September 6, 2017
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON -- Verastem, Inc. ’s experimental blood cancer drug hit its endpoints in a Phase III trial and the news has sent shares of company stock up more than 30 percent this morning.
Verastem said duvelisib, an inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, demonstrated a superior progression free survival over ofatumumab (Novartis’ Arzerra), a standard of care treatment, in the Phase III DUO trial. The experimental drug is being tested in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL). PI3K signaling is believed to lead to the proliferation of malignant B-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment, the company said. Duvelisib is designed to suppress those enzymes.
In a statement this morning, the company said patients taking duvelisib achieved a statistically significant improvement in median progression free survival of 13.3 months. That was compared to 9.9 months for patients taking ofatumumab. The company said those numbers represent a 48 percent reduction in the risk of progression or death.
Based on the outcome of the Phase III trial, Verastem is poised to seek regulatory approval next year. Although it did not provide a specific month, Verastem said it will file a New Drug Application with the U.S. Food and Drug Administration in the first half of 2018. The company said the NDA will be filed with data from the DUO trial as well as the DYNAMO study in indolent non-Hodgkin’s lymphoma. In that study, duvelisib achieved an objective response rate of 46 percent.
Robert Forrester, president and chief executive officer of Verastem, said duvelisib has been an important acquisition for the company. The company licensed the drug in 2016 from Infinity Pharmaceuticals .
“Both of our late-stage trials with duvelisib monotherapy (DUO and DYNAMO) have now achieved their primary endpoints, highlighting the significant potential of duvelisib in the treatment of advanced hematologic malignancies. We anticipate sharing these results with the FDA in preparation for a potential NDA filing during the first half of 2018 and look forward to exploring subsequent development opportunities for duvelisib in additional cancers,” Forrester said in a statement.
Ian Flinn, director of the Blood Cancer Research Program at Sarah Cannon Research Institute and the lead investigator on the DUO study, said that although treatment of CLL/SLL has advanced in the past few years, there is still an unmet need in treating patients who relapse following currently available therapies. He said the duvelisib results demonstrate that the drug will be a “valuable addition to the treatment landscape.”
Shares of Verastem are trading at $4.98 as of 10:42 a.m. It was trading as high as $5.54 when the market opened.
