Sumitomo Pharma Oncology Receives Orphan Drug Designation for DSP-5336, an Investigational Menin and Mixed-Lineage Leukemia Binding Protein for Treatment of Acute Myeloid Leukemia

Nov 03, 2022

Posted by Sumitomo Pharma Oncology, Inc.

CAMBRIDGE, Mass., August 3, 2022 /PRNewswire/Sumitomo Pharma Oncology, Inc., a clinical-stage company focused on novel cancer therapeutics, today announced 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).


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 (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).


About Sumitomo Pharma Oncology, Inc.

Sumitomo Pharma Oncology, Inc., is a wholly owned subsidiary of Sumitomo Pharma Co., Ltd. As a global oncology organization with teams in the U.S. and Japan, SMP Oncology is committed to advancing purposeful science by transforming new discoveries into meaningful treatments for patients with cancer. SMP Oncology’s robust and diverse pipeline of preclinical and clinical-stage assets spans multiple areas, including oncogenic pathways, survival mechanisms and novel protein interactions, which aim to address unmet clinical needs in oncology. For more information, visit


About Sumitomo Pharma Co., Ltd.

Sumitomo Pharma Co., Ltd. is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China, and other Asian countries with about 7,000 employees worldwide. Sumitomo Pharma Co., Ltd. defines its corporate mission as “To broadly contribute to society through value creation based on innovative research and development activities for the betterment of healthcare and fuller lives of people worldwide.” Additional information about Sumitomo Pharma Co., Ltd. is available through its corporate website at


Disclaimer Regarding Forward-Looking Statements

This press release contains “forward-looking statements,” as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding the research, development, and commercialization of pharmaceutical products. The forward-looking statements in this press release are based on management’s assumptions and beliefs in light of information presently available and involve both known and unknown risks and uncertainties. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical advice.




  1. Acute Myeloid Leukemia (AML). Updated July 21, 2021. Accessed July 11, 2022.
  2. Cierpicki T, Grembecka J. Challenges and opportunities in targeting the menin-MLL Interaction. Future Med Chem. 2014; 6(4):447-462. doi:10.4155/fmc.13.214.
  3. Matkar S, Thiel A, Hua X. Menin: a scaffold protein that controls gene expression and cell signaling. Trends Biochem Sci. 2013; 38(8):394-402. doi:10.1016/j.tibs.2013.05.005.
  4. Yokoyama A, Somervaille, Smith KS, et al. The menin tumor suppressor protein is an essential oncogenic cofactor for MLL-associated leukemogenesis. Cell. 2005;123:207-218. doi:10.1016/j.cell.2005.09.025.
  5. Grembecka J, He S, Shi A, et al. Menin-MLL inhibitors reverse oncogenic activity of MLL fusion proteins in leukemia. Nat Chem Biol. 2012; 8(3): 277-284. doi:10.1038/nchembio.773.
  6. Kuhn MWM, Song E, Feng Z, et al. Targeting chromatin regulators inhibits leukemogenic gene expression in NPM1 mutant leukemia. Cancer Discov. 2016; 6(10):1166-1181. doi:10.1158/2159-8290.CD-16-0237.

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