Sumitomo Dainippon Pharma Oncology Announces First Patient Dosed in Phase 1/2 Study of DSP-5336

Apr 08, 2022

in Patients with Acute Leukemia with and without Mixed Lineage Leukemia (MLL)-rearrangement or Nucleophosmin 1 (NPM1) Mutation

Posted by Sumitomo Pharma Oncology, Inc.

CAMBRIDGE, Mass., Mar. 29, 2022 – Sumitomo Dainippon Pharma Oncology, Inc., a clinical-stage pharmaceutical company focused on research and development for novel cancer therapeutics, today announced that the first patient has been dosed in the Phase 1/2 study of DSP-5336, an inhibitor of menin binding to mixed-lineage leukemia (MLL) protein, in patients with relapsed or refractory acute myeloid leukemia (AML) or acute lymphocytic leukemia (ALL).  

“Patients with refractory or relapsed AML or ALL have an unfavorable prognosis and respond poorly to available treatments.1 Dosing the first patient in our Phase 1/2 study is an important milestone as we evaluate DSP-5336 and its safety and potential benefits for this patient population,” said Patricia S. Andrews, Chief Executive Officer, Global Head of Oncology, Sumitomo Dainippon Pharma Oncology, Inc (SDP Oncology). “It is our hope that the data generated by this study furthers our goal of advancing meaningful treatments for patients with blood cancer.”

The Phase 1/2 open-label study consists of two parts, dose escalation and dose expansion. The primary objectives of the Phase 1 dose escalation portion of the study are to assess the safety and tolerability of DSP-5336 in relapsed or refractory AML or ALL and to determine the recommended Phase 2 dose (RP2D). Secondary objectives include assessment of preliminary clinical activity of DSP-5336 in adult patients with relapsed or refractory AML or ALL.

Following the completion of the Phase 1 dose escalation portion of the study, the study will move into the Phase 2 dose expansion. The primary objective of the Phase 2 dose expansion portion of the study is to further evaluate the safety and clinical activity of DSP-5336 in adult patients with relapsed or refractory AML who have MLL rearrangement or nucleophosmin 1 (NPM1) mutation. 

Additional information on this trial, including comprehensive inclusion and exclusion criteria, can be accessed at (NCT04988555). 

About DSP-5336

DSP-5336 is a 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 Binding of menin to MLL fusion and wildtype proteins leads to the upregulation of HOXA family and MEIS1 genes that function to stall myeloid cellular differentiation and induce leukemogenic transformation.2,4,5 Preclinical evidence shows that the disruption of fusion and wild-type menin-MLL interactions inhibits leukemic cell proliferation and restores terminal differentiation of MLL-rearranged and nucleophosmin 1 (NPM1)–mutated leukemic cells.1,6 (NCT04988555).

About Sumitomo Dainippon Pharma Oncology

Sumitomo Dainippon Pharma Oncology, Inc., is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. As a global oncology organization with teams in the U.S. and Japan, SDP Oncology is relentlessly committed to advancing purposeful science by transforming new discoveries into meaningful treatments for patients with cancer. SDP Oncology’s robust and diverse pipeline of preclinical and advanced-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 Dainippon Pharma

Sumitomo Dainippon Pharma is among the top-10 listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and other Asian countries. Sumitomo Dainippon Pharma aims to create innovative pharmaceutical products in the Psychiatry & Neurology area, the Oncology area and Regenerative medicine/Cell therapy field, which have been designated as the focus therapeutic areas. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 7,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma 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. Slany RK. When epigenetics kills: MLL fusion proteins in leukemia. Hematol Oncol. 2005;1-9. doi: 10.1002/hon.739.
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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