On August 3, 2022 Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, reported the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for DSP-5336, an investigational small molecule inhibitor against the binding of menin and mixed-lineage leukemia (MLL) protein, for the treatment of acute myeloid leukemia (AML) (Press release, Sumitomo Pharmaceuticals, AUG 3, 2022, View Source [SID1234617431]).
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"We are pleased to have received this designation for DSP-5336 which reinforces the need to identify novel therapeutic options to improve outcomes for patients with AML," said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. "We are excited to contribute to the advancement of research in this hematologic malignancy."
The FDA’s Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. AML is a blood cancer that starts in bone marrow. Bone marrow typically produces white blood cells, red blood cells and platelets. However, in people with AML, the bone marrow makes abnormal cancerous white blood cells called myeloid blasts. AML swiftly moves from the bone marrow into your bloodstream and can even involve other parts of your body.1
"MLL gene rearrangements and nucleophosmin 1 (NPM1) mutations are potent drivers of leukemia growth, and menin is a necessary copilot. DSP-5336 is a small molecule that blocks the MLL-menin partnership which inhibits normal differentiation and promotes leukemia growth."2-5 detailed Jatin J. Shah, M.D., Chief Medical Officer of Sumitomo Pharma Oncology, Inc. "We’re encouraged by preclinical evidence showing that blocking the menin-MLL interaction may stop the development of leukemia and restore normal terminal differentiation in MLL rearranged and NPM1-mutant malignant myeloid cells."2,6
DSP-5336 is currently being evaluated in a Phase 1/2 clinical trial to evaluate the safety and efficacy of DSP-5336 in patients with Relapsed/Refractory AML/ ALL with or without MLL Rearrangement or NPM1 Mutation, which is being conducted in the United States and Japan. To learn more about the study and eligibility for enrollment, visit clinicaltrials.gov (NCT04988555).
This is the third recently announced Orphan Drug Designation from SMP Oncology. TP-3654, the company’s proprietary investigational oral inhibitor of PIM kinases, was granted Orphan Drug Designation for the treatment of myelofibrosis (NCT04176198) as well as DSP-0390, an investigational emopamil-binding protein (EBP) inhibitor, was also granted Orphan Drug Designation for the treatment of brain cancer (NCT05023551). These designations support the strength and diversity of SMP Oncology’s pipeline and commitment to oncology research and development.
About DSP-5336
DSP-5336 is an investigational small molecule inhibitor against the binding of menin and mixed-lineage leukemia (MLL) protein. Menin is a scaffold nuclear protein that plays various key roles in biological pathways, including cell growth regulation, cell cycle control, genomic stability, bone development, and hematopoiesis.2,3 In preclinical studies DSP-5336 has shown selective growth inhibition in human acute leukemia cell lines with MLL rearrangements or NPM1 mutations.2,6 DSP-5336 is currently being evaluated in a Phase 1/2 dose escalation/dose expansion study of DSP-5336 in patients with relapsed or refractory AML (NCT04988555).