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FDA Issues Approval for Phase 2 Clinical Trial of Potential New Mesothelioma Treatment


FDA Issues Approval for Phase 2 Clinical Trial of Potential New Mesothelioma Treatment

The FDA has issued approval for biopharmaceutical company Verastem to begin a Phase 2 clinical study of a new drug for the treatment of malignant pleural mesothelioma. VS-6063 is an orally available, small molecule inhibitor of a crucial signaling pathway inside stem cells called the Focal Adhesion Kinase (FAK) pathway.

FAK is vital for tumor development and is critical for the survival of cancer stem cells. VS-6063 was well-tolerated in a Phase 1 study and demonstrated signs of clinical activity in advanced solid tumors.

Dr. Dean Fennell, Chair of Thoracic Medical Oncology at the University of Leicester, incoming President of the International Mesothelioma Interest Group (iMig) and a member of the Verastem Mesothelioma Steering Committee, presented promising data at a briefing session on VS-6063 at the annualAmerican Society of Clinical Oncology meeting in Chicago, IL, in early June of this year.

Studies by Verastem and others have shown that chemotherapy agents most commonly used in the treatment of mesothelioma do not kill cancer stem cells in mesothelioma, but actually increase growth of cancer stem cells. This includes the only FDA approved chemotherapy drug pemetrexed (Alimta), along with other commonly used agents cisplatin, vinorelbine and gemcitabine. VS-6063 has the ability to kill these cancer stem cells.

Studies also show that mesothelioma patients who lack the tumor suppressor gene Merlin have increased sensitivity to FAK inhibition, which would greatly enhance the effectiveness of a drug like VS-6063. Currently a biomarker test is being developed in conjunction with LabCorp to identify mesothelioma patients low in Merlin, this accounts for approximately 40-50% of mesothelioma patients

“These early results suggest that a targeted therapy, particularly when used in combination with a specific biomarker, has the potential to significantly improve treatment of this aggressive and deadly disease,” said Dr. Fennell, “There is a large unmet medical need in mesothelioma and an opportunity to develop targeted agents to bring new hope to patients struggling with their disease.”

VS-6063 is also being studied in a Phase 1/1b trial in combination with paclitaxel for the treatment of ovarian cancer.