VS-4718

September 28, 2015 By Mark Terry , BioSpace.com Breaking News Staff Needham, Mass.-based Verastem, Inc. announced today that it has halted enrollment in its Phase II clinical trial of VS-6063 in patients with mesothelioma due to insufficient efficacy. Analysis found that the drug was generally well tolerated for safety, but did not show enough efficacy to give the company a reason to continue. “Malignant pleural mesothelioma is among the most aggressive and lethal cancers with only one approved therapy,” said Lou Vaickus , interim chief medical officer of Verastem in a statement. “With the aggressiveness of this disease, the use of single agent VS-6063 as a maintenance treatment following chemotherapy where all patients had residual disease was not sufficient. There remains a significant unmet medical need for new treatment options for patients suffering from this very complex, difficult-to-treat cancer.” VS-6063 (defactinib), is an oral compound that targets cancer stem cells by inhibiting focal adhesion kinase (FAK). FAK activity is a key component in helping cancer stem cells grow and survive. In late August, the company’s stock dropped as a result of initial speculation on social media platform Twitter regarding the premature release of data of the Phase II study of VS-6063 in patients with KRAS mutant non-small cell lung cancer (NSCLC) at the 16th World Conference on Lung Cancer . Rumor was that two of the subjects of the study died and others showed severe side effects. In regards to that study, Verastem published a statement, saying, “There have been over 300 patients treated to date with VS-6063, including patients on the drug for more than one year. Verastem remains encouraged by the clinical potential of targeting cancer stem cells through FAK inhibition, and is unwavering in its commitment to delivering novel, safe and effective treatments to patients with unmet medical needs.” has had a tough year. Shares traded for $6.94 on Feb. 4, 2015, rose to a high of $12.10 on Mar. 20, dropped to $6.86 on June 29, rose again to $7.99 on Aug. 12, then plunged to $4.91 on Aug. 26. Shares crept back up to $6.53 on Sept. 17, but are currently trading for $2.27. The company had about $132 million at the end of its half-year point, and indicates it will redirect its efforts toward two other drugs for solid tumors, VS-4718 and VS-5584. In addition, VS-6063 is being studied in lung and ovarian cancers. Verastem was co-founded in August 2010 by Christoph Westphal based on research from MIT biologist Robert Weinberg . Westphal headed Sirtris Pharmaceuticals , which he sold to GlaxoSmithKline for $720 million in 2008. Verastem launched an initial public offering (IPO) in 2012. In 2013, Westphal stepped down as chief executive officer of Verastem and became executive chairman. In 2014 he co-founded Flex Pharma , a company that focuses on treatment for muscle cramps. He also founded Longwood Founders Fund , a venture fund, with Rich Aldrich and Michelle Dipp . In addition, he has helped found Alnylam Pharmaceuticals , Momenta Pharmaceuticals, Inc. and Acceleron Pharma . “We have stopped further enrollment and initiated an orderly wind-down of the COMMAND study,” said Robert Forrester , president and chief executive officer of Verastem in a statement. “We are disappointed with the COMMAND outcome, but we are deeply grateful for the support and commitment from the patient participating in the study, their families, and the study investors. Based on these results, we will reevaluate our clinical priorities and direct our resources toward further development of VS-6063, VS-4718, and VS-5584.”