Verastem has commenced a Phase I trial of novel, small molecule inhibitor of focal adhesion kinase (FAK), VS-4718, in patients with advanced cancer.

A new small molecule inhibitor targeting the FAK pathway that is believed to be the key regulator of cancer stem cells, VS-4718 is the second compound in the company’s portfolio to enter clinical development.

Cedars-Sinai Medical Center, Los Angeles, experimental therapeutics programme co-director and principal investigator Dr Alain Mita said that significant scientific evidence suggest the importance of FAK as therapeutic target in metastatic solid tumours.

"There is significant scientific evidence to suggest that FAK could be an important therapeutic target in metastatic solid tumors," Mita added.

"We have incorporated a biomarker strategy into the design of this trial to help us further define the role of cancer stem cells and FAK in disease progression for patients with advanced tumors."

The open-label, multicentre study will assess the safety, pharmacokinetic and pharmacodynamic profile of VS-4718 in around 40 patients to be enrolled at three US locations.

The candidate’s initial evidence of activity as determined by cancer stem cell biomarkers will also be evaluated in the dose-escalation study.

Verastem chief medical officer Dr Joanna Horobin said FAK inhibition is likely to help in directly addressing the underlying cell population in tumours.

"This study should give us the necessary safety profile and dosing information to determine the future course of clinical development for VS-4718," Horobin said.

Several preclinical models of multiple tumour types conducted by the company have demonstrated that the FAK pathway is critical for the growth and survival of cancer stem cells, which are an underlying cause of tumour metastasis and recurrence.