Verastem's defactinib gets FDA orphan drug designation for treatment of mesothelioma

Verastem, Inc., (NASDAQ: VSTM) focused on discovering and developing drugs to treat cancer by the targeted killing of cancer stem cells, announced that lead cancer stem cell inhibitor, VS-6063 (defactinib), has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for use in the treatment of mesothelioma, a rare form of lung cancer. The designation is designed to encourage the development of drugs which may provide significant benefit to patients suffering from rare diseases.

“We believe new treatment options targeting cancer stem cells will be critical to achieve a durable clinical benefit for patients. This designation will provide us with many benefits as we pursue the development of defactinib for the treatment of mesothelioma.”

"Mesothelioma is among the most aggressive and lethal cancers but has limited treatment options," said Robert Forrester, Verastem President and Chief Executive Officer. "We are pleased that the FDA recognizes the significant unmet medical need in mesothelioma. We previously received orphan medicinal product status for defactinib in Europe and these two designations are an important component of our development strategy."

Verastem recently outlined details of the registration-directed clinical study of defactinib in patients with malignant pleural mesothelioma. This study is designed as a double-blind, placebo-controlled trial with an expected enrollment of approximately 350-400 patients at clinical sites in 11 countries.

"We are in discussions with the regulatory agencies and clinical investigators worldwide," said Dr. Joanna Horobin, Verastem Chief Medical Officer. "We recently held our investigator meetings for the physicians conducting the trial in the US and Australia and we are on track to begin enrolling patients in the third quarter. We plan to open sites worldwide on a rolling basis as we clear regulatory and clinical review in each country."

Orphan drug designation is granted by the FDA Office of Orphan Drug Products to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design, and an exemption from FDA user fees.

"Cancer stem cells play a central role in treatment resistance in many types of cancers," said Christoph Westphal, M.D., Ph.D., Verastem Executive Chairman. "We believe new treatment options targeting cancer stem cells will be critical to achieve a durable clinical benefit for patients. This designation will provide us with many benefits as we pursue the development of defactinib for the treatment of mesothelioma."

In addition to mesothelioma, Verastem recently announced the completion of the Phase 1 stage and initial data from an ongoing Phase 1/1b study of defactinib in combination with weekly paclitaxel for patients with ovarian cancer. Verastem also expects to initiate a Phase 1 study in Japan, and a Phase 2 trial in KRAS-mutated Non-Small Cell Lung Cancer, for defactinib during the third quarter of 2013.

SOURCE Verastem, Inc.