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Press Release

Cancer Research UK, MSD and Verastem Collaborate to Trial New Combination of Immunotherapy Drugs

Posted on: 30 Sep 16

CANCER RESEARCH UK has today (Friday) announced its first cross-company deal as part of its Experimental Cancer Medicine Centre (ECMC) Combinations Alliance.

MSD, Verastem, Inc. and Cancer Research UK will trial a new combination of immunotherapy drugs in mesothelioma, non small cell lung and pancreatic cancers. The trial will run through the ECMC network at centres in Edinburgh-Dundee, Southampton, Glasgow, Leicester and Belfast.

The phase Ib/IIa trial will investigate whether a focal adhesion kinase (FAK) inhibitor drug from Verastem called VS-6063 (defactinib)* can boost the effectiveness of a PD-1 immunotherapy drug from MSD called Keytruda (pembrolizumab).

It is based on discoveries by scientists at the Edinburgh Cancer Research UK Centre at the University of Edinburgh who showed that inhibiting FAK can release the cancer immune response. Defactinib may be able to take down a barrier of immune cells which are tricked into protecting the cancer cells while pembrolizumab can activate cancer-killing immune cells to attack those exposed cancer cells.

Around 50-60 cancer patients will take the drug combination, starting with a small dose and building this up to find what is safe. The scientists will also study how the treatments target the cancers and what effects the drug combination has on the tumours.

The trial will open between late 2016 and early 2017. It will be managed by the Cancer Research UK clinical trials unit in Glasgow and co-sponsored by the University of Glasgow and NHS Greater Glasgow and Clyde.

Dr Mercia Page, medical director of oncology at MSD, said: “We look forward to working with Cancer Research UK and Verastem on this promising combination. Strategic collaborations such as this reinforce the commitment we have to bringing a range of new treatments to the forefront, helping people with cancer who need a number of options available to them.”

Dr Greg Berk, Verastem chief medical officer, said: “Combining defactinib and MSD’s pembrolizumab through the Combinations Alliance expands our potential to deliver transformative therapies to patients with many types of cancer.

“We are delighted to be working with the Combinations Alliance, MSD, and world class scientists and medical centres throughout the UK on this trial. This study will build on the single agent activity of defactinib observed in early clinical trials in patients with non-small cell lung cancer and other tumour types, and follows from substantial preclinical research which has demonstrated that FAK inhibition optimizes the tumour immune balance and potentiates efficacy of PD-1 checkpoint inhibition.”

Dr Ian Walker, director of clinical research at Cancer Research UK, said: “It’s vital that we find new treatments for these three cancers which take tens of thousands of lives each year in the UK and we’re delighted to be working with MSD and Verastem on this.

“Our Combinations Alliance was set up to help develop partnerships between drug development companies and researchers to try new combinations of drugs in the hope of improving treatments and saving more lives from cancer. This is our first success in bringing together two organisations and we hope that this combination of immunotherapy drugs will benefit patients.”

Mesothelioma, pancreatic and non small cell lung cancers have very low survival – with more than half (60 per cent) of mesothelioma patients, more than three quarters (79 per cent) of pancreatic cancer patients and two thirds (68 per cent) of lung cancer patients dying within a year of diagnosis in England and Wales.*

Trial co-lead Dr Stefan Symeonides, from the University of Edinburgh, said: “Immunotherapy is a very exciting area of cancer research and we’ve seen remarkable benefits from pembrolizumab for some patients with hard-to-treat cancers, like melanoma and lung cancer. We’re hoping that the addition of defactinib will extend those benefits to more patients.

“This work could one day give a new treatment option that saves lives for this group of patients who have few options.”

Notes to editor:

* There are 2,688 new cases of mesothelioma and 2,429 deaths from the disease in the UK each year. There are 8,875 new cases of pancreatic cancer and 8,662 deaths from the disease in the UK each year. There are 48,488 new cases of lung cancer and 33,371 deaths from the disease in the UK each year – 90 per cent of lung cancer cases are non small cell lung cancer.

About MSD

At MSD we believe the most important thing we make is a difference. We operate in more than 140 countries and through our prescription medicines, vaccines, biologic therapies, and animal health products we work with customers to bring innovative healthcare solutions to those who need them the most. We also demonstrate our commitment to increasing access to healthcare through far-reaching policies, programmes and partnerships. For more information visit The company is known as Merck & Co., Inc., Kenilworth, NJ, USA in the United States and Canada. Everywhere else, we are known as MSD.

About Verastem, Inc.

Verastem, Inc. (NASDAQ:VSTM) is a biopharmaceutical company focused on discovering and developing drugs to improve outcomes for patients with cancer. Our product candidates utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. Our most advanced clinical product candidates are the Focal Adhesion Kinase inhibitors, VS-6063 and VS-4718, and the dual PI3K/mTOR inhibitor, VS-5584. For more information, please visit

About Focal Adhesion Kinase

Focal Adhesion Kinase (FAK) is a non-receptor tyrosine kinase encoded by the PTK-2 gene that is involved in cellular adhesion and, in cancer, metastatic capability. VS-6063 (defactinib) and VS-4718 are orally available compounds that are potent inhibitors of FAK. VS-6063 and VS-4718 utilize a multi-faceted approach to treat cancer by reducing cancer stem cells, enhancing anti-tumor immunity, and modulating the local tumor microenvironment. VS-6063 and VS-4718 are currently being studied in multiple clinical trials for their ability to improve patient outcome.

About the ECMC network

The Experimental Cancer Medicine Centres (ECMC) Network is an initiative funded in partnership between Cancer Research UK and the four Health Departments of England, Scotland, Northern Ireland, and Wales. Launched in 2007, this £35 million infrastructure award supports a network of 18 adult and 9 paediatric Centres of Excellence distributed throughout the UK. The ECMC Network strives to push the boundaries of cancer care, by bringing together world-class pockets of scientific and clinical expertise in each of its adult and paediatric ECMCs whilst providing industry partners with the opportunity to obtain access to cutting-edge, early-stage innovation. In 2014-15 alone, ECMCs leveraged over £73,000,000 from commercial partnerships, with over 2,500 patients recruited onto the 389 ECMC-supported clinical trials. Find out more at or @ECMC_UK.

About Cancer Research UK

  • Cancer Research UK is the world’s leading cancer charity dedicated to saving lives through research.
  • Cancer Research UK’s pioneering work into the prevention, diagnosis and treatment of cancer has helped save millions of lives.
  • Cancer Research UK receives no government funding for its life-saving research. Every step it makes towards beating cancer relies on every pound donated.
  • Cancer Research UK has been at the heart of the progress that has already seen survival in the UK double in the last forty years.
  • Today, 2 in 4 people survive their cancer for at least 10 years. Cancer Research UK’s ambition is to accelerate progress so that 3 in 4 people will survive their cancer for at least 10 years within the next 20 years.
  • Cancer Research UK supports research into all aspects of cancer through the work of over 4,000 scientists, doctors and nurses.
  • Together with its partners and supporters, Cancer Research UK's vision is to bring forward the day when all cancers are cured.

For further information about Cancer Research UK's work or to find out how to support the charity, please call 0300 123 1022 or visit . Follow us on Twitter and Facebook .

Verastem forward-looking statements notice

This press release includes forward-looking statements about Verastem’s strategy, future plans and prospects, including statements regarding the development and activity of Verastem’s product candidate, VS-6063, and Verastem’s FAK program generally, and the potential for combination of FAK inhibitors with immuno-oncology agents, including a potential indication for pembrolizumab in combination with VS-6063 for mesothelioma, non small cell lung and pancreatic cancers. 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 Verastem’s product candidates and preliminary or interim data from clinical trials may not be predictive of the results or success of ongoing or later clinical trials, that data may not be available when we expect it to be, that enrollment of clinical trials may take longer than expected, that our product candidates will cause unexpected safety events, that Verastem will be unable to successfully initiate or complete the clinical development of its product candidates, that the development of Verastem’s product candidates will take longer or cost more than planned, and that Verastem’s product candidates will not receive regulatory approval or become commercially successful products. Other risks and uncertainties include those identified under the heading “Risk Factors” in Verastem’s Annual Report on Form 10-K for the year ended December 31, 2015 and in any subsequent SEC filings. The forward-looking statements contained in this press release reflect Verastem’s current views with respect to future events, and Verastem does not undertake and specifically disclaims any obligation to update any forward-looking statements.

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Last updated on: 30/09/2016

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