounted cash flows that theassets are expected to generate. If such assets are considered to be impaired, the impairment to be recognized is measured by the amount by which thebook value of the assets exceed their fair value, which is measured based on the projected discounted future net cash flows arising from the assets. Nosuch impairment losses have been recorded through December 31, 2013.Organizational costs All organizational costs have been expensed as incurred.Research and development costs The Company expenses research and development costs to operations as incurred. Research and development expenses consist of costs associatedwith research activities, including drug discovery efforts and the development of therapeutic product candidates and companion diagnostics. TheCompany accounts for nonrefundable advance payments for goods and services that will be used in future research and development activities asexpenses when the service has been performed or when the goods have been received rather than when the payment is made. Research and developmentexpenses consist of:•employee-related expenses, including salaries, benefits, travel and stock-based compensation expense; •external research and development expenses incurred under arrangements with third parties, such as contract research organizations, orCROs, clinical trial sites, manufacturing organizations and consultants, including the scientific advisory board; •license fees; and •facilities, depreciation and other expenses, which include direct and allocated expenses for rent and maintenance of facilities, depreciationof equipment, and laboratory supplies.F-13Laboratory equipment 5 yearsFurniture 5 yearsComputer equipment 3 yearsLeasehold Improvements Lesser of useful life or life of leaseTable of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued)Stock-based compensation The Company expenses the fair value of employee stock options over the requisite service period, which is the vesting period. Compensationexpense is measured using the fair value of the award at the grant date, net of estimated forfeitures, and is adjusted annually to reflect actual forfeitures.The grant date fair value of each stock-based award is estimated using the Black-Scholes option pricing model and is expensed on a straight-line basisover the vesting period. Awards subject to performance based vesting requirements are expensed utilizing an accelerated attribution model ifachievement of the performance criteria is determined to be probable. Stock-based awards issued to nonemployees, including directors for non-board related services, are accounted for based on the fair value of suchservices received or of the equity instruments issued, whichever is more reliably measured. Stock option awards are revalued at each reporting date andupon vesting using the Black-Scholes option pricing model and are expensed on a straight-line basis over the vesting period. Stock-based compensation awards which allow for greater than the minimum statutory tax withholdings are classified as liabilities. These awardsare revalued at each reporting date and are expensed on a straight-line basis over the vesting period. Upon settlement, the awards are revalued and theamounts are reclassified to additional paid-in capital. Shares are withheld to cover the tax withholding and amounts paid to settle the tax liability aretreated as a reduction of additional paid-in capital.Income taxes The Company accounts for income taxes under the asset and liability method. Deferred tax assets and liabilities are recognized for the future taxconsequences attributable to differences between the financial statement carrying amounts of existing assets and liabilities and their respective tax basesusing enacted tax rates in effect for the year in which the differences are expected to affect taxable income. Tax benefits are recognized when it is morelikely than not that a tax position will be sustained during an audit. Deferred tax assets are reduced by a valuation allowance if current evidence indicatesthat it is considered more likely than not that these benefits will not be realized.Net loss per share Basic and diluted net loss per common share is calculated by dividing net loss applicable to common stockholders by the weighted-average numberof common shares outstanding during the period, without consideration for common stock equivalents. The Company's potentially dilutive shares,which include redeemable convertible preferred stock, outstanding stock options, restricted stock units, and unvested restricted stock are considered tobe common stock equivalents and are only included in the calculation of diluted net loss per share when their effect is dilutive. All potentially dilutivesecurities were excluded from the calculation of diluted net loss per share as the securities were anti-dilutive for all periods presented.F-14Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)2. Significant accounting policies (Continued) The following potentially dilutive securities were excluded from the calculation of diluted net loss per share due to their anti-dilutive effect:3. Property and equipment Property and equipment and related accumulated depreciation are as follows (in thousands): Total depreciation expenses amounted to $83,000, $207,000, $236,000 and $526,000 for the years ended December 31, 2011, 2012 and 2013 andthe period from August 4, 2010 (inception) to December 31, 2013, respectively.4. Prepaid expenses and other current assets Prepaid expenses and other current assets consist of the following (in thousands):F-15 Period fromAugust 4, 2010(inception) toDecember 31,2013 Years Ended December 31, 2011 2012 2013 Preferred Stock 11,740,794 — — — Outstanding stock options 405,141 1,424,241 2,388,062 2,388,062 Restricted stock units — 899,204 529,850 529,850 Unvested restricted stock 1,434,734 747,000 329,282 329,282 13,580,669 3,070,445 3,247,194 3,247,194 December 31,2012 December 31,2013 Laboratory equipment $1,017 $1,035 Computer equipment 27 27 Furniture 44 44 Leasehold improvements — 38 1,088 1,144 Less: accumulated depreciation (277) (513) $811 $631 December 31,2012 December 31,2013 Prepaid contract research organization costs $274 $411 Interest receivable 161 123 Prepaid other 71 23 $506 $557 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)5. Accrued expenses Accrued expenses are as follows (in thousands):6. Redeemable convertible preferred stock On February 1, 2012, immediately prior to the closing of the Company's initial public offering, all outstanding shares of the Company's redeemableconvertible preferred stock were converted into 11,740,794 shares of common stock. As of December 31, 2013, the Company does not have anyredeemable convertible preferred stock issued or outstanding. In November 2010, the Company sold 4 million shares of Series A redeemable convertible preferred stock (Series A Preferred Stock) at a price of$1.00 per share for gross proceeds of $4 million. In accordance with the terms of the Series A Stock Purchase Agreement, the Company sold anadditional 12 million shares at $1.00 per share in a second subsequent closing. The Company incurred approximately $79,000 of issuance costs as partof the first closing of the Series A Preferred Stock. No additional issuance costs were incurred as part of the second closing. In July 2011, the Company sold approximately 16 million shares of series B redeemable convertible preferred stock (Series B Preferred Stock) at aprice of $2.00 per share for gross proceeds of approximately $32 million. The Company incurred approximately $113,000 of issuance costs as part ofthe closing of the Series B Preferred Stock. In November 2011, the Company sold approximately 9.1 million shares of Series C redeemable convertible preferred stock (Series C PreferredStock) at a price of $2.25 per share for gross proceeds of $20.4 million. The Company incurred approximately $153,000 of issuance costs as part of theclosing of the Series C Preferred Stock. The issuance costs associated with the Series A Preferred Stock, Series B Preferred Stock and Series CPreferred Stock (collectively, the Preferred Stock) were accreted through the earliest redemption date. The Company assessed the Preferred Stock for any beneficial conversion features or embedded derivatives that would require bifurcation from thePreferred Stock and receive separate accounting treatment. On the date of each issuance, the value of the common stock into which the Preferred Stockwas convertible had a fair value less than the effective conversion price of the Preferred Stock and, as such, there was no intrinsic value on therespective commitment dates. No embedded derivatives were identified that would require bifurcation.F-16 December 31,2012 December 31,2013 Contract research organization costs $69 $1,918 Compensation and related benefits 173 1,687 License milestones — 360 Professional fees 183 237 Deferred rent 36 38 Other 90 87 $551 $4,327 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)7. Common stock As of December 31, 2012 and 2013, the Company had reserved the following shares of common stock for the issuance of common stock forvested restricted stock units and the exercise of stock options (in thousands): Each share of common stock is entitled to one vote. The holders of the common stock are also entitled to receive dividends whenever funds arelegally available and when declared by the board of directors.Initial public offering In February 2012, the Company closed the initial public offering (IPO) of its common stock pursuant to a registration statement on Form S-1, asamended. An aggregate of 6,325,000 shares of common stock registered under the registration statement were sold at a price of $10.00 per share,including the over-allotment option. Net proceeds of the IPO were $56.8 million. All shares of the Company's Redeemable Convertible Preferred Stockwere converted into 11,740,794 shares of common stock as part of the IPO.Reverse stock split In January 2012, the Company's board of directors and stockholders approved a one-for-3.5 reverse stock split of the Company's common stock.The reverse stock split became effective on January 10, 2012. All share and per share amounts in the financial statements have been retroactivelyadjusted for all periods presented to give effect to the reverse stock split, including reclassifying an amount equal to the reduction in par value toadditional paid-in capital.8. Stock-based compensation Stock-based compensation expense as reflected in the Company's consolidated statements of operations and comprehensive loss was as follows (inthousands): Of the $10.4 million of stock-based compensation expense recorded during the year ended December 31, 2013, approximately $717,000 has beenrecorded as liability classified stock-basedF-17 December 31,2012 December 31,2013 Shares reserved under equity compensation plans 3,930 4,330 3,930 4,330 Period fromAugust 4, 2010(Inception) toDecember 31,2013 Year Ended December 31, 2011 2012 2013 Research and development $981 $2,956 $3,575 $7,536 General and administrative 654 4,444 6,818 11,944 Total stock-based compensation expense $1,635 $7,400 $10,393 $19,480 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)8. Stock-based compensation (Continued)compensation awards on the consolidated balance sheet, as the underlying awards allow for greater than minimum tax withholding. The Company has awards outstanding under two equity compensation plans, the 2012 Incentive Plan (the "2012 Plan") and the 2010 EquityIncentive Plan (the "2010 Plan"). Terms of stock award agreements, including vesting requirements, are determined by the board of directors, subject tothe provisions of the individual plans. To date, most options granted by the Company vest twenty five percent (25%) one year from vesting start dateand seventy-five percent (75%) in equal installments over the subsequent twelve quarters (subject to acceleration of vesting in the event of certainchange of control transactions) and are exercisable from the date of grant for a period of ten years.2012 Incentive Plan In December 2011, the Company adopted the 2012 Plan. The 2012 Plan became effective immediately upon the closing of the Company's IPO inFebruary 2012. Upon effectiveness of the 2012 Plan, the Company ceased making awards under the 2010 Plan. The 2012 Plan allows the Company togrant awards for up to 3,428,571 shares of common stock plus the number of shares of common stock available for grant under the 2010 Plan as of theeffectiveness of the 2012 Plan (which was an additional 30,101 shares) plus that number of shares of common stock related to awards outstandingunder the 2010 Plan which terminate by expiration, forfeiture, cancellation or otherwise. The 2012 Plan includes an "evergreen provision" that allowsfor an annual increase in the number of shares of common stock available for issuance under the 2012 Plan. The annual increase will be added on thefirst day of each year beginning in 2013 and each subsequent anniversary until the expiration of the 2012 Plan, equal to the lowest of 1,285,714 sharesof common stock, 4.0% of the number of shares of common stock outstanding and an amount determined by the board of directors. On January 1,2013, the number of shares available for issuance under the 2012 Plan increased by 844,448 shares under this provision. Awards under the 2012 Planmay include the following award types: incentive stock options, nonqualified stock options, stock appreciation rights, restricted stock awards, restrictedstock units (RSUs), other stock-based or cash-based awards and any combination of the foregoing. As of December 31, 2013, under the 2012 Plan, theCompany has granted stock options for 2,160,214 shares of common stock, of which 83,212 have been forfeited and restricted stock units for 909,918shares of common stock, of which 49,285 have been forfeited. The exercise price of each option has been equal to the closing price of a share of ourcommon stock on the grant date.2010 Equity Incentive Plan In November 2010, the Company adopted the 2010 Plan, under which it was authorized to grant stock-based awards to purchase up to 404,762shares of common stock to eligible employees, officers, directors and consultants. In March 2011, the 2010 Plan was amended to increase the aggregatenumber of shares of common stock available to be issued under the 2010 Plan to 571,242 shares of common stock. The Company granted a total of405,141 options and 256,000 restricted stock awards under the 2010 Plan, 5,395 options have been exercised and 126,891 have been forfeited andrepurchased. As of February 2012, the Company ceased making awards under the 2010 Plan and the remaining 30,101 shares available for futuregrants were added to the total number of shares reserved for issuance under the 2012 Plan. For options granted under the 2010 Plan, the exercise priceequaledF-18Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)8. Stock-based compensation (Continued)the estimated fair value of the common stock as determined by the board of directors on the date of grant.Restricted common stock In connection with the Company's formation, the founders purchased an aggregate of 2,857,138 shares of Company common stock at fair value onthe date of issuance. The shares were issued subject to restricted stock agreements between the Company and each founder, which allow the Company,at its discretion, to repurchase unvested shares if the founder's relationship with the Company is terminated. Under these agreements, twenty fivepercent (25%) of the shares vested immediately, and the remaining seventy-five percent (75%) of shares vest ratably in quarterly installments over thesubsequent four years. The Company records stock-based compensation expense for the common stock subject to repurchase, or restricted common stock grants, basedon the grant date fair value for employees and the reporting date and upon vesting fair value for non-employees. The fair value of the award isconsidered the intrinsic value as of each measurement date. All of the restricted shares were issued at a purchase price equal to the fair value of thecommon stock on the date of issuance. The Company recorded stock-based compensation expense associated with restricted common stock grants ofapproximately $1.4 million, $3.3 million, $3.4 million and $8.2 million for the years ended December 31, 2011, 2012 and 2013 and the period fromAugust 4, 2010 (inception) to December 31, 2013, respectively. A summary of the Company's restricted stock activity and related information is as follows: The weighted-average grant date fair value of restricted stock granted during the year ended December 31, 2011 and the period from August 4,2010 (inception) to December 31, 2013 was $0.28 and $0.02 per share, respectively. No restricted stock was granted during the years endedDecember 31, 2012 and 2013. The total fair value of shares vested during the years ended December 31, 2011, 2012, 2013 and for the period fromAugust 4, 2010 (inception) to December 31, 2013 was approximately, $1.1 million, $3.4 million, $3.3 million and $7.9 million, respectively. As ofDecember 31, 2013, there was $2.0 million of total unrecognized stock-based compensation expense related to unvested restricted stock. The Companyexpects to recognize this expense over a remaining weighted-average period of 0.75 years.F-19 Shares Weighted-averagefair valueper share Unvested at December 31, 2012 747,000 $0.027 Vested (417,718) 0.022 Unvested at December 31, 2013 329,282 $0.034 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)8. Stock-based compensation (Continued)Restricted stock units A summary of the Company's RSUs and related information is as follows: The weighted-average grant date fair value of restricted stock units granted during the year ended December 31, 2012 and the period fromAugust 4, 2010 (inception) to December 31, 2013 was $10.62. No restricted stock units were granted during the years ended December 31, 2011 and2013. The total fair value of restricted stock units vested during the years ended December 31, 2013 and the period from August 4, 2010 (inception) toDecember 31, 2013 was $3.4 million. No restricted stock units vested during the years ended December 31, 2011 and 2012. As of December 31, 2013,there was $4.3 million of total unrecognized stock-based compensation expense related to unvested RSUs granted under the 2012 Plan. The expense isexpected to be recognized over a weighted-average period of 2.1 years. During 2012, the Company issued a restricted stock unit for 103,306 shares to an employee. The award vests up to 25% per year based on theachievement of stated objectives. The objectives related to 2013 were established on January 15, 2013 and the objectives were determined to be met onDecember 17, 2013. The Company recorded $294,000 of stock-based compensation expense in 2013 based on the achievement of the stated objectives.The objectives related to the 2014 and 2015 objectives were not determined as of December 31, 2013 and therefore the Company did not have sufficientinformation to determine if the achievement of the objectives was probable, and therefore no expense related to these objectives has been recorded as ofDecember 31, 2013. During the first quarter of 2013, the Company amended the terms of certain RSUs related to a total of 697,060 shares of common stock to allowfor tax withholdings greater than the minimum required statutory withholding amount. As a result of this change in the terms of the awards, theoutstanding RSUs are considered to be liability instruments. As a result of this modification, the Company records a liability for the fair value of theawards as of each reporting date with the change in fair value recorded through the statement of operations. The Company will record stock-basedcompensation expense equal to the greater of the original grant date fair value of the awards or the settlement date fair value. During the year endedDecember 31, 2013, the Company deposited with tax authorities $1.3 million to settle the tax liability for awards that settled during the respectiveperiods. The liability related to these awards of approximately $717,000 is recorded as liability classified stock-based compensation awards on theconsolidated balance sheet as of December 31, 2013.F-20 Shares Weighted-averagegrant datefair valueper share Unvested at December 31, 2012 899,204 $10.70 Vested (330,783)$10.54 Forfeited (38,571)$11.00 Unvested at December 31, 2013 529,850 $10.78 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)8. Stock-based compensation (Continued)Stock Options A summary of the Company's stock option activity and related information follows: The fair value of each employee stock option was estimated at the date of grant using a Black-Scholes option-pricing model with the followingassumptions: The Company uses the simplified method as prescribed by the Securities and Exchange Commission Staff Accounting Bulletin Topic 14.D.2 tocalculate the expected term as it does not have sufficient historical exercise data to provide a reasonable basis upon which to estimate the expected termfor options granted to employees. The expected term is applied to the stock option grant group as a whole, as the Company does not expect substantiallydifferent exercise or post-vesting termination behavior among its employee population. The computation of expected volatility is based on the historicalvolatility of a representative group of public biotechnology and life sciences companies with similar characteristics to the Company, including earlystage of product development and therapeutic focus. The Company also considers including its own historical volatility based on future expectations.The risk-free interest rate is based on a treasury instrument whose term is consistent with the expected term of the stock options. Management assessesexpected forfeitures based on the experience of the Company coupled with comparison to data from the representative group of companies andrecognizes compensation costs only for those equity awards expected to vest.F-21 Shares Weighted-averageexercise price pershare Weighted-averageremainingcontractual term(years) Aggregateintrinsic value(in thousands) Outstanding at December 31, 2012 1,424,241 $6.90 9.3 $3,451 Granted 1,120,500 $10.11 Exercised (81,795)$0.40 Forfeited (74,884)$5.88 Outstanding at December 31, 2013 2,388,062 $8.66 8.8 $6,768 Vested at December 31, 2013 775,479 $7.50 8.5 $3,032 Vested and expected to vest atDecember 31, 2013(1) 2,261,959 $8.60 8.8 $6,544 (1)This represents the number of vested options as of December 31, 2013, plus the number of unvested options expected to vest asof December 31, 2013, based on the unvested options at December 31, 2013, adjusted for the estimated forfeiture rate. Year EndedDecember 31, 2011 2012 2013 Risk-free interest rate 1.57% 1.14% 1.14%Volatility 69% 76% 75%Dividend yield — — — Expected term (years) 6.1 6.0 6.0 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)8. Stock-based compensation (Continued) The Company recorded stock-based compensation expense associated with employee stock options of $19,000, $1.6 million, $6.8 million and$8.4 million for the years ended December 31, 2011, 2012 and 2013 and the period from August 4, 2010 (inception) to December 31, 2013,respectively. The weighted-average grant date fair value of options granted in the years ended December 31, 2011, 2012 and 2013 and the period fromAugust 4, 2010 (inception) to December 31, 2013 was $0.75, $6.03, $6.55 and $5.73 per share, respectively. At December 31, 2013, there was $8.2 million of total unrecognized compensation cost related to unvested stock options. As of December 31,2013, the Company expects to recognize this cost over a remaining weighted-average period of 2.7 years.9. Income taxes As of December 31, 2013 the Company had federal net operating loss carryforwards of approximately $65.7 million and state net operating losscarryforwards of $65.0 million, which are available to reduce future taxable income. The Company also had federal tax credits of $1.5 million and statetax credits of $514,000, which may be used to offset future tax liabilities. The net operating loss (NOL) and tax credit carryforwards will expire atvarious dates through 2033. Net operating loss and tax credit carryforwards are subject to review and possible adjustment by the Internal RevenueService and state tax authorities and may become subject to an annual limitation in the event of certain cumulative changes in the ownership interest ofsignificant shareholders over a three-year period in excess of 50%, as defined under Sections 382 and 383 of the Internal Revenue Code, as well assimilar state provisions. This could limit the amount of tax attributes that can be utilized annually to offset future taxable income or tax liabilities. Theamount of the annual limitation is determined based on the value of the Company immediately prior to the ownership change. Subsequent ownershipchanges may further affect the limitation in future years. A reconciliation of income taxes computed using the U.S. federal statutory rate to that reflected in operations follows:F-22 Year endedDecember 31, 2012 2013 Income tax benefit using U.S. federal statutory rate 34.00% 34.00%State tax benefit, net of federal benefit 4.61% 4.69%Research and development tax credits —% 1.67%Permanent items (5.96)% (4.76)%Change in the valuation allowance (32.19)% (35.20)%Other (0.46)% (0.40)% —% —% Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)9. Income taxes (Continued) The principal components of the Company's deferred tax assets are as follows (in thousands): The Company has recorded a valuation allowance against its deferred tax assets at December 31, 2013 because the Company's managementbelieves that it is more likely than not that these assets will not be fully realized. The increase in the valuation allowance of $4.9 million, $10.3 million,$14.5 million and $29.7 million in the years ended December 31, 2011, 2012 and 2013 and the period from August 4, 2010 (inception) throughDecember 31, 2013, respectively, primarily relates to the net loss incurred by the Company. The Company's reserves related to taxes are based on a determination of whether and how much of a tax benefit taken by the Company in its taxfilings or positions is more likely than not to be realized following resolution of any potential contingencies present related to the tax benefit. Frominception and through December 31, 2013, the Company had no unrecognized tax benefits or related interest and penalties accrued. The Company hasnot, as yet, conducted a study of research and development (R&D) credit carryforwards. This study may result in an adjustment to the Company's R&Dcredit carryforwards; however, until a study is completed and any adjustment is known, no amounts are being presented as an uncertain tax position. Afull valuation allowance has been provided against the Company's R&D credits and, if an adjustment is required, this adjustment would be offset by anadjustment to the valuation allowance. Thus, there would be no impact to the consolidated balance sheet or statement of operations if an adjustment wererequired. The Company would recognize both accrued interest and penalties related to unrecognized benefits in income tax expense. The Company'suncertain tax positions are related to years that remain subject to examination by relevant tax authorities. Since the Company is in a loss carryforwardposition, the Company is generally subject to examination by the U.S. federal, state and local income tax authorities for all tax years in which a losscarryforward is available.10. Commitments and contingencies From November 2010 through May 2011, the Company leased office space from a shareholder. There was no formal lease arrangement with theshareholder. Rent paid to the shareholder was $34,000 and $46,000 for the year ended December 31, 2011 and for the period from August 4, 2010(inception)F-23 December 31, 2012 2013 Deferred tax assets: Net operating loss carryforwards $12,532 $25,782 Capitalized research and development 1,664 1,839 Research and development credits 508 1,856 Stock-based compensation 797 511 Other (23) (4) Gross deferred tax assets 15,478 29,984 Valuation allowance (15,478) (29,984) Net deferred tax asset $— $— Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)10. Commitments and contingencies (Continued)to December 31, 2013, respectively. There was no rent paid to a shareholder for the years ended December 31, 2012 and 2013. In May 2011, the Company entered into a non-cancelable operating lease for office and laboratory space, which expires October 31, 2014. Thelease agreement provides for free rent for the first four months of the lease term and includes escalating rent payments. The rent expense is recorded ona straight-line basis over the lease term. The Company is also obligated to pay for certain operating costs and a proportional share of certain commonarea costs. The Company has the right to extend the lease for a two-year period. The annual rent for each additional year is determined annually at thethen fair market rate. The Company secured a letter of credit for $86,000 in connection with the lease, which is included in restricted cash on theconsolidated balance sheets. The minimum aggregate future lease commitments are as follows (in thousands): The Company recorded rent expense of $251,000, $323,000, $366,000 and $952,000 for the year ended December 31, 2011, the year endedDecember 31, 2012, the year ended December 31, 2013 and the period from August 4, 2010 (inception) to December 31, 2013, respectively. Pursuant to the terms of various agreements, the Company may be required to pay various development, regulatory and commercial milestones. Inaddition, if any products related to these agreements are approved for sale, the Company may be required to pay significant royalties on future sales. Thepayment of these amounts, however, is contingent upon the occurrence of various future events, which have a high degree of uncertainty of occurring.11. License agreements In October 2010, the Company entered into an exclusive license agreement, which was amended and restated in January 2012, with the WhiteheadInstitute for Biomedical Research (the Licensor) for certain intellectual property. The Company paid the Licensor an upfront license fee and reimbursedpatent related fees and costs incurred by the Licensor and affiliates of the Licensor totaling $104,000 in the aggregate and issued 166,664 shares ofcommon stock to the Licensor and entities and individuals affiliated with the Licensor. The fair value of the common stock was determined to be $0.28per share, and the fair value was determined to be more readily determinable than the fair value of the license. As a result, the fair value of the shares ofapproximately $46,000 was recorded as research and development expense. Under the terms of the agreement, the Company also agreed to pay annuallicense maintenance fees, milestone payments, royalties as a percentage of net sales and a percentage of sublicense income the Company receives.Annual license maintenance fees are creditable against royalties earned during the same calendar year and are not material to the financial statements.Milestone payments are triggered upon the achievement of specified development, regulatory and commercialization milestones and are not creditableagainst royalties. Actual amounts due under the agreement will vary depending on the number of products developed, the type and development path ofthe products, and other related factors. Milestone payments could total up to $1.6 million. The Company may terminate the agreement at any time with90 days' prior written notice. On December 16, 2011, the Company amended and restated an existing non-exclusive license agreement with the Licensor pursuant to which theCompany obtained an exclusive license to certainF-242014 $307 Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)11. License agreements (Continued)intellectual property. The Company paid the Licensor an upfront license fee and agreed to make milestone payments of up to $825,000 upon theachievement of specified regulatory and commercialization milestones. In addition, the Company agreed to pay royalties as a percentage of net sales oflicensed products. Under the license agreement with Poniard that the Company entered into in November 2011 relating to VS-4718 and certain other compounds, theCompany paid an upfront license fee and agreed to pay Poniard milestone payments upon the achievement of specified development and regulatorymilestones. In February 2014, the Company purchased the assets which were the subject of the license agreement with Poniard from Encarta, Inc.("Encarta"), who had previously purchased these assets in 2013. In consideration for these assets, the Company issued 97,500 shares of common stock,a warrant to purchase 142,857 shares of common stock with an exercise price equal to $17.16 per share and paid $25,000. All existing obligationsunder the license agreement, including an achieved development milestone and an obligation to issue a warrant, were settled as part of this transaction.The Company expects to incur $1.3 million of research and development expense in 2014 as a result of this transaction. In connection with the assetpurchase agreement, we also assumed the rights and obligations under the Scripps License Agreement. Pursuant to the Scripps License Agreement, theCompany is obligated to pay Scripps potential product development milestone payments of up to an aggregate of $3,000,000 upon the achievement ofspecified development and regulatory milestones. In addition, the Company is obligated to pay Scripps low single-digit royalties as a percentage of netsales of licensed products, subject to adjustments in certain circumstances. The Company's obligation to pay royalties on net sales is on a country bycountry basis. On May 11, 2012, the Company acquired from S*Bio Pte Ltd (S*Bio), compounds identified as dual inhibitors of PI3K and mTOR, includingrelated patent rights. Under the agreement, the Company paid S*Bio an upfront fee of $350,000 and has agreed to pay S*Bio milestone payments of upto an aggregate of approximately $21.0 million upon the achievement of specified development and regulatory milestones. In addition, the Companyagreed to pay to S*Bio tiered, low to mid single digit royalties as a percentage of annual net sales of each product containing an acquired compound asan ingredient. The obligation to pay royalties continues on a product by product and country by country basis until the expiration of all acquired patentrights covering the product in such country. If the Company obtains a license from a third party in order to commercialize an acquired compoundcontained in a product in a particular country, then the Company may deduct up to 50% of the amount paid to such third party from the royaltypayments that Company owes to S*Bio for such product. The deduction is subject to specified limitations, including that in no event will any suchdeduction reduce a royalty payment owed to S*Bio by more than 50% as a result of all such deductions in the aggregate. There were no ongoing clinicaltrials at the time of the acquisition of the compounds, and the compounds acquired do not have alternative future uses, nor have they reached a stage oftechnological feasibility. As no process or activities were acquired, the Company accounted for the transaction as an asset acquisition by recording the$350,000 payment made to S*Bio to research and development expense for the year ended December 31, 2012. On July 11, 2012, the Company entered into a license agreement with Pfizer Inc. (Pfizer), under which Pfizer granted the Company worldwide,exclusive rights to research, develop, manufacture and commercialize products containing certain of Pfizer's inhibitors of focal adhesion kinase(Products) for all therapeutic, diagnostic and prophylactic uses in humans. The Company is solely responsible, at itsF-25Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)11. License agreements (Continued)expense, for the clinical development of the Products, which is to be conducted in accordance with an agreed-upon development plan. The Company isalso responsible for all manufacturing and commercialization activities at its own expense. Pfizer is required to provide the Company with an initialquantity of clinical supply of one of the Products for an agreed upon price. Under the agreement, the Company made a one-time cash payment to Pfizerin the amount of $1.5 million and issued 192,012 shares of its common stock. Pfizer is also eligible to receive up to $2 million in developmentalmilestones and up to an additional $125 million based on the successful attainment of regulatory and commercial sales milestones. Pfizer is also eligibleto receive high single to mid double digit royalties on future net sales of Products. The Company's royalty obligations with respect to each Product ineach country begin on the date of first commercial sale of the Product in that country, and end on the later of 10 years after the date of first commercialsale of the Product in that country or the date of expiration or abandonment of the last claim contained in any issued patent or patent application licensedby Pfizer to the Company that covers the Product in that country. The Company accounted for the license agreement as the licensing of in-processresearch and development with no alternative future use. Accordingly, the Company recorded research and development expense for the year endedDecember 31, 2012 associated with the cash payment of $1.5 million and the fair value of shares of common stock issued to Pfizer of $2.0 million.12. Employee benefit plan In June 2011, the Company adopted a 401(k) retirement and savings plan (the 401(k) Plan) covering all employees. The 401(k) Plan allowsemployees to make pre-tax or post-tax contributions up to the maximum allowable amount set by the IRS. Under the 401(k) Plan, the Company maymake discretionary contributions as approved by the board of directors. The Company made contributions to the 401(k) Plan of $46,000, $116,000,$160,000 and $322,000 for the period from the years ended December 31, 2011, 2012 and 2013 and the period from August 4, 2010 (inception) toDecember 31, 2013, respectively.F-26Table of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)13. Quarterly Financial Information (unaudited, in thousands, except per share data) First QuarterEndedMarch 31,2013 Second QuarterEndedJune 30,2013 Third QuarterEndedSeptember 30,2013 Fourth QuarterEndedDecember 31,2013 Operating expenses: Research and development $5,296 $6,045 $6,789 $7,800 General and administrative 3,785 4,239 3,855 3,593 Total operating expenses 9,081 10,284 10,644 11,393 Loss from operations (9,081) (10,284) (10,644) (11,393)Interest income 44 34 53 69 Net loss $(9,037)$(10,250)$(10,591)$(11,324) Net loss per share applicable tocommon stockholders—basic anddiluted $(0.44)$(0.49)$(0.44)(d)$(0.45)Weighted-average number of commonshares used in net loss per shareapplicable to common stockholders—basic and diluted 20,483 20,729 24,127(b)(d) 25,277 First QuarterEndedMarch 31,2012 Second QuarterEndedJune 30,2012 Third QuarterEndedSeptember 30,2012 Fourth QuarterEndedDecember 31,2012 Operating expenses: Research and development $4,803 $4,683 $8,132(c)$4,094 General and administrative 2,125 2,213 2,298 3,882 Total operating expenses 6,928 6,896 10,430 7,976 Loss from operations (6,928) (6,896) (10,430) (7,976)Interest income 57 71 63 55 Net loss $(6,871)$(6,825)$(10,367)$(7,921) Net loss per share applicable to commonstockholders—basic and diluted $(0.47)$(0.34)$(0.51)$(0.39)Weighted-average number of commonshares used in net loss per shareapplicable to common stockholders—basic and diluted 14,693(a) 19,863 20,160 20,309 (a)In February 2012, the Company completed its initial public offering, which resulted in net proceeds of approximately$56.8 million from the issuance of 6,325,000 shares of common stock, which includes the sale of 825,000 shares under theunderwriters' over-allotment option. In connection with the initial public offering, all of the outstanding shares of the Company'sredeemable convertible preferred stock were converted into 11,740,794 of common stock. F-27(b)In July 2013, the Company closed a public offering in which it sold 4,255,000 shares of its common stock to the public at a priceof $15.00 per share, including 555,000 shares issued pursuant to theTable of ContentsVerastem, Inc.(A development stage company)NOTES TO CONSOLIDATED FINANCIAL STATEMENTS (Continued)13. Quarterly Financial Information (unaudited, in thousands, except per share data) (Continued) The Company was incorporated on August 4, 2010; however, it did not commence operations until the fourth quarter of 2010. Activity incurredduring the period from August 4, 2010 (inception) to September 30, 2010 is limited to organizational costs which were expensed when incurred.14. Subsequent events The Company reviews all activity subsequent to year end but prior to the issuance of the consolidated financial statements for events that couldrequire disclosure or that could impact the carrying value of assets or liabilities as of the balance sheet date. All significant subsequent events have beenproperly disclosed in the consolidated financial statements.F-28exercise of the underwriters' option to purchase additional shares, which resulted in net proceeds of approximately $59.8 million.(c)In the quarter ended September 30, 2012, the Company paid a license fee of $1.5 million in cash and issued 192,012 shares ofthe Company's common stock, which were valued at $2.0 million on the date of issuance. (d)Adjusted to correct an immaterial error in weighted-average share calculation.Table of ContentsEXHIBIT INDEX Exhibitnumber Description of exhibit 3.1 Restated Certificate of Incorporation of the Registrant (incorporated by reference to Exhibit 3.1 to AnnualReport on Form 10-K (File No. 001-35403) filed by the Registrant on March 30, 2012) 3.2 Amended and Restated Bylaws of the Registrant (incorporated by reference to Exhibit 3.4 to AmendmentNo. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant onJanuary 13, 2012) 4.1 Second Amended and Restated Investors' Rights Agreement, dated November 1, 2011, by and among theRegistrant and the other parties thereto (incorporated by reference to Exhibit 4.2 to the RegistrationStatement on Form S-1 (File No. 333-177677) filed by the Registrant on November 3, 2011) 4.2 Registration Rights Agreement, dated July 11, 2012, by and between the Registrant and Pfizer Inc.(incorporated by reference to Exhibit 4.1 to Current Report on Form 8-K (File No. 001-35403) filed bythe Registrant on July 11, 2012) 4.3 Specimen certificate evidencing shares of common stock (incorporated by reference to Exhibit 4.1 toAmendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filed by theRegistrant on January 13, 2012) 10.1#2010 Equity Incentive Plan (incorporated by reference to Exhibit 10.1 to the Registration Statement onForm S-1 (File No. 333-177677) filed by the Registrant on November 3, 2011) 10.2#2012 Incentive Plan (incorporated by reference to Exhibit 10.2 to Amendment No. 3 to the RegistrationStatement on Form S-1 (File No. 333-177677) filed by the Registrant on January 13, 2012) 10.3#Form of Incentive Stock Option Agreement under 2012 Incentive Plan (incorporated by reference toExhibit 10.3 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filedby the Registrant on January 13, 2012) 10.4#Form of Nonqualified Stock Option Agreement under 2012 Incentive Plan (incorporated by reference toExhibit 10.4 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filedby the Registrant on January 13, 2012) 10.5#Form of Restricted Stock Unit Agreement under 2012 Incentive Plan (incorporated by reference toExhibit 10.16 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677)filed by the Registrant on January 13, 2012) 10.6#Amended and Restated Employment Agreement between the Registrant and Robert Forrester(incorporated by reference to Exhibit 10.5 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant on January 13, 2012) 10.7#Amended and Restated Employment Agreement between the Registrant and Jonathan Pachter(incorporated by reference to Exhibit 10.6 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant on January 13, 2012) 10.8#Form of Indemnification Agreement between the Registrant and each director (incorporated by referenceto Exhibit 10.7 to Amendment No. 1 to the Registration Statement on Form S-1 (File No. 333-177677)filed by the Registrant on December 7, 2011) Table of ContentsExhibitnumber Description of exhibit 10.9 Lease Agreement, dated May 2, 2011, between the Registrant and ARE-MA Region No. 38, LLC(incorporated by reference to Exhibit 10.8 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant on November 3, 2011) 10.10†Amended and Restated Exclusive Patent License Agreement and Tangible Property Agreement, datedJanuary 11, 2012, by and among the Registrant and the Whitehead Institute for Biomedical Research(incorporated by reference to Exhibit 10.9 to Amendment No. 3 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant on January 13, 2012) 10.11†Exclusive Patent License Agreement, dated December 16, 2011, by and among the Registrant and theWhitehead Institute for Biomedical Research (incorporated by reference to Exhibit 10.10 to AmendmentNo. 2 to the Registration Statement on Form S-1 (File No. 333-177677) filed by the Registrant onDecember 22, 2011) 10.12 License Agreement, dated November 17, 2011, between the Registrant and Poniard Pharmaceuticals, Inc.(incorporated by reference to Exhibit 10.15 to Amendment No. 2 to the Registration Statement onForm S-1 (File No. 333-177677) filed by the Registrant on December 22, 2011) 10.13†Letter Agreement, dated October 1, 2010, between the Registrant and the Broad Institute (incorporated byreference to Exhibit 10.11 to the Registration Statement on Form S-1 (File No. 333-177677) filed by theRegistrant on November 3, 2011) 10.14#Letter Agreement, dated August 20, 2010, between the Registrant and Eric Lander, Ph.D. (incorporatedby reference to Exhibit 10.13 to the Registration Statement on Form S-1 (File No. 333-177677) filed bythe Registrant on November 3, 2011) 10.15#Letter Agreement, dated July 30, 2010, as amended October 18, 2010, between the Registrant and RobertWeinberg, Ph.D. (incorporated by reference to Exhibit 10.14 to the Registration Statement on Form S-1(File No. 333-177677) filed by the Registrant on November 3, 2011) 10.16#Restricted Stock Purchase Agreement, dated August 11, 2010, between the Registrant and ChristophWestphal (incorporated by reference to Exhibit 10.17 to Annual Report on Form 10-K (File No. 001-35403) filed by the Registrant on March 30, 2012) 10.17#Restricted Stock Purchase Agreement, dated August 11, 2010, between the Registrant and RichardAldrich (incorporated by reference to Exhibit 10.18 to Annual Report on Form 10-K (File No. 001-35403) filed by the Registrant on March 30, 2012) 10.18#Employment Agreement, dated March 1, 2012, between the Registrant and Daniel Paterson (incorporatedby reference to Exhibit 10.18 to Annual Report on Form 10-K (File No. 001-35403) filed by theRegistrant on March 26, 2013) 10.19†Asset Purchase Agreement, dated May 10, 2012, by and between the Registrant and S*Rio Pte Ltd.(incorporated by reference to Exhibit 10.1 to Quarterly Report on Form 10-Q (File No. 001-35403) filedby the Registrant on August 13, 2012) 10.20†License Agreement, dated July 11, 2012, by and between the Registrant and Pfizer Inc. (incorporated byreference to Exhibit 10.2 to Quarterly Report on Form 10-Q (File No. 001-35403) filed by the Registranton August 13, 2012) 10.21#Offer Letter, dated as of September 18, 2012, by and between the Registrant and Christoph Westphal,M.D., Ph.D. (incorporated by reference to Exhibit 10.1 to Quarterly Report on Form 10-Q (File No. 001-35403) filed by the Registrant on November 13, 2012)35403) filed by the Registrant on November 13, 2012) Table of ContentsExhibitnumber Description of exhibit 10.22#Restricted Stock Unit Agreement, dated as of September 18, 2012, by and between the Registrant andChristoph Westphal, M.D., Ph.D. (incorporated by reference to Exhibit 10.2 to Quarterly Report onForm 10-Q (File No. 001-35403) filed by the Registrant on November 13, 2012) 10.23#Restricted Stock Unit Agreement, dated as of September 18, 2012, by and between the Registrant andChristoph Westphal, M.D., Ph.D. (incorporated by reference to Exhibit 10.3 to Quarterly Report onForm 10-Q (File No. 001-35403) filed by the Registrant on November 13, 2012) 10.24#Employment Agreement, dated as of October 23, 2012, by and between the Registrant and JoannaHorobin (incorporated by reference to Exhibit 10.4 to Quarterly Report on Form 10-Q (File No. 001-35403) filed by the Registrant on November 13, 2012) 10.25#Amendment to Form of Restricted Stock Unit Agreement under 2012 Incentive Plan (incorporated byreference to Exhibit 10.25 to Annual Report on Form 10-K (File No. 001-35403) filed by the Registranton March 26, 2013) 10.26#Employment Agreement, dated May 10, 2013, by and between the Registrant and John B. Green, CPA(incorporated by reference to Exhibit 99.2 to current report on Form 8-K (File No. 001-35403) filed bythe Registrant on May 14, 2013) 10.27#Letter Agreement, dated June 6, 2013, by and between the Registrant and Robert Forrester (incorporatedby reference to Exhibit 10.2 to Quarterly Report on Form 10-Q (File No. 001-35403) filed by theRegistrant on August 13, 2013) 10.28 Letter Agreement, dated December 1, 2011 by and between the Registrant and PoniardPharmaceuticals, Inc. (incorporated by reference to Exhibit 10.1 to Quarterly Report on Form 10-Q (FileNo. 001-35403) filed by the Registrant on November 12, 2013) 10.29 Letter Agreement, dated March 6, 2013 by and among the Registrant, The Scripps Research Institute andPoniard Pharmaceuticals, Inc. (incorporated by reference to Exhibit 10.2 to Quarterly Report on Form 10-Q (File No. 001-35403) filed by the Registrant on November 12, 2013) 10.30†Consent and Assumption Agreement, dated September 4, 2013, by and between the Registrant andEncarta Inc. and joined in by Poniard, LLC (incorporated by reference to Exhibit 10.2 to Quarterly Reporton Form 10-Q (File No. 001-35403) filed by the Registrant on November 12, 2013) 10.31*‡Letter Agreement, dated December 7, 2012, by and between the Registrant and Pfizer Inc. 10.32*#Amended and Restated Employment Agreement, dated November 22, 2013, by and between theRegistrant and Robert Forrester 10.33#Letter Agreement with Robert Forrester, dated June 6, 2013 (incorporated by reference to Exhibit 10.2 toQuarterly Report on Form 10-Q (File No. 001-35403) filed by the Registrant on August 13, 2013) 21.1*Subsidiaries of the Registrant 23.1*Consent of Ernst & Young LLP 31.1*Certification of the Chief Executive Officer pursuant to Exchange Act Rule 13a-14(a) 31.2*Certification of the Chief Financial Officer pursuant to Exchange Act Rule 13a-14(a) 32.1*Certification of the Chief Executive Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant to 32.1*Certification of the Chief Executive Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant toSection 906 of the Sarbanes-Oxley Act of 2002. Table of ContentsExhibitnumber Description of exhibit 32.2*Certification of the Chief Financial Officer pursuant to 18 U.S.C. Section 1350, as adopted pursuant toSection 906 of the Sarbanes-Oxley Act of 2002. 101.INS††XBRL Instance Document 101.SCH††XBRL Taxonomy Extension Schema Document 101.CAL††XBRL Taxonomy Extension Calculation Linkbase Document 101.DEF††XBRL Taxonomy Extension Definition Linkbase Document 101.LAB††XBRL Taxonomy Extension Label Linkbase Document††Submitted electronically herewith. *Filed herewith. †Confidential treatment granted as to portions of the exhibit. Confidential materials omitted and filed separately with the Securitiesand Exchange Commission. ‡Confidential treatment requested under 17 C.F.R. §200.80(b)(4) and Rule 24b-2. The confidential portions of this exhibit havebeen omitted and are marked accordingly. The confidential portions have been provided separately to the SEC pursuant to theconfidential treatment request. #Management contract or compensatory plan, contract or agreement.Exhibit 10.31 [**] = Portions of this exhibit have been omitted pursuant to a confidential treatment request. An unredacted version of the exhibit has been filed separatelywith the Commission. PFIZER INC.235 East 42nd StreetNew York, New York 10017 December 7, 2012 Verastem, Inc.215 First Street, Suite 440Cambridge, MA 02142 Ladies and Gentlemen: Reference is made to the license agreement dated as of July 11, 2012 (the “License Agreement”) between Verastem, Inc. (“Verastem”) and Pfizer Inc. (“Pfizer”). Capitalized terms used but not defined herein shall have the meanings set forth in the License Agreement. Verastem and Pfizer desire to amend the LicenseAgreement to include the manufacture and supply by Pfizer of certain quantities of active pharmaceutical ingredient of PF-04554878 as set forth herein, andthis letter agreement sets forth the agreement of Verastem and Pfizer with respect to such amendment. Each of Verastem and Pfizer agrees as follows: 1. Section 4.4 of the License Agreement hereby is amended by inserting the following paragraph at the end of such Section: 4.4.3. Additional API Manufacturing. Pfizer shall manufacture and supply to LICENSEE approximately [**] kg of the activepharmaceutical ingredient of PF-04554878 (the “API”). Upon delivery of such API, LICENSEE shall pay to Pfizer $[**]. Pfizer shall useits commercially reasonable efforts to ship such API by April 30, 2013. Pfizer shall be responsible for procuring PF-00844048-14 of asufficient quantity to manufacture such API and shall also procure additional PF-00844048-14 of a sufficient quantity to permit themanufacture of up to an additional [**] kg of APT (the “Additional API”) on or prior to December 31, 2013, provided, however, that Pfizershall not be under any obligations to manufacture and supply to LICENSEE, and LICENSEE shall be under no obligation to purchase theAdditional API unless Pfizer and LICENSEE enter into a written agreement providing for such manufacture and supply, and in no eventshall LICENSEE have any right, title or interest in any raw materials except to the extent they are  used in the manufacture of API or Additional API purchased by LICENSEE. 2. Section 2.1 of Schedule B of the License Agreement hereby is amended by deleting such section and replacing it with the following: 2.1. Pharmaceutical Sciences/Manufacturing. PFIZER shall package and ship those amounts of Compound in PFIZER’s inventory to theextent set forth on Schedule C to a storage facility of LICENSEE’s choosing; such transfer will take place within [**] Business Daysfollowing PFIZER’s receipt from LICENSEE of its written nomination of a storage facility. Notwithstanding anything to the contraryherein, LICENSEE must identify this facility no later than [**] months after the Effective Date. LICENSEE shall bear all costs andexpenses incurred by PFIZER after the Effective Date related to packaging and shipping of the Compound pursuant to this Section of thisSchedule. Notwithstanding anything to the contrary in the License Agreement (including in this Schedule B), Pfizer shall use commerciallyreasonable efforts to transfer the [**] kg of API, PF-04554878 listed in Schedule C to LICENSEE by April 30, 2013. As modified by this letter agreement, the parties confirm that the License Agreement is in full force and effect except as noted herein. Please indicate your acknowledgement of and agreement with the foregoing by having each counterpart of this letter agreement executed on behalf of Verastem. Very truly yours, PFIZER INC. By:/s/ John A. DeYoungName:John A. DeYoungTitle:Vice President 2 ACKNOWLEDGED AND AGREED: VERASTEM, INC. By:/s/ Daniel PatersonName:Daniel PatersonTitle:V.P. Corporate Development 3Exhibit 10.32 AMENDED AND RESTATED EMPLOYMENT AGREEMENT THIS AMENDED AND RESTATED EMPLOYMENT AGREEMENT (the “Agreement”), dated November, 22, 2013, by and betweenVerastem, Inc. (the “Company”), a Delaware corporation with its principal place of business at 215 First Street, Suite 440, Cambridge, MA 02199, andRobert Forrester (the “Executive”) of 346 Gay Street, Westwood, MA 02142 amends and restates in its entirety the Amended and Restated EmploymentAgreement, dated as of January 13, 2012 between the Company and the Executive. WHEREAS, the Executive is possessed of certain experience and expertise that qualify him to provide management direction and leadership for theCompany. WHEREAS, the Company has employed the Executive to serve as its Chief Operating Officer and now wishes to employ the Executive to serve as itsChief Executive Officer. NOW, THEREFORE, in consideration of the mutual covenants and promises contained herein and other good and valuable consideration, the receiptand sufficiency of which are hereby acknowledged, the Company offers and the Executive accepts employment upon the following terms and conditions: 1. Position and Duties. Upon the terms and subject to the conditions set forth in this Agreement, the Company hereby offers and theExecutive hereby accepts continued employment with the Company to serve as its Chief Executive Officer. The Executive agrees to perform the duties of theExecutive’s position and such other duties as reasonably may be assigned to the Executive from time to time. The Executive also agrees that while employed bythe Company, the Executive will devote one hundred percent (100%) of the Executive’s business time and the Executive’s reasonable commercial efforts,business judgment, skill and knowledge exclusively to the advancement of the business and interests of the Company and to the discharge of the Executive’sduties and responsibilities for it. The Executive may, however, hold one (1) outside directorship with a publicly traded company and one (1) outsidedirectorship with a privately held or not for profit company, in each case with the prior approval of the Nominating Committee of the Board of Directors of theCompany (the “Board”), with such approval not to be unreasonably withheld. 2. Compensation and Benefits. During the Executive’s employment, as compensation for all services performed by the Executive for theCompany, the Company will provide the Executive the following pay. (a) Base Salary; Annual Bonus. Retroactive to July 1, 2013, the Company will pay the Executive a base salary at the rate of FourHundred Ninety Thousand Dollars ($490,000) per year. Such amount shall be payable in accordance with the regular payroll practices of the Company for itsexecutives, as in effect from time to time, and subject to increase from time to time by the Board in its discretion. For avoidance of doubt, the parties agree thatExecutive’s base salary shall not decrease without his consent. The Executive shall have the opportunity to earn an annual target bonus measured againstperformance criteria to be determined reasonably and in good faith by the Board (or a committee thereof) in consultation with the Executive, and set at 60% ofthe Executive’s then current annual base salary (the  “Target Bonus”). Any bonus amount payable by the Company, if any, shall be paid no later than March 15 of the year following the year in which suchbonus is earned. (b) Participation in Employee Benefit Plans. The Executive will be entitled to participate in all Employee Benefit Plans from timeto time in effect for employees of the Company generally, except to the extent such plans are duplicative of benefits otherwise provided the Executive under thisAgreement (e.g., severance pay) or under any other agreement. The Executive’s participation will be subject to the terms of the applicable plan documents andgenerally applicable Company policies. The Company may alter, modify, add to or delete its Employee Benefit Plans at any time as it, in its sole judgment,determines to be appropriate, without recourse by the Executive. For purposes of this Agreement, “Employee Benefit Plan” shall have the meaning ascribed tosuch term in Section 3(3) of ERISA, as amended from time to time. (c) Vacation. The Executive will accrue three weeks paid vacation per year (or such greater amount as is generally made available tothe Company’s executive officers) in accordance with the Company’s policies from time to time in effect and receive paid holidays in accordance with theCompany holiday schedule. Vacation may be taken at such times and intervals as the Executive shall determine, subject to the business needs of theCompany, and otherwise shall be subject to the policies of the Company, as in effect from time to time. (d) Business Expenses. The Company will pay or reimburse the Executive for all reasonable business expenses incurred or paid bythe Executive in the performance of his duties and responsibilities for the Company, subject to any maximum annual limit and other restrictions on suchexpenses set by the Company and to such reasonable substantiation and documentation as it may specify from time to time. Any such reimbursement thatwould constitute nonqualified deferred compensation subject to Section 409A shall be subject to the following additional rules: (i) no reimbursement of anysuch expense shall affect the Executive’s right to reimbursement of any other such expense in any other taxable year; (ii) reimbursement of the expense shall bemade, if at all, not later than the end of the calendar year following the calendar year in which the expense was incurred; and (iii) the right to reimbursementshall not be subject to liquidation or exchange for any other benefit. 3. Confidential Information, Non-Competition and Proprietary Information. The Executive acknowledges that he has executed, andwill continue to comply with, the Company’s standard Employee Non-Solicitation, Non-Competition, Confidential Information and Inventions AssignmentAgreement. It is understood and agreed that breach by the Executive of the Employee Non-Solicitation, Non-Competition, Confidential Information andInventions Assignment Agreement shall constitute a material breach of this Agreement. 4. Termination of Employment. The Executive’s employment under this Agreement shall continue until terminated pursuant to thisSection 4. (a) The Company may terminate the Executive’s employment for “Cause” upon written notice to the Executive setting forth inreasonable detail the nature of the Cause. The following, as determined by the Board in good faith and using its reasonable judgment, shall constitute Causefor termination: (i) the Executive’s willful failure to perform, or gross  negligence in the performance of, the Executive’s material duties and responsibilities to the Company and its Affiliates which is not remedied within thirty (30)days of written notice thereof; (ii) material breach by the Executive of any material provision of this Agreement or any other agreement with the Company orany of its Affiliates which is not remedied within thirty (30) days of written notice thereof; (iii) fraud, embezzlement or other dishonesty with respect to theCompany and any of its Affiliates, taken as a whole, which, in the case of such other dishonesty, causes or could reasonably be expected to cause materialharm to the Company and any of its Affiliates, taken as a whole; or (iv) the Executive’s conviction of a felony. (b) The Company may terminate the Executive’s employment at any time other than for Cause upon one month’s written notice to theExecutive. (c) The Executive may terminate his employment hereunder for Good Reason by providing notice to the Company of the conditiongiving rise to the Good Reason no later than thirty (30) days following the occurrence of the condition, by giving the Company thirty (30) days to remedy thecondition and by terminating employment for Good Reason within thirty (30) days thereafter if the Company fails to remedy the condition. For purposes ofthis Agreement, “Good Reason” shall mean, without the Executive’s consent, the occurrence of any one or more of the following events: (i) material diminutionin the nature or scope of the Executive’s responsibilities, duties or authority, provided that, in the absence of a Change of Control, neither (x) the Company’sfailure to continue the Executive’s appointment or election as a director or officer of any of its Affiliates nor (y) any diminution in the nature or scope of theExecutive’s responsibilities, duties or authority that is reasonably related to a substantial diminution of the business of the Company as a whole that isreasonably projected to be sustained shall constitute “Good Reason;” (ii) a material reduction in the Executive’s base salary other than one temporary reductionof not more than 120 days and not in excess of 20% of the Executive’s base salary in connection with and in proportion to a general reduction of the basesalaries of the Company’s executive officers; (iii) failure of the Company to provide the Executive the salary or benefits in accordance with Section 2 hereofafter thirty (30) days’ notice during which the Company does not cure such failure; or (iv) relocation of the Executive’s office more than forty (40) miles fromthe location of the Company’s principal offices as of immediately prior to such relocation. (d) The Executive may terminate his employment with the Company other than for Good Reason at any time upon one month’s noticeto the Company. In the event of termination of the Executive’s employment in accordance with this Section 4(d), the Board may elect to waive the period ofnotice, or any portion thereof, and, if the Board so elects, the Company will pay the Executive his then current base salary for the period so waived. (e) This Agreement shall automatically terminate in the event of the Executive’s death during employment. The Company mayterminate the Executive’s employment, upon notice to the Executive, in the event the Executive becomes disabled during employment and, as a result, is unableto continue to perform substantially all of his material duties and responsibilities under this Agreement for one-hundred and fifty (150) days during any periodof three hundred and sixty-five (365) consecutive calendar days. If any question shall arise as to whether the Executive is disabled to the extent that theExecutive is unable to perform substantially all of his material duties and responsibilities for the Company and its Affiliates, the Executive shall, at theCompany’s request and expense, submit to a medical examination by a  physician selected by the Company to whom the Executive or the Executive’s guardian, if any, has no reasonable objection to determine whether the Executiveis so disabled and such determination shall for the purposes of this Agreement be conclusive of the issue. If such a question arises and the Executive fails tosubmit to the requested medical examination, the Company’s determination of the issue shall be binding on the Executive. For avoidance of all doubt, thisprovision is not intended to and does not affect the Executive’s rights under the Company’s long term and short term disability plans, nor his legal rightsunder state or federal law. 5. Severance Payments and Other Matters Related to Termination. (a) Termination pursuant to Section 4(b) or 4(c). (i) Except as provided in Section 5(c) below, in the event of termination of the Executive’s employment either by theCompany other than for Cause pursuant to Section 4(a) of this Agreement or by the Executive for Good Reason pursuant to Section 4(c) ofthis Agreement, (a) all unvested options, restricted stock, and restricted stock units granted prior to the date this Agreement is fully executedwhich, by their terms, vest only based on the passage of time (disregarding any acceleration of the vesting of such options, restricted stockor restricted stock units based on individual or Company performance) that are outstanding immediately prior to the date of terminationand, but for the termination of the Executive’s employment, would have vested during the twelve (12) month period immediately followingthe date of termination, shall, notwithstanding the terms of the stock or equity compensation plans and any applicable award agreements,remain outstanding and eligible to vest until the Payment Commencement Date and, subject to Section 5(a)(iii), automatically become fullyvested as of the Payment Commencement Date with respect to such portion of each such award that would have vested during such twelve(12) month period and (b) the Company shall pay the Executive’s then-current annual base salary for a period of twelve (12) months inaccordance with the Company’s payroll practice then in effect, beginning on the Payment Commencement Date. (ii) Except as provided in Section 5(c) below, in the event of termination of the Executive’s employment either by theCompany other than for Cause pursuant to Section 4(a) of this Agreement or by the Executive for Good Reason pursuant to Section 4(c) ofthis Agreement, if the Executive is participating in the Company’s group health plan and/or dental plan at the time the Executive’semployment terminates, and the Executive exercises his right to continue participation in those plans under the federal law known asCOBRA, or any successor law, the Company will pay or, at its option, reimburse the Executive, for the full premium cost of thatparticipation for twelve (12) months following the date on which the Executive’s employment with the Company terminates or, if earlier,until the date the Executive becomes eligible to enroll in the health (or, if applicable, dental) plan of a new employer, payable in accordancewith regular payroll practices for benefits beginning on the Payment Commencement Date. In the event that the Company elects toreimburse the  Executive for the full premium cost of that participation, it shall gross up such payment’s tax consequences, if any, to the Executive.Notwithstanding the foregoing, if the payment or reimbursement by the Company of the premium costs, including payment of any grossup for any tax consequences, described in the preceding sentence will subject or expose the Company to taxes or penalties, the Executive andthe Company agree to make good faith efforts to renegotiate the provisions of this Section 5(a)(ii) and to enter into a substitute arrangementpursuant to which the Company may not be subjected or exposed to taxes or penalties but which will not adversely affect the full economicvalue to the Executive of the benefits promised by this provision. For avoidance of any doubt, nothing in this provision will require theExecutive to accept any renegotiated agreement that would disadvantage him economically or require that he accept a lesser quality of healthcare coverage for himself and his family. The Company will also pay the Executive on the date of termination any base salary earned butnot paid through the date of termination and pay for any vacation time accrued but not used to that date. In addition, the Company will paythe Executive any bonus which has been awarded to the Executive, but not yet paid on the date of termination of his employment, payablein a lump sum at such time when bonuses are paid to executives of the Company generally in accordance with the timing rules ofSection 2(a). (iii) Any obligation of the Company to provide the Executive severance payments or other benefits under this Section 5(a) orSection 5(c) is conditioned on the Executive’s signing an effective and reasonable release of claims (the “Employee Separation Release”) andthe termination of any period in which the Executive may revoke such Employee Release (without the Executive revoking) within 60 daysfollowing the termination of the Executive’s employment (“Release Effective Date”). Such Employee Separation Release shall not (A) applyto (i) claims for indemnification in the Executive’s capacity as an officer or director of the Company under the Company’s Certificate ofIncorporation, By-laws or agreement, if any, providing for director or officer indemnification, (ii) rights to receive insurance paymentsunder any policy maintained by the Company and (iii) rights to receive retirement benefits that are accrued and fully vested at the time ofthe Executive’s termination and rights under such plans protected by ERISA, nor shall such Employee Separation Release (B) prevent theExecutive from defending himself against a released party in the event that any such released party initiates legal action against himpertaining to events or circumstances arising prior to the effective date of the Employee Release other than a legal action that solely allegesclaims involving or arising out of a violation of law that would constitute a felony or other crime involving moral turpitude by the Executivethat amounts to criminal misconduct on the Executive’s part. In such a circumstance described in (B), the Employee Separation Releaseshall be null and void as against that party alone, and the Executive may bring any counter-claim he may have against the party thatinitiated the litigation. (iv) The severance payments shall commence on the first payroll period following the Release Effective Date, but the amountof the first  installment shall reflect the period retroactive to the date of termination (the “Payment Commencement Date”), and each subsequentinstallment payment shall be a series of separate, substantially equal payments. (v) Notwithstanding the foregoing, if the 60th day following the date of termination occurs in the calendar year followingthe calendar year of the termination, then the Payment Commencement Date shall be no earlier than January 1 of such subsequent calendaryear. The Executive agrees to provide the Company prompt notice of the Executive’s eligibility to participate in the health plan and, ifapplicable, dental plan of any employer. (b) Termination other than pursuant to Section 4(b) or 4(c). In the event of any termination of the Executive’s employment, otherthan a termination by the Company pursuant to Section 4(b) of this Agreement or a termination by the Executive for Good Reason pursuant to Section 4(c) ofthis Agreement, the Company will pay the Executive any base salary earned but not paid through the date of termination and pay for any vacation timeaccrued but not used to that date. In addition, the Company will pay the Executive any bonus which has been awarded to the Executive, but not yet paid, atsuch time when bonuses are paid to executives of the Company generally in accordance with the timing rules of Section 2(a). The Company shall have noother obligation to the Executive under this Agreement. (c) Upon a Change of Control. (i) Subject to the provisions of Section 5(a)(iii), if, within ninety (90) days prior to a Change of Control or within one yearfollowing a Change of Control (as defined in Section 6 hereof), the Company or any successor thereto terminates the Executive’semployment other than for Cause, or the Executive terminates his employment for Good Reason, then, in lieu of any payments to theExecutive or on the Executive’s behalf under Section 5(a) hereof, (a) all of the Executive’s then remaining unvested options, restricted stock,and restricted stock units which, by their terms, vest only based on the passage of time (disregarding any acceleration of the vesting ofsuch options, restricted stock or restricted stock units based on individual or Company performance) that are outstanding immediatelyprior to the date of termination shall, notwithstanding the terms of the stock or equity compensation plans and any applicable awardagreements, remain outstanding and eligible to vest until the Payment Commencement Date and, subject to Section 5(a)(iii), automaticallybecome fully vested as of the Payment Commencement Date; and (b) the Company shall pay, on the Payment Commencement Date, a lumpsum equal to two (2) times (x) the Executive’s then-current annual base salary plus (y) the Executive’s Target Bonus; provided, however,that if such termination occurs prior to a Change of Control, such severance payments shall be made at the time and in the manner set forthin Section 5(a)(i) during the period beginning on the date of termination through the date of the Change of Control with any severanceremaining to be paid under this Section 5(c)(i) payable in a lump sum on the closing date of the Change of Control (or, if later, the PaymentCommencement Date); and,  (ii) If the Executive is participating in the Company’s group health plan and/or dental plan at the time the Executive’semployment terminates (and such termination is as described in 5(c)(i) above), and the Executive exercises his right to continueparticipation in those plans under the federal law known as COBRA, or any successor law, then, in lieu of any payments to the Executiveor on the Executive’s behalf under Section 5(a) hereof, the Company will pay or, at its option, reimburse the Executive, for the fullpremium cost of that participation for twenty-four (24) months (or such shorter period during which COBRA participation is permitted bylaw) following the date on which the Executive’s employment with the Company terminates or, if earlier, until the date the Executivebecomes eligible to enroll in the health (or, if applicable, dental) plan of a new employer, with such amount payable on a pro-rata basis inaccordance with the Company’s regular payroll practices for benefits beginning on the Payment Commencement Date. In the event that theCompany elects to reimburse the Executive for the full premium cost of that participation, it shall gross up such payment’s taxconsequences, if any, to the Executive. Notwithstanding the foregoing, if the payment or reimbursement by the Company of the premiumcosts, including payment of any gross up for any tax consequences, described in the preceding sentence will subject or expose theCompany to taxes or penalties, the Executive and the Company agree to make good faith efforts to renegotiate the provisions of thisSection 5(a)(ii) and to enter into a substitute arrangement pursuant to which the Company may not be subjected or exposed to taxes orpenalties but which will not adversely affect the full economic value to the Executive of the benefits promised by this provision. Foravoidance of any doubt, nothing in this provision will require the Executive to accept any renegotiated agreement that would disadvantagehim economically or require that he accept a lesser quality of health care coverage for himself and his family. The Company will also paythe Executive on the date of termination any base salary earned but not paid through the date of termination and pay for any vacation timeaccrued but not used to that date. In addition, the Company will pay the Executive any bonus which has been awarded to the Executive, butnot yet paid on the date of termination of his employment, payable in a lump sum at such time when bonuses are paid to executives of theCompany generally in accordance with the timing rules of Section 2(a). (d) Golden Parachute Tax Provisions. (i) The Company and the Executive agree that in the event the payment or provision of any or all amounts or benefitsunder this Agreement (such amounts and benefits, the “Benefits”), together with any other payments or benefits payable or provided to orfor the benefit of the Executive in connection with a Change of Control (together with the Benefits, the “Total Benefits”) would, withoutgiving effect to this Section 5(d), be subject to the excise tax imposed under Section 4999 of the Code , then, subject toSection 5(d)(ii) below, Company shall pay to the Executive a Gross-Up Payment. Gross-Up Payment means an amount (A) such that thenet amount retained by Executive, after payment of the excise tax and any federal, state, and local income or employment tax and any  interest or penalties imposed, in each case, with respect to such excise tax, is equal to the Total Benefits (such amount (A), the “Total GUAmount”), multipied by a fraction (B) the numerator of which is the Total GU Amount arising from payments or benefits payable orprovided to or for the benefit of Executive in connection with a Change in Control to the extent such payments or benefits are payable orprovided in connection with options and other equity and equity-related awards first granted to the Executive prior to the execution of thisAgreement, and the denominator of which is the Total GU Amount arising from payments or benefits payable or provided to or for thebenefit of Executive in connection with a Change in Control to the extent such payments or benefits are payable or provided in connectionwith all options and other equity and equity-related awards granted to the Executive. Any such Gross-Up Payment shall be paid to theExecutive within 60 days following the date that the Excise Tax is due. (ii) If reduction of the unpaid Benefits would result in greater Net After-Tax Receipts than would payment of the unpaidBenefits, the unpaid Benefits shall be reduced to the amount (the “Optimal Benefit”) that produces the greatest amount of Net After-TaxReceipts. If the greatest amount of Net After-Tax Receipts may result from two or more different Optimal Benefit amounts, the unpaidBenefits shall be reduced to the lesser of such Optimal Benefit amounts. “Net After-Tax Receipts” means the amount of the Total Benefitsplus the amount of the Gross-Up Payment, if any, net of all federal, state and local income and excise taxes imposed on the Total Benefitsand the Gross-Up Payment. Any such reduction shall be made in the manner that results in the greatest economic benefit for the Executive. If more than one method of reduction will result in the same economic benefit, the method of reduction shall be determined by the Company. (iii) All calculations necessary to make any determinations under this Section 5(d) shall be performed by the accountingfirm engaged (as of the day prior to the effective date of the Change in Control) by the Company for general tax compliance purposes,unless otherwise agreed by the Company and the Executive. The Company shall bear all expenses incurred in connection with suchcalculations. (iv) The intent of this Section 5(d) is for the Executive to receive either the full amount of the unpaid Benefits plus theGross-Up Payment described in Section 5(d)(i) or a reduction in the unpaid Benefits as described in Section 5(d)(ii), whichever results inthe greater economic benefit for the Executive. (e) Except for any right the Executive may have under applicable law to continue participation in the Company’s group health anddental plans under COBRA, or any successor law, benefits shall terminate in accordance with the terms of the applicable benefit plans based on the date oftermination of the Executive’s employment, without regard to any continuation of base salary or other payment to the Executive following termination. (f) Provisions of this Agreement shall survive any termination if so provided in this Agreement or if necessary or desirable toaccomplish the purposes of other surviving provisions, including without limitation the Executive’s obligations under Section 3 of this Agreement and under the Employee Non-Solicitation, Non-Competition, Confidential Information and Inventions Assignment Agreement. In the event that the Company’s Board of Directors determines in good faiththat the Executive has not continued to perform in all material respects under Section 3 hereof, under the Employee Non-Solicitation, Non-Competition,Confidential Information and Inventions Assignment Agreement and under any subsequent agreement between the Executive and the Company or any of itsAffiliates relating to confidentiality, non-competition, proprietary information or the like, unless (1) such failure is determined to amount to willful misconductor gross negligence or (2) such failure is not capable of being cured, the Company will provide the Executive reasonable notice of its determination and provideExecutive with a 30 day period to cure any perceived deficits in his performance. If the Board determines in good faith at the end of such 30-day period, ifapplicable, or immediately upon any such initial determination in the case of a determination described in clause (1) or (2) of the preceding sentence, that theExecutive is not performing under any of such agreements in all material respects, the Company may cease making payments under Section 5 of thisAgreement. Nothing in this provision limits the Executive’s ability to challenge that determination or to assert that it is in breach of the obligations undertakenby the Company in Section 5. In the event that the Company ceases to make payments to the Executive or on the Executive’s behalf under Section 5 of thisAgreement pursuant to this provision and the Executive challenges such action on the part of the Company before a court of competent jurisdiction, theCompany will pay the Executive’s reasonable attorney’s fees in the event that the Executive prevails in a decision that has become unappealable. (g) In the event of any conflict between the vesting provisions applicable to equity-based awards set forth in this Section 5 and theterms of the stock or equity compensation plans and any applicable award agreements, the provisions of this Section 5 shall govern except as may beexpressly provided in an award agreement making specific reference to this Section 5(g). 6. Definitions. For purposes of this agreement; the following definitions apply: “Affiliates” means all persons and entities directly or indirectly controlling, controlled by or under common control with the Company,where control may be by management authority, equity interest or otherwise. “Change of Control” shall mean (i) the acquisition of beneficial ownership (as defined in Rule 13d-3 under the Exchange Act) directly orindirectly by any “person” (as such term is used in Sections 13(d) and 14(d) of the Exchange Act), of securities of the Company representing a majority ormore of the combined voting power of the Company’s then outstanding securities, other than an acquisition of securities for investment purposes pursuant to abona fide financing of the Company; (ii) a merger or consolidation of the Company with any other corporation in which the holders of the voting securities ofthe Company prior to the merger or consolidation do not own more than 50% of the total voting securities of the surviving corporation; or (iii) the sale ordisposition by the Company of all or substantially all of the Company’s assets other than a sale or disposition of assets to an Affiliate of the Company or aholder of securities of the Company; notwithstanding the foregoing, no transaction or series of transactions shall constitute a Change of Control unless suchtransaction or series of transactions  constitutes a “change in control event” within the meaning of Treasury Regulation Section 1.409A-3(i)(5)(i). “Person” means an individual, a corporation, an association, a partnership, an estate, a trust and any other entity or organization, otherthan the Company or any of its Affiliates. 7. Conflicting Agreements. The Executive hereby represents and warrants that his signing of this Agreement and the performance of hisobligations under it will not breach or be in conflict with any other agreement to which the Executive is a party or is bound with any third party, and that theExecutive is not now subject to any covenants against competition or similar covenants or any court order that could affect the performance of the Executive’sobligations under this Agreement. The Executive agrees that he will not disclose to or use on behalf of the Company any proprietary information of a thirdparty without that party’s consent. 8. Withholding; Other Tax Matters. Anything to the contrary notwithstanding, (a) all payments required to be made by the Companyhereunder to Executive shall be subject to the withholding of such amounts, if any, relating to tax and other payroll deductions as the Company mayreasonably determine it should withhold pursuant to any applicable law or regulation, and (b) all severance payments and benefits payable pursuant toSections 5(a) and 5(c) hereof shall be subject to the terms and conditions set forth on Exhibit A attached hereto 9. Assignment. Neither the Executive nor the Company may make any assignment of this Agreement or any interest in it, by operation oflaw or otherwise, without the prior written consent of the other; provided, however, that the Company may assign its rights and obligations under thisAgreement without the Executive’s consent to one of its Affiliates or to any Person with whom the Company shall hereafter affect a reorganization, consolidatewith, or merge into or to whom it transfers all or substantially all of its properties or assets. This Agreement shall inure to the benefit of and be binding uponthe Executive and the Company, and each of our respective successors, executors, administrators, heirs and permitted assigns. 10. Severability. If any portion or provision of this Agreement shall to any extent be declared illegal or unenforceable by a court of competentjurisdiction, then the remainder of this Agreement, or the application of such portion or provision in circumstances other than those as to which it is sodeclared illegal or unenforceable, shall not be affected thereby, and each portion and provision of this Agreement shall be valid and enforceable to the fullestextent permitted by law. 11. Miscellaneous. This Agreement, together with the Employee Non-Solicitation, Non-Competition, Confidential Information and InventionsAssignment Agreement, sets forth the entire agreement between the Executive and the Company and replaces all prior communications, agreements andunderstandings, written or oral, with respect to the terms and conditions of the Executive’s employment. This Agreement may not be modified or amended,and no breach shall be deemed to be waived, unless agreed to in writing by the Executive and an expressly authorized representative of the Board. Theheadings and captions in this Agreement are for convenience only and in no way define or describe the scope or content of any provision of this Agreement.This Agreement may be executed in two or more counterparts, each of which  shall be an original and all of which together shall constitute one and the same instrument. This is a Massachusetts contract and shall be governed andconstrued in accordance with the laws of the Commonwealth of Massachusetts, without regard to the conflict-of-laws principles thereof. 12. Notices. Any notices provided for in this Agreement shall be in writing and shall be effective when delivered in person, consigned to areputable national courier service for overnight delivery or deposited in the United States mail, postage prepaid, and addressed to the Executive at theExecutive’s last known address on the books of the Company or, in the case of the Company, to it by notice to the Chairman of the Board of Directors, c/oVerastem, Inc. at its principal place of business, or to such other addressees) as either party may specify by notice to the other actually received. [Remainder of page intentionally left blank.]  IN WITNESS WHEREOF, this Agreement has been executed as a sealed instrument by the Company, by its duly authorized representative, and bythe Executive, as of the date first stated above. THE EXECUTIVETHE COMPANY /s/ Robert Forrester/s/ Stephen SherwinRobert ForresterStephen SherwinChairman of the Compensation Committee /s/ Christoph WestphalChristoph WestphalExecutive Chairman  Exhibit A Payments Subject to Section 409A 1. Subject to this Exhibit A, any severance payments that may be due under the Agreement shall begin only upon the date of the Executive’s“separation from service” (determined as set forth below) which occurs on or after the termination of Executive’s employment. The following rules shall applywith respect to distribution of the severance payments, if any, to be provided to Executive under the Agreement, as applicable: (a) It is intended that each installment of the severance payments under the Agreement provided under shall be treated as a separate“payment” for purposes of Section 409A. Neither the Company nor Executive shall have the right to accelerate or defer the delivery of any such paymentsexcept to the extent specifically permitted or required by Section 409A. (b) If, as of the date of Executive’s “separation from service” from the Company, Executive is not a “specified employee” (asdetermined by the Board within the meaning of Section 409A), then each installment of the severance payments shall be made on the dates and terms set forthin the Agreement. (c) If, as of the date of Executive’s “separation from service” from the Company, Executive is a “specified employee” (as determinedby the Board within the meaning of Section 409A), then: (i) Each installment of the severance payments due under the Agreement that, in accordance with the dates and terms setforth herein, will in all circumstances, regardless of when Executive’s separation from service occurs, be paid within the short-term deferralperiod (as defined under Section 409A) shall be treated as a short-term deferral within the meaning of Treasury Regulation Section 1.409A-1(b)(4) to the maximum extent permissible under Section 409A and shall be paid on the dates and terms set forth in the Agreement; and (ii) Each installment of the severance payments due under the Agreement that is not described in this Exhibit A,Section 1(c)(i) and that would, absent this subsection, be paid within the six-month period following Executive’s “separation from service”from the Company shall not be paid until the date that is six months and one day after such separation from service (or, if earlier,Executive’s death), with any such installments that are required to be delayed being accumulated during the six-month period and paid in alump sum on the date that is six months and one day following Executive’s separation from service and any subsequent installments, ifany, being paid in accordance with the dates and terms set forth herein; provided, however, that the preceding provisions of this sentenceshall not apply to any installment of payments if and to the maximum extent that that such installment is deemed to be paid under aseparation pay plan that does not provide for a deferral of compensation by reason of the application of Treasury Regulation 1.409A-1(b)(9)(iii) (relating to separation pay upon an involuntary separation from service). Any installments  that qualify for the exception under Treasury Regulation Section 1.409A-1(b)(9)(iii) must be paid no later than the last day of Executive’ssecond taxable year following the taxable year in which the separation from service occurs. 2. The determination of whether and when Executive’s separation from service from the Company has occurred shall be made and in amanner consistent with, and based on the presumptions set forth in, Treasury Regulation Section 1.409A-1(h). Solely for purposes of this Exhibit A,Section 2, “Company” shall include all persons with whom the Company would be considered a single employer under Section 414(b) and 414(c) of the Code. 3. The Company makes no representation or warranty and shall have no liability to Executive or to any other person if any of the provisionsof the Agreement (including this Exhibit) are determined to constitute deferred compensation subject to Section 409A but that do not satisfy an exemption from,or the conditions of, that section. Exhibit 21.1 List of Registrant’s Subsidiaries Verastem Securities Company, incorporated in Massachusetts, a wholly owned subsidiary. QuickLinks -- Click here to rapidly navigate through this documentExhibit 23.1 Consent of Independent Registered Public Accounting Firm We consent to the incorporation by reference in the Registration Statements (Form S-8 Nos. 333-180475 and 333-190578 and Form S-3 No. 333-192968) of Verastem, Inc. and in the related Prospectus of our report dated March 6, 2014, with respect to the consolidated financial statements ofVerastem, Inc., included in this Annual Report (Form 10-K) for the year ended December 31, 2013./s/ Ernst & Young LLPBoston, MassachusettsMarch 6, 2014QuickLinksExhibit 23.1Consent of Independent Registered Public Accounting FirmQuickLinks -- Click here to rapidly navigate through this documentExhibit 31.1 CERTIFICATIONS I, Robert Forrester certify that: 1. I have reviewed this Annual Report on Form 10-K of Verastem, Inc.; 2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to makethe statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by thisreport; 3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respectsthe financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; 4. The registrant's other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as definedin Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have: a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under oursupervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by otherswithin those entities, particularly during the period in which this report is being prepared; b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed underour supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements forexternal purposes in accordance with generally accepted accounting principles; c) Evaluated the effectiveness of the registrant's disclosure controls and procedures and presented in this report our conclusions about theeffectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and d) Disclosed in this report any change in the registrant's internal control over financial reporting that occurred during the registrant's mostrecent fiscal quarter (the registrant's fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely tomaterially affect, the registrant's internal control over financial reporting; and 5. The registrant's other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting,to the registrant's auditors and the audit committee of the registrant's board of directors (or persons performing the equivalent functions): a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which arereasonably likely to adversely affect the registrant's ability to record, process, summarize and report financial information; and b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant'sinternal control over financial reporting.1 /s/ ROBERT FORRESTERRobert ForresterChief Executive OfficerDate: March 6, 2014 QuickLinksExhibit 31.1CERTIFICATIONSQuickLinks -- Click here to rapidly navigate through this documentExhibit 31.2 CERTIFICATIONS I, John B. Green, CPA, certify that: 1. I have reviewed this Annual Report on Form 10-K of Verastem, Inc.; 2. Based on my knowledge, this report does not contain any untrue statement of a material fact or omit to state a material fact necessary to makethe statements made, in light of the circumstances under which such statements were made, not misleading with respect to the period covered by thisreport; 3. Based on my knowledge, the financial statements, and other financial information included in this report, fairly present in all material respectsthe financial condition, results of operations and cash flows of the registrant as of, and for, the periods presented in this report; 4. The registrant's other certifying officer and I are responsible for establishing and maintaining disclosure controls and procedures (as definedin Exchange Act Rules 13a-15(e) and 15d-15(e)) and internal control over financial reporting (as defined in Exchange Act Rules 13a-15(f) and 15d-15(f)) for the registrant and have: a) Designed such disclosure controls and procedures, or caused such disclosure controls and procedures to be designed under oursupervision, to ensure that material information relating to the registrant, including its consolidated subsidiaries, is made known to us by otherswithin those entities, particularly during the period in which this report is being prepared; b) Designed such internal control over financial reporting, or caused such internal control over financial reporting to be designed underour supervision, to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements forexternal purposes in accordance with generally accepted accounting principles; c) Evaluated the effectiveness of the registrant's disclosure controls and procedures and presented in this report our conclusions about theeffectiveness of the disclosure controls and procedures, as of the end of the period covered by this report based on such evaluation; and d) Disclosed in this report any change in the registrant's internal control over financial reporting that occurred during the registrant's mostrecent fiscal quarter (the registrant's fourth fiscal quarter in the case of an annual report) that has materially affected, or is reasonably likely tomaterially affect, the registrant's internal control over financial reporting; and 5. The registrant's other certifying officer and I have disclosed, based on our most recent evaluation of internal control over financial reporting,to the registrant's auditors and the audit committee of the registrant's board of directors (or persons performing the equivalent functions): a) All significant deficiencies and material weaknesses in the design or operation of internal control over financial reporting which arereasonably likely to adversely affect the registrant's ability to record, process, summarize and report financial information; and b) Any fraud, whether or not material, that involves management or other employees who have a significant role in the registrant'sinternal control over financial reporting.Date: March 6, 20141 /s/ JOHN B. GREEN, CPAJohn B. Green, CPAChief Financial OfficerQuickLinksExhibit 31.2CERTIFICATIONSQuickLinks -- Click here to rapidly navigate through this documentExhibit 32.1 CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350,AS ADOPTED PURSUANT TOSECTION 906 OF THE SARBANES-OXLEY ACT OF 2002 In connection with the Annual Report on Form 10-K of Verastem, Inc. (the "Company") for the period ended December 31, 2013 as filed with theSecurities and Exchange Commission (the "SEC") on the date hereof (the "Report"), the undersigned, Robert Forrester, Chief Executive Officer of theCompany, hereby certify, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that to myknowledge: (1) the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934 as amended; and (2) the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of theCompany. A signed original of this written statement required by Section 906 has been provided to the Company and will be retained by the Company andfurnished to the SEC or its staff upon request.1 /s/ ROBERT FORRESTERRobert ForresterChief Executive OfficerDate: March 6, 2014 QuickLinksExhibit 32.1CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO SECTION 906 OF THE SARBANES-OXLEYACT OF 2002QuickLinks -- Click here to rapidly navigate through this documentExhibit 32.2 CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350,AS ADOPTED PURSUANT TOSECTION 906 OF THE SARBANES-OXLEY ACT OF 2002 In connection with the Annual Report on Form 10-K of Verastem, Inc. (the "Company") for the period ended December 31, 2013 as filed with theSecurities and Exchange Commission (the "SEC") on the date hereof (the "Report"), the undersigned, John B. Green, CPA, Chief Financial Officer ofthe Company, hereby certify, pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002, that to myknowledge: (1) the Report fully complies with the requirements of Section 13(a) or 15(d) of the Securities Exchange Act of 1934 as amended; and (2) the information contained in the Report fairly presents, in all material respects, the financial condition and results of operations of theCompany. A signed original of this written statement required by Section 906 has been provided to the Company and will be retained by the Company andfurnished to the SEC or its staff upon request.1 /s/ JOHN B. GREEN, CPAJohn B. Green, CPAChief Financial OfficerDate: March 6, 2014 QuickLinksExhibit 32.2CERTIFICATION PURSUANT TO 18 U.S.C. SECTION 1350, AS ADOPTED PURSUANT TO SECTION 906 OF THE SARBANES-OXLEYACT OF 2002

Continue reading text version or see original annual report in PDF format above