On April 14, 2025 Sumitomo Pharma America, Inc. (SMPA) reported it has entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), part of the U.S. National Institutes of Health (NIH), to evaluate enzomenib, an investigational, oral, small molecule designed to inhibit the menin and KMT2A protein interaction present in certain difficult to treat cancers (Press release, Sumitomo Pharmaceuticals, APR 14, 2025, View Source [SID1234651916]).

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This unique partnership aims to harness the combined expertise of SMPA and the NCI to further expand the scientific evaluation of enzomenib as a potential therapeutic and explore additional cancers to address unmet patient needs.

Enzomenib is an investigational, oral, small-molecule currently being clinically evaluated by SMPA in Phase 1/2 clinical trials for relapsed or refractory acute leukemia. Enzomenib is designed to target the menin and mixed-lineage leukemia protein interaction, a key interaction for acute leukemia and other cancer cell growth. In preclinical studies, enzomenib demonstrated selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations – cancers with particularly high mortality rates and limited treatment options.1,3

As part of the collaboration, enzomenib will be explored in MyeloMATCH (Myeloid Malignancies Molecular Analysis for Therapy Choice), an NCI precision medicine clinical trial designed to improve treatment outcomes for individuals diagnosed with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). Enzomenib will also be explored with other important NCI programs.

"We are highly encouraged by the preliminary data showing promising clinical activity of enzomenib in patients with relapsed/refractory acute leukemia, and with the support and partnership of the National Cancer Institute, we are poised to continue this positive momentum by expanding exploration of its application across additional cancers," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "We’re honored to partner with the National Cancer Institute whose scientific expertise, resources and commitment to advancing oncological research will enable an even broader pursuit of scientific innovation on behalf of patients with urgent and unmet needs."

About Enzomenib
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and mixed-lineage leukemia (MLL) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.1,2 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A (MLL) rearrangements or NPM1 mutations.1,3 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1, and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with MLL rearrangements and NPM1 mutation.4,5 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose escalation/dose expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with MLLr or NPM1m in September 2024.