Verastem, Inc., a clinical-stage biopharmaceutical company, has entered into an agreement with Laboratory Corporation of America Holdings (LabCorp) to validate biomarkers for its lead focal adhesion kinase (FAK) inhibitor VS-6063 in the development of an applicable companion diagnostic.

The biomarkers will be the subject of clinical studies in ovarian cancer and mesothelioma, including a potentially pivotal study of VS-6063 in mesothelioma expected to initiate later this year.

“The identification of patients most likely to benefit from targeted therapy is critical to accelerating the drug development and approval process,” said Henri Termeer, lead director, Verastem.

Clinical assay validation is an integral component to all companion diagnostics entering an FDA approval process.

Robert Weinberg, Ph.D., Verastem co-founder and chair of the Scientific Advisory Board, and others have demonstrated that FAK signaling plays a central role in the tumor-initiating capability of cancer stem cells and ultimate disease progression. VS-6063 is designed to target and kill cancer stem cells by inhibiting FAK signaling.

Mesothelioma tumours lacking the tumour suppressor Merlin appear to be particularly sensitive to FAK inhibitors. As shown recently at the EORTC-NCI-AACR Symposium on Molecular Targets and Cancer Therapeutics, sensitivity due to lack of Merlin is evident in both research models and early clinical proof-of-concept.

“We believe the approximately 50 per cent of mesothelioma patients lacking Merlin may be particularly responsive to treatment with FAK inhibitors,” said Dan Paterson, Verastem vice president, Head of Corporate Development and Diagnostics. “LabCorp is the perfect partner to progress our research on Merlin into a companion diagnostic for VS-6063.”

“We are pleased to collaborate with Verastem in guiding the clinical development of cancer stem cell-targeted agents,” said David P King, chairman and chief executive officer of LabCorp. “This relationship represents another example of our accelerating initiatives in companion diagnostics and personalized medicine.”

VS-6063 is an oral small molecule inhibitor of focal adhesion kinase (FAK) which is a critical pathway for cancer stem cells. VS-6063 was well-tolerated and demonstrated signs of clinical activity in a phase I study in advanced solid tumours. Verastem has demonstrated that loss of the tumour suppressor Merlin confers increased susceptibility to FAK inhibition. Verastem is planning to initiate multiple clinical trials in 2013 with VS-6063 including a potentially pivotal study in mesothelioma.

Verastem, Inc. is a clinical-stage biopharmaceutical company focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells.