BREAKING NEWS: New Clinical Trial Opening

New press release daily newspaper headlinePRESS RELEASE

Contact Verastem, Inc.
Brian Sullivan, 617-252-9314

CAMBRIDGE, MA – Sep. 10, 2013 – Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, today announced the initiation of COMMAND (Control Of Mesothelioma with MAiNtenance Defactinib). COMMAND is a registration-directed trial of lead cancer stem cell inhibitor defactinib (VS-6063) in patients with malignant pleural mesothelioma, a highly aggressive form of lung cancer with a high percentage of cancer stem cells.

COMMAND is a registration-directed, double-blind, placebo-controlled trial with Progression Free Survival (PFS) and Overall Survival (OS) as the primary endpoints. The study incorporates the opportunity to enrich for patients with tumors low in the biomarker, merlin. Preclinical and early clinical research has demonstrated that low merlin levels are predictive of increased effectiveness of FAK inhibitors such as defactinib. The COMMAND study will stratify patients to evaluate the effect of defactinib in both the overall patient population and the subgroup that are merlin low.

“FAK inhibition holds significant potential in malignant pleural mesothelioma, a cancer whose aggressiveness and treatment resistance proves all too often to be devastating for patients,” said Dr. Joanna Horobin, Verastem Chief Medical Officer. “Development of a drug that preferentially kills cancer stem cells is a promising approach, as many standard-of-care treatments have been shown to either have no effect on, or actually enrich the population of, these chemoresistant cells.”

COMMAND is expected to enroll approximately 350-400 patients at clinical sites in 11 countries, including the US, UK, Australia, Canada, South Africa, New Zealand and countries in mainland Europe. Eligible patients who had a partial response or stable disease following standard first-line therapy with platinum/pemetrexed will be stratified to merlin low or high and then randomized 1:1 to receive either placebo or 400 mg of defactinib twice daily.

“The incidence of mesothelioma, among the most aggressive and lethal cancers, is increasing worldwide, underscoring an urgent need to expand the very limited treatment options for patients fighting this disease,” said Professor Dean Fennell, Chair of Thoracic Medical Oncology at the University of Leicester, incoming President of the International Mesothelioma Interest Group (iMig) and Coordinating Investigator for COMMAND in the UK. “This well-designed study is expected to deliver a one-two punch to the tumor by treating with defactinib following first-line therapy that achieves initial disease control, but has not eliminated the cancer stem cells nor historically stopped disease progression, in an attempt to prolong clinical response. There is significant enthusiasm within the mesothelioma community for the COMMAND study.”

Verastem was recently granted orphan drug designation for defactinib in mesothelioma by the U.S. Food and Drug Administration’s Office of Orphan Drug Products.  The Company received a similar designation from the European Commission in June of this year. Adding to an intellectual property portfolio including composition of matter for defactinib, these designations provide eligibility for seven- and ten-year periods of market exclusivity, respectively, after product approval, as well as other development incentives.

“Timely initiation of this study marks yet another important milestone in our development of defactinib, and highlights the diligent execution of our clinical strategy by Verastem’s research and development team,” said Robert Forrester, Verastem President and Chief Executive Officer. “We will provide an update on the progress of this study in our year-end update in the first quarter of 2014.”

In addition to defactinib in mesothelioma, Verastem recently announced the completion of the Phase 1 stage and initial data from an ongoing Phase 1/1b study of defactinib in combination with weekly paclitaxel for patients with ovarian cancer. Verastem expects to initiate additional studies of defactinib including a Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer and a Phase 1 study in Japan during the third quarter of 2013. The Company also announced the recent initiation of a Phase 1 study of FAK inhibitor VS-4718 in patients with advanced cancer, and that it expects to enter the dual mTORC1/2 and PI3K inhibitor VS-5584 into the clinic later this year.

About Malignant Pleural Mesothelioma

Malignant pleural mesothelioma is an aggressive form of cancer that occurs in the mesothelium, the thin layer of tissue that covers the lungs. Mesothelioma is associated with exposure to asbestos in most cases. According to the World Health Organization, a total of 59,000 deaths occur worldwide each year due to mesothelioma. Most mesotheliomas begin as one or more nodules that progressively grow to form a solid coating of tumor surrounding the lung leading to eventual suffocation and death. A high percentage of mesotheliomas contain cancer stem cells which are generally resistant to the currently available treatment options for advanced mesothelioma.

About Defactinib

Defactinib (VS-6063) is an oral compound designed to target cancer stem cells through the potent inhibition of focal adhesion kinase (FAK). Cancer stem cells are an underlying cause of tumor resistance to chemotherapy, recurrence and ultimate disease progression. Research by Robert Weinberg, Ph.D., scientific cofounder and chair of Verastem’s Scientific Advisory Board, and Verastem has demonstrated that the FAK pathway is critical for the growth and survival of cancer stem cells.  Defactinib is currently being studied in a registration-directed COMMAND trial in mesothelioma and a Phase 1/1b study in ovarian cancer. A Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer and a Phase 1 study in Japan are expected to begin in the third quarter of 2013.  Defactinib has been granted orphan drug designation in the U.S. and E.U. for use in mesothelioma.

About Verastem, Inc.

Verastem, Inc. (NASDAQ: VSTM) is discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells. Cancer stem cells are an underlying cause of tumor recurrence and metastasis. Verastem is developing small molecule inhibitors of signaling pathways that are critical to cancer stem cell survival and proliferation: FAK, PI3K/mTOR and Wnt. For more information, please visit

Forward-looking statements:

This press release includes forward-looking statements about the Company’s strategy, future plans and prospects, including statements regarding the development of the Company’s compounds, including VS-6063, VS-4718 and VS-5584, and the Company’s FAK, PI3K/mTOR and diagnostic programs generally, the timeline for clinical development and regulatory approval of the Company’s compounds, the expected timing for the reporting of data from ongoing trials, the structure of the Company’s planned or pending clinical trials and estimates of the Company’s ability to fund operations. The words “anticipate,” “appear,” “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. Each forward-looking statement is subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement. Applicable risks and uncertainties include the risks that the preclinical testing of the Company’s compounds and preliminary data from clinical trials may not be predictive of the results or success of later clinical trials, that data may not be available when we expect it to be that the Company will be unable to successfully complete the clinical development of its compounds, including VS-6063, VS-4718 and VS-5584, that the development of the Company’s compounds will take longer or cost more than planned, and that the Company’s compounds will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2012 and in any subsequent SEC filings. The forward-looking statements contained in this presentation reflect the Company’s current views with respect to future events, and the Company does not undertake and specifically disclaims any obligation to update any forward-looking statements.


The Anatomy of Clinical Trials for Mesothelioma

Recently, we had the honor to interview Melissa Culligan, RN, BSN, MS, a thoracic surgery nurse with specialized experience designing clinical trials. To listen press PLAY in the above podcast box (it may take a minute for the podcast to load).

In a cancer like mesothelioma, for which there is only one FDA-approved treatment, clinical trials play an integral role in many patients’ treatment regimens. For this reason, we wanted to provide patients with a solid overview of clinical trials, their design, and implementation.

Melissa Culligan with Dr. Friedberg, Karen Mudrick, and Mary Hesdorffer at Meso Foundation's 2013 Symposium.

Melissa Culligan with Dr. Friedberg, Karen Mudrick, and Mary Hesdorffer at Meso Foundation’s 2013 Symposium.

Melissa Culligan, RN, BSN, MS has worked as a thoracic surgery nurse for over 25 years and has worked in the field of thoracic oncology research for the past 15 years. She currently works in the Division of Thoracic Surgery at Penn-Presbyterian Medical Center and is the Director of Clinical Services for the Penn Mesothelioma and Pleural Program. She has a special interest in the areas of mesothelioma and advanced stage lung cancer and has recently graduated from Drexel University’s School of Medicine’s Master’s Program in Clinical Research Organization and Management.

Are you interested in learning more about clinical trials for mesothelioma? Do you have questions about mesothelioma clinical trials? Talk to our mesothelioma specialists through our Ask the Expert feature now.

Using the Measles Virus to Treat Mesothelioma

dnaLast night, Dr. Tobias Peikert of the Mayo Clinic joined us on a conference call, as part of the Meso Foundation’s Meet the Experts series, to discuss an exciting new clinical trial. The trial, which uses the measles virus to attack the mesothelioma cells, is currently enrolling pleural mesothelioma patients regardless of whether they’ve undergone prior therapy. This is a Phase 1 trial, meaning that the goal of the trial is to determine the level of toxicity of the agent and to establish the optimal dose.

According to Dr. Peikert, the measles virus is particularly interesting in the treatment of cancers. Historically, the notion of using this virus came to scientists mostly by accident. They noticed that when people with cancer became ill with measles, their cancer nearly or all disappeared. As a result, the idea has already been used for some time with treatment of lymphoma and other hematologic cancers.

This trial has now been open for six months and is administered in Rochester, Minnesota.

The Mesothelioma Applied Research Foundation conducts Meet the Experts conference calls every couple of months. To receive notifications of these calls, please sign up for news from the Meso Foundation.

The full podcast of the interview with Dr. Peikert is available on Meso Foundation’s website.

Meso Foundation Clinical Trial Consortium for Faster Advancement of Mesothelioma Research

by Lee M. Krug, MD

At the International Mesothelioma Interest Group Meeting in Boston, a group of investigators convened at a Clinical Trials Design Workshop to discuss the challenges of conducting trials in this disease. The rarity of mesothelioma poses various issues with regards to proper study design, accrual, and interest by pharmaceutical companies. One theme that emerged from that workshop was the need to establish a clinical trials consortium. This concept has been quite successful with other rare cancers such as pediatric tumors and sarcoma. I propose that the Meso Foundation would provide the optimal mechanism to initiate such a consortium for mesothelioma trials.

The Meso Foundation Trials Consortium (MFTC) would offer the following advantages:

  • It would provide scientific input to ensure that the trials are scientifically sound. The investigators involved in the Consortium best understand the biologic underpinnings of the disease. They would have important insight into the optimal design of the trials. Trials could be prioritized based on scientific merit and promise. This would ensure that valuable resources would not be wasted on trials unlikely to be successful.
  • It would establish an international network of participating institutions with the highest volume of mesothelioma patients. This would have drastic effects on speeding accrual to complete trials in a timely fashion. The vorinostat phase III trial took over 5 years to complete enrollment, despite enlisting 125 institutions in 23 countries. The majority of those sites enrolled only 1 or 2 patients over that time. Typically, pharmaceutical companies hire Contract Research Organizations (CROs) who identify sites to conduct trials and collect the data. CROs do not know which institutions will have the appropriate patients for the trial.
  • It would offer a referral mechanism to boost accrual. Patients look to the Meso Foundation as the primary source for medical advice, so they could be steered toward institutions that are participating in the designated studies.

Of course, the Meso Foundation does not have the resources to conduct clinical trials itself. The funding will initially need to come from the pharmaceutical industry. The process would work as such: Pharmaceutical companies interested in conducting large trials in mesothelioma would formulate an agreement to work with the Meso Foundation. Their protocols would be reviewed by a subcommittee of the Science Advisory Board who would provide feedback on the design and technical aspects. The Meso Foundation would solicit key institutions with strong research programs and high patient volumes to open the trial at their sites. Patients would be preferentially referred to these studies for participation. Once the success of this mechanism has been established, other companies will surely look to the Foundation for assistance. Ultimately, investigator-initiated trials could even be considered.

This year, more pharmaceutical companies than ever are planning clinical trials for patients with mesothelioma. This heightens the urgency to develop this consortium. As a research community, we cannot wait any longer. Let’s work together to make this happen.

Lee M. Krug, MD, is an Associate Attending Physician in the Division of Thoracic Oncology, Department of Medicine at Memorial Sloan-Kettering Cancer Center in New York, NY. Dr. Krug is also the Director of the Mesothelioma Program at Memorial Sloan-Kettering Cancer Center and Chair of the Science Advisory Board of the Mesothelioma Applied Research Foundation.


Tonight, as part of the “Meet the Experts” podcasts presented exclusively by the Meso Foundation, Dr. Julie Brahmer, Associate Professor of Oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins Hospital, sat down with Mary Hesdorffer, Nurse Practitioner and Medical Liaison for the Meso Foundation, to discuss her new trial targeting pleural mesothelioma. In her talk “NGR-hTTNF: A New Multi-Center Drug Trial for Pleural Mesothelioma”, Dr. Brahmer discussed a new chemotherapy treatment, designed specifically for pleural mesothelioma patients.

To participate in the trial, one prior treatment regimen is required (for example Alimta and Cisplatin) which usually consists of 4 to 6 treatments. The NGR-hTTNF trial can then be offered in the second line setting. Patients will receive an active chemotherapy agent, and plus or minus the experimental drug NGR-hTTNF. This trial is available worldwide. In the United States, it can be obtained at Johns Hopkins in Baltimore, Maryland, as well as a few others sites in Texas, California, and New York. According to Dr. Brahmer, it consists of a weekly treatment, with the most common side effects resulting in fatigue, nausea, headaches, and generalized weakness. The enrollment process into the NGR-hTTNF trial begins with an appointment with an investigator in trial.

The investigator reviews whether or not the patient is eligible based on treatments already received, the patient’s stage of mesothelioma, and his or her general physical well-being. Following this appointment, the patient must sign the consent form, after which eligibility is finalized through a few additional tests. If patients have responded positively or remain stable, chemotherapy will be discontinued after six cycles and patients will be extended an opportunity to receive NGR-hTTNF as a maintenance therapy. The primary end point of this trial will be to extend survival rates with secondary endpoints of improving quality of life and diminished symptoms of mesothelioma.

Having just returned from the annual Annual Meeting of the American Society for Clinical Oncology (ASCO) conference in Chicago, IL, where another expert from our “Meet the Experts” series — Dr. Lee M. Krug — gave his own summary of the event, Dr. Brahmer feels optimistic and excited about possibilities ahead. “I think a lot of the things we’re learning in lung cancer research may affect patients with mesothelioma,” she states. “There are some therapies being developed that stimulate the immune system, and I hope those types of drugs will have some type of role in combatting mesothelioma, as well.”

Dr. Brahmer’s research and clinical practice focuses on the development of new therapies for the treatment and prevention of lung cancer and mesothelioma. Dr. Brahmer has published several papers in this area of research and is a member of the American Society of Clinical Oncology and the Eastern Cooperative Oncology Group (ECOG) Thoracic Committee and Cancer Prevention Steering Committee. She is one of the founding Board members for the National Lung Cancer Partnership (formerly Women Against Lung Cancer). Within the National Lung Cancer Partnership she currently serves as a member and the Chairman of the Scientific Executive Committee. She serves on the medical advisory board of the Lung Cancer Research Fund and is a former member of the Mesothelioma Applied Research Foundation’s Science Advisory Board.

More information about this trial is available at the Meso Foundation by emailing or by calling (703) 879-3820. If you missed tonight’s “Meet the Experts” presentation, you can replay Dr. Brahmer’s talk and find out more about our other mesothelioma research resources at