Results from the pivotal QuANTUM-R phase 3 study of single agent quizartinib show that the drug met its primary endpoint of significantly prolonging overall survival (OS) compared to salvage chemotherapy in patients with relapsed/refractory acute myeloid leukemia (AML) with FLT3-ITD mutations after first-line treatment with or without hematopoietic stem cell transplantation (HSCT).
The study enrolled 367 patients FLT3-
ITD-mutated AML who were refractory to or in relapse (with duration of remission of 6 months of less) following standard first-line AML therapy with or without HSCT.
Patients in the study were randomized 2:1 to receive either single agent oral quizartinib or salvage chemotherapy.
Quizartinib, currently in phase 3 development by Daiichi Sankyo Cancer Enterprise, is an oral selective FLT3 inhibitor intended for the treatment of relapsed/refractory and newly-diagnosed AML with FLT3
-ITD mutations globally, and phase 2 development for relapsed/refractory AML with FLT3-
ITD mutations in Japan.
gene mutation is one of the most typical genetic abnormalities in individuals with AML. FLT3
-ITD is the most common FLT3
mutation, affecting an estimated 25% of patients.
"Single agent quizartinib is the first FLT3 inhibitor to show a significant improvement in overall survival compared to cytotoxic chemotherapy in a randomized phase 3 study of patients with relapsed/refractory AML with FLT3-ITD mutations, a very aggressive form of the disease with limited treatment options," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo in a press release
. "We sincerely thank all of the investigators and patients who participated in this study and will share the results of the QuANTUM-R study at an upcoming medical meeting. We look forward to working with regulatory authorities worldwide to potentially bring quizartinib to patients as quickly as possible."
The company intends to initiate regulatory submissions worldwide for quizartinib based on the QuANTUM-R results, and topline results are expected to be presented at an upcoming scientific conference.
Quizartinib has previously been granted both Fast Track and Orphan Drug designations by the U.S. Food and Drug Administration (FDA). In September, Daiichi Sankyo announced that it would be partnering with the University of Texas MD Anderson Cancer Center in a multi-year collaboration
dedicated to the acceleration of the drug’s development, as well as the development of at least 3 other novel therapies intended to treat AML.
In 2017, U.S. News & World Report ranked MD Anderson first for cancer care.
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