As reported online June 13, 2013 by the Wall Street Journal, “Verastem Investigational Mesothelioma Treatment Gets Orphan Status in EU”:
Verastem Inc. (VSTM) was granted orphan status by European regulators for its investigational treatment for mesothelioma, an asbestos-related cancer with limited treatment options.
The cancer-drug developer, which is focused on developing drugs to treat cancer by the targeted killing of cancer stem cells, also is seeking orphan status for the treatment–known as VS-6063–from the U.S. Food and Drug Administration.
“We are in discussions with regulatory agencies world-wide,” said Verastem President and Chief Operating Officer Robert Forrester. The company plans to begin a trial of the oral treatment–a inhibitor of focal adhesion kinase known as VS-6063–in mesothelioma later this summer, Mr. Forrester said.
A Veristem press release issued that same day, “Verastem Hosts Dr. Dean Fennell for a Mesothelioma Briefing at ASCO”, provides some additional contextual information about this important development:
Dr. Fennell is the 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. Dr. Fennell presented together with Verastem Chief Medical Officer Dr. Joanna Horobin and Head of Research Dr. Jonathan Pachter.
“Asbestos is the causative agent in the vast majority of mesothelioma cases” said Dr. Fennell. “Sadly the use of asbestos continues worldwide. Due to the 10-50 year latency period from asbestos exposure to development of disease we expect that the current trend of increasing mesothelioma incidence worldwide will continue. 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.”…
“We are moving quickly to bring new treatment options to patients with mesothelioma” said Dr. Horobin. “Our lead compound VS-6063 is an orally-available agent that inhibits the Focal Adhesion Kinase or FAK pathway….
Approximately 40-50% of mesothelioma patients lack Merlin and studies by Verastem and others have shown that Merlin-low mesothelioma cells and tumors appear to be particularly sensitive to FAK inhibition.
We will watch for clinical trial study findings as regards this new mesothelioma treatment VS-6063 as well as other investigational mesothelioma treatments.
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