Sumitomo Pharma Oncology Receives Orphan Drug Designation for DSP-0390, an Investigational Emopamil-binding Protein (EBP) Inhibitor for the Treatment of Brain Cancer

Jul 19, 2022

Posted by Sumitomo Pharma Oncology, Inc.

CAMBRIDGE, Mass., July 18, 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-0390, an investigational emopamil-binding protein (EBP) inhibitor for the treatment of brain cancer.


“This designation for DSP-0390 underscores the profound need for novel brain cancer treatment options,” said Patricia S. Andrews, CEO and Global Head of Oncology, Sumitomo Pharma Oncology, Inc. “We are excited to contribute to advancing critical research in brain cancer.”  


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. Brain cancer refers to brain tumors, made up of abnormal growth of cells in the brain, that are malignant. The exact cause of most brain cancer is unknown.1


“DSP-0390 is an emopamil-binding protein (EBP) inhibitor that mediates de novo cholesterol synthesis for cell membrane structure and signaling, enabling aberrant growth of tumors,”2-5 detailed Jatin J. Shah, M.D., Chief Medical Officer of Sumitomo Pharma Oncology, Inc. “Notably, in patients with glioblastoma multiforme (GBM), a form of high-grade glioma, increased de novo cholesterol synthesis is correlated with poor survival and preclinical evidence has shown that DSP-0390 has cytotoxic activity against GBM cell lines.”6


DSP-0390 is currently being evaluated in a Phase 1 clinical trial to evaluate the safety and efficacy of DSP-0390 in patients with recurrent, high-grade glioma, which is being conducted in the United States and Japan. To learn more about the study and eligibility for enrollment, visit (NCT05023551).


This is the second recently announced Orphan Drug Designations from SMP Oncology. TP-3654, the company’s proprietary investigational oral inhibitor of PIM kinases, was also granted Orphan Drug Designation for the treatment of myelofibrosis (NCT04176198).


About DSP-0390

DSP-0390 is an investigational emopamil-binding protein (EBP) inhibitor. EBP is an endoplasmic reticulum membrane protein involved in cholesterol biosynthesis.2 DSP-0390 has shown cytotoxic activity against GBM, colorectal cancer, and triple-negative breast cancer in vitro. DSP-0390 is currently being evaluated in a Phase 1, interventional clinical trial to evaluate the safety and efficacy of DSP-0390 in patients with recurrent high-grade glioma (NCT05023551).


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 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 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. Brain cancer. Updated April 21, 2022. Accessed May 31, 2022.


2. Long T, Hassan A, Thompson BM, McDonald JG, Wang J, LI X. Structural basis for human sterol isomerase in cholesterol biosynthesis and multidrug recognition. Nat Commun. 2019;10(1):2452.


3. Yang C, McDonald JG, Patel A, et al. Sterol intermediates from cholesterol biosynthetic pathway as liver X receptor ligands. J Biol Chem. 2006;281(38):27816-27826.


4. Dong F, Mo Z, Eid W, Courtney KC, Zha X. Akt inhibition promotes ABCA1-mediated cholesterol efflux to apoA-1 through suppressing mTORC1. PLOS ONE. 2014;9(11):e113789.


5. Segala G, David M, de Medina P, et al. Dendrogenin A drives LXR to trigger lethal autophagy in cancers. Nat Commun. 2017;8(1):1903.


6. Kambach DM, Halim AG, Cauer AS, et al. Disabled cell density sensing leads to dysregulated cholesterol synthesis in glioblastoma. Oncotarget. 2017;8(9):14860-14875.

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