QuANTUM-R phase III study of AC 220 consistent with overall survival data in acute myeloid leukemia.- Daiichi Sankyo.

Daiichi Sankyo announced comprehensive analyses of overall survival from the pivotal QuANTUM-R phase III study of single agent AC 220 (quizartinib) compared to salvage chemotherapy in patients with FLT3-ITD acute myeloid leukemia (AML), which was refractory or relapsed within six months of first remission. Pre-specified sensitivity analyses of overall survival and event-free survival as well as predefined subgroup analyses from the QuANTUM-R study of quizartinib were found to be consistent with the primary analysis of overall survival.

Analyses of key exploratory endpoints such as composite complete remission (CRc), duration of CRc and hematopoietic stem cell transplant (HSCT) rate also were consistent with, and supportive of, the primary overall survival benefit demonstrated in QuANTUM-R. Incidence of treatment-emergent adverse events was comparable between patients who received single agent quizartinib and those who received salvage chemotherapy. The most common adverse drug reactions (more than 30 percent, any Grade) in patients treated with quizartinib included infections, bleeding, nausea, asthenic conditions, pyrexia, febrile neutropenia and vomiting, and the most common Grade of at least 3 adverse drug reactions (more than 20 percent) were infection and febrile neutropenia. Data were presented during an oral presentation at the 60th Annual Meeting of the American Society of Hematology.

Comment: Regulatory marketing applications for quizartinib are currently under expedited review in the U.S, Japan and EU. Quizartinib has been granted Priority Review and Breakthrough Therapy designation for the treatment of adult patients with relapsed/refractory FLT3-ITD AML, and Fast Track designation for the treatment of relapsed/refractory AML by the FDA. Quizartinib also has been granted accelerated assessment by the European Medicines Agency for the treatment of adults with relapsed or refractory AML which is FLT3-ITD positive, and granted Orphan Drug designation by both the FDA and the European Commission for the treatment of AML and by the Japan Ministry of Health, Labour and Welfare for the treatment of FLT3-mutated AML.