Astellas Reports XOSPATA, Azacitidine Combination Did Not Meet Endpoint

Astellas Pharma announced that a Phase 3 trial of XOSPATA® (gilteritinib) plus azacitidine versus azacitidine alone in newly diagnosed FLT3 mutation-positive (FLT3mut+) acute myeloid leukemia (AML) patients who were ineligible for intensive induction chemotherapy did not meet its primary endpoint of overall survival at a planned interim analysis of the LACEWING trial. An independent Data Monitoring Committee recommended terminating the study for futility, concluding results are unlikely to show a statistically significant increase in overall survival. Astellas has stopped enrollment in the trial and is reviewing the results for other action as needed.

"Although we are disappointed by the primary outcome of LACEWING, we are conducting a thorough review of the data and plan to share detailed results at a later date," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas. "These results do not affect other ongoing gilteritinib trials. We remain committed to our comprehensive program investigating gilteritinib across a wide range of AML patients with a positive FLT3 mutation, building on gilteritinib's earlier, positive data in patients with relapsed or refractory FLT3 mutation-positive AML."

AML, a cancer of the blood and bone marrow, is one of the most common types of leukemia in adults. It has the lowest survival rate of all types of leukemia. Approximately one-third of people with AML have a FLT3 mutation. This mutation is associated with worsened disease-free survival and overall survival, and a higher risk of getting the disease more than once. Among patients with FLT3mut+ AML, an estimated 30-40 percent are not candidates for intensive chemotherapy regimens because of age, performance status, and/or comorbid conditions.

Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and commercialize gilteritinib. Gilteritinib is available as XOSPATA® in the U.S., Japan and selected European countries, among others, for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML. Gilteritinib is an FMS-like tyrosine kinase 3 (FLT3) inhibitor with demonstrated activity against FLT3-ITD, a common driver mutation that presents with a high burden and poor prognosis, and FLT3-TKD mutations.