The investigational Acute Myeloid Leukemia (AML) Franchise of the Daiichi Sankyo Cancer Enterprise is pushing the boundaries of science aiming to define a new standard of care for patients with AML, a fast-growing form of leukemia that has the lowest 5-year survival rate of all leukemias. Advancements in the understanding of the molecular biology of AML are creating an opportunity for our researchers to discover and develop therapies that target the underlying drivers of the disease.
Daiichi Sankyo Approach to AML
For more than 30 years, the standard of care for the treatment of AML went unchanged in part due to the complexity of the biology of AML. While a few new treatments have been approved recently, there is still significant unmet need and much work to be done to continue to expand the treatment options available for patients with AML. Our AML Franchise is evaluating a portfolio of therapies that leverage three distinct strategies for the treatment of AML, including quizartinib in phase 3 clinical development, milademetan (DS-3032), DS-3201 and PLX51107 in phase 1 clinical development, and DS-1001 in preclinical development. Our investigational AML Franchise will evaluate combination regimens including these and other compounds for their potential to change the standard of care for patients with AML.
Relentless Focus on Transforming Science
Molecular subtyping (classifying tumors into genetically distinct categories) is creating new opportunities to better understand the science behind AML and improve on current treatment options. Our investigational AML Franchise is currently developing a portfolio of therapies that leverage three distinct strategies:
- Growth Factor Receptor Inhibition with quizartinib, a FLT3 inhibitor in phase 3 clinical development
- Tumor Suppressor p53 Reactivation with milademetan (DS-3032), an MDM2 inhibitor in phase 1 development
- Targeting Epigenetic Regulation with DS-3201, a dual EZH1/EZH2 inhibitor in phase 1 development, PLX51107, a BRD4 inhibitor in phase 1 development, and DS-1001, a mutant IDH1 inhibitor in preclinical development
Pursuing multiple pathways and studying these investigational compounds in combination with other therapies may enable us to deliver more effective treatment options and expedite development to reach patients sooner.
These are investigational agents and have not been approved by the EMA, FDA or any other regulatory agency worldwide as a treatment for any indication. Safety and efficacy have not been established.
Quizartinib FLT3 Inhibitor
Quizartinib is the lead agent in the investigational AML Franchise of the Daiichi Sankyo Cancer Enterprise. A broad and comprehensive clinical development program is currently underway with quizartinib:
Pivotal Phase 3 Clinical Studies
QuANTUM-R: Single agent quizartinib versus salvage chemotherapy in relapsed/refractory FLT3-ITD AML. Data from QuANTUM-R was presented as a late-breaking oral presentation in the plenary program at the 23rd Congress of the European Hematology Association (EHA). (US, EU)
QuANTUM-First: Quizartinib in combination with induction and consolidation chemotherapy as well as a maintenance therapy for newly-diagnosed FLT3-ITD AML. For more information about QuANTUM-First, visit www.QuantumFirstStudy.com. (US, EU, Asia)
Phase 2 Clinical Studies
Relapsed/Refractory FLT3-ITD AML: Single agent quizartinib in relapsed/refractory FLT3-ITD AML (Japan)
Phase 1 Clinical Studies
Relapsed/Refractory and Newly-Diagnosed FLT3-ITD AML: Quizartinib in combination with milademetan (DS-3032) in relapsed/refractory FLT3-ITD AML or newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy (US)
Quizartinib has been granted 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 U.S. Food and Drug Administration (FDA).
Quizartinib also has received Orphan Drug designation by both the FDA and the European Medicines Agency (EMA) for the treatment of AML.
Milademetan (DS-3032): MDM2 Inhibitor
Milademetan (DS-3032) is an investigational oral selective MDM2 inhibitor currently being evaluated in four phase 1 clinical trials for solid and hematological malignancies, including AML, lymphoma and myelodysplastic syndrome (MDS).
Relapsed/Refractory and Newly-Diagnosed FLT3-ITD AML: Milademetan (DS-3032) in combination with quizartinib in relapsed/refractory FLT3-ITD AML or newly-diagnosed FLT3-ITD AML unfit for intensive chemotherapy (US)
Relapsed/Refractory AML and High-Risk MDS: Milademetan as a single agent or in combination with 5-azacitidine in relapsed/refractory AML or high-risk MDS (US)
Solid Tumors and Lymphomas: Milademetan in advanced solid tumors or lymphomas that have relapsed from or are refractory to standard treatment or for which no standard treatment is available (US)
Solid Tumors and Lymphomas: Milademetan in advanced solid tumors or lymphomas that have relapsed from or are refractory to standard treatment or for which no standard treatment is available (Japan)
DS-3201: EZH1/2 Inhibitor
DS-3201 is an investigational and potential first-in-class dual EZH1/2 inhibitor in phase 1 clinical development for hematologic cancers including AML, acute lymphocytic leukemia (ALL) and non-Hodgkin's lymphoma (NHL). DS-3201 is currently being evaluated in two open-label phase 1 studies:
AML and ALL: DS-3201 in relapsed or refractory AML or ALL (US)
NHL: DS-3201 in NHL (adult T-cell leukemia/lymphoma or peripheral T-cell lymphoma) that has relapsed or is refractory to standard treatment or for which no standard treatment is available (Japan)