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Verastem, Inc.,
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.
“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.
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