Product Profile

Defactinib is an oral small molecule inhibitor of FAK and PYK2. Verastem has patent protection of defactinib through 2029, and has received orphan drug designation for defactinib in ovarian and mesothelioma in the US, the EU, and Australia.

Clinical Development in Advanced Cancers

Defactinib has been studied in over 300 patients with a well tolerated safety profile. It has demonstrated initial signs of activity in clinical studies as a monotherapy in KRAS mt NSCLC and in relapsed ovarian cancer in combination with paclitaxel.

Clinical Investigation of Defactinib Combinations

Preclinical research by Verastem scientists and collaborators at world-renowned research institutions has described the central role FAK signaling has in effectively reducing cancer stem cells, enhancing immune response by decreasing immuno-suppressive cells and increasing cytotoxic T cells, and reducing stromal density which allows both drug and tumor-killing immune cells to enter the tumor. Additionally, immuno-modulation effects were shown to substantially increase survival of mice when FAK inhibitors were combined with an anti-PD-1 immune checkpoint antibody. These results have indicated the potential promise of combination therapy in future clinical studies.

Defactinib is currently being evaluated as a potential combination therapy for pancreatic cancer, non-small cell lung cancer, ovarian cancer, and mesothelioma.

Ongoing studies with defactinib include:

  • A Phase 1b study in combination with Merck & Co.’s PD-1 inhibitor pembrolizumab and gemcitabine in patients with advanced pancreatic cancer.
  • A Phase 1/2 collaboration with Pfizer Inc. and Merck KGaA to evaluate combination therapy with avelumab, an anti-PD-L1 antibody, in patients with relapsed ovarian cancer.
  • A Phase 1/2 collaboration with Merck & Co. and CRUK to evaluate combination therapy with pembrolizumab (anti-PD-1). Following dose escalation, three expansion arms are planned in NSCLC, relapsed pancreatic cancer, and mesothelioma.

Investigator Sponsored Trials (IST)

We are committed to advancing the science and development of our compounds towards improving outcomes for patients with cancer. We welcome collaboration with you and your sponsoring institution towards this shared goal.

To submit a research proposal, please complete and submit the following form to

Investigator Sponsored Trial Proposal Form