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BOSTON, MA, USA I February 7, 2018 I Verastem, Inc. (NASDAQ: VSTM), focused on discovering and developing drugs to improve the survival and quality of life of cancer patients, today announced it has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking full approval for its lead product candidate duvelisib, a first-in-class oral dual inhibitor of phosphoinositide-3-kinase (PI3K)-delta and PI3K-gamma, for the treatment of relapsed or refractory chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) and accelerated approval for the treatment of relapsed or refractory follicular lymphoma (FL). Duvelisib has received Fast Track Designation from the FDA for patients with CLL or peripheral T-cell lymphoma (PTCL) who have received at least one prior therapy and for patients with FL who have received at least two prior therapies. In addition, duvelisib received orphan drug designation in the United States and the European Union for patients with CLL, SLL and FL. “The submission of our first NDA for duvelisib is a major milestone for Verastem and is the culmination of substantial effort by our employees and the investigators who have dedicated themselves toward developing a potential treatment option for patients who are in need of additional therapies. We are immensely grateful to all of the patients that participated in the duvelisib clinical trial program over the last few years,” said Robert Forrester, President and Chief Executive Officer of Verastem. “Oral duvelisib is the first PI3K inhibitor to show efficacy as a monotherapy in a randomized Phase 3 study in patients with relapsed or refractory CLL/SLL. Duvelisib monotherapy has also demonstrated significant clinical activity in patients with double-refractory FL. We believe duvelisib will offer a convenient oral treatment alternative. We look forward to working with the FDA during the review process and to a potential U.S. approval decision for duvelisib in early 2019.” The NDA is supported by clinical data from the randomized Phase 3 DUO™ study demonstrating significant efficacy, along with a consistent and manageable safety profile, of duvelisib monotherapy in patients with relapsed or refractory CLL/SLL. The DUO study met its primary endpoint with oral duvelisib monotherapy achieving a statistically significant improvement in progression-free survival (PFS) compared to ofatumumab in patients with relapsed or refractory CLL/ SLL (median PFS of 13.3 months versus 9.9 months, respectively; HR=0.52; p<0.0001), representing a 48% reduction in the risk of disease progression or death. The NDA is also supported by results from the Phase 2 DYNAMO™ study in patients with indolent non-Hodgkin’s lymphoma that are double-refractory to both rituximab and chemotherapy or radioimmunotherapy, which also achieved its primary endpoint with an objective response rate (ORR) of 46% (p<0.0001). In the subset of patients enrolled in DYNAMO with double-refractory FL (n=83), duvelisib demonstrated an ORR of 41%. About Duvelisib Duvelisib is a first-in-class investigational, dual inhibitor of phosphoinositide 3-kinase (PI3K)-delta and PI3K-gamma, two enzymes known to help support the growth and survival of malignant B-cells and T-cells. PI3K signaling may lead to the proliferation of malignant B- and T-cells and is thought to play a role in the formation and maintenance of the supportive tumor microenvironment.1,2,3 Duvelisib was evaluated in late- and mid-stage extension trials, including DUO™, a randomized, Phase 3 monotherapy study in patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL),4 and DYNAMO™, a single-arm, Phase 2 monotherapy study in patients with refractory indolent non-Hodgkin lymphoma (iNHL).5 Both DUO and DYNAMO achieved their primary endpoints and Verastem has submitted a New Drug Application (NDA) requesting the full approval of duvelisib for the treatment of patients with relapsed or refractory CLL/SLL, and accelerated approval for the treatment of patients with relapsed or refractory follicular lymphoma (FL). Duvelisib is also being developed by Verastem for the treatment of peripheral T-cell lymphoma (PTCL), and is being investigated in combination with other agents through investigator-sponsored studies.6 Information about duvelisib clinical trials can be found on www.clinicaltrials.gov. About Verastem, Inc. Verastem, Inc. (NASDAQ:VSTM) is a biopharmaceutical company focused on discovering and developing drugs to improve outcomes for patients with cancer. Verastem is currently developing duvelisib, a dual inhibitor of PI3K-delta and PI3K-gamma, which has successfully met its primary endpoint in a Phase 2 study in iNHL and a Phase 3 clinical trial in patients with CLL/SLL. In addition, Verastem is developing the FAK inhibitor defactinib, which is currently being evaluated in three separate clinical collaborations in combination with immunotherapeutic agents for the treatment of several different cancer types, including pancreatic cancer, ovarian cancer, non-small cell lung cancer, and mesothelioma. Verastem’s product candidates seek to treat cancer by modulating the local tumor microenvironment, enhancing anti-tumor immunity, and reducing cancer stem cells. For more information, please visit www.verastem.com. |
SOURCE: Verastem
