AML Franchise edits 12 2017
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, DS-3032, DS-3201 and PLX51107 in phase 1 clinical development, and DS-1001 in preclinical development. Our 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 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 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
Source: Adapted from: Dohner-H et al., NEJM 2015; 373:1136-1152, Thol-F et al., Blood 2015; 126:319-327, Khan et al., Clin Can Res, 2012; Ramos-N, et al., J. Clin. Med. 2015; 4:665-695, Isidori-A et al., Can Res Frontiers 2016; 2:226-251
Pursuing multiple pathways and studying these investigational compounds in combination
s 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 FDA or any other regulatory agency worldwide as a treatment for any indication. Safety and efficacy have not been established.
To learn more about the AML Franchise, click here.
Acute Myeloid Leukemia (AML) Franchise
Quizartinib is an investigational oral selective FLT3 inhibitor in phase 3 development for newly-diagnosed and relapsed/refractory AML with FLT3-ITD mutations. Quizartinib also has been granted Fast Track Designation by the FDA for the treatment of relapsed/refractory AML. Quizartinib has been granted Orphan Drug Designation by the FDA and EMA for the treatment of AML. Two phase 3 studies of quizartinib are currently underway:
QuANTUM-First: This study is evaluating quizartinib in combination with induction and consolidation chemotherapy as well as a maintenance therapy for newly-diagnosed AML with FLT3-ITD mutations. For more information about QuANTUM-First, visit www.QuantumFirstStudy.com.
QuANTUM-R: This study is evaluating quizartinib versus salvage chemotherapy in relapsed / refractory AML with FLT3-ITD mutations.
A phase 2 study is also ongoing in Japan in relapsed/refractory FLT3-ITD mutated AML.
DS-3032 is an investigational oral selective MDM2 inhibitor currently being evaluated in three phase 1 clinical trials for solid and hematological malignancies, including AML, acute lymphocytic leukemia (ALL), chronic myeloid leukemia (CML) in blast phase, lymphoma and myelodysplastic syndrome (MDS).
Hematological Malignancies (U.S): A phase 1 study evaluating DS-3032 in several hematological malignancies including relapsed/refractory acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL), chronic myeloid leukemia (CML) in blast phase and myelodysplastic syndrome (MDS).
Solid Tumors and Lymphomas (U.S): A phase 1 study evaluating DS-3032 in advanced solid tumors or lymphomas that have relapsed from or are refractory to standard treatment or for which no standard treatment is available.
Solid Tumors and Lymphomas (Japan): A phase 1 study evaluating DS-3032 in advanced solid tumors or lymphomas that have relapsed from or are refractory to standard treatment or for which no standard treatment is available.
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 (US): A phase 1 trial evaluating DS-3201 in relapsed or refractory AML or ALL.
NHL (Japan): A phase 1 dose escalation trial evaluating DS-3201 in NHL that has relapsed or is refractory to standard treatment or for which no standard treatment is available.