NCI Enters Research Cooperative Agreement to Advance Oral Agent for Acute Leukemia, Other Cancers

A Cooperative Research and Development Agreement (CRADA) has begun between Sumitomo Pharma America, Inc (SMPA), and the US National Cancer Institute (NCI) to bolster clinical research on enzomenib for acute leukemia and other cancers. SMPA, the US arm of the Japan-based global pharmaceutical company Sumitomo Pharma Co, Ltd, announced the new collaboration in a press release.

“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,” stated Jatin Shah, MD, chief medical officer, oncology, of SMPA.

Enzomenib, an orally administered investigational small molecule agent, inhibits the interaction between menin and mixed-lineage leukemia (MLL) proteins behind tumor cell growth in acute leukemia and other cancers. In preclinical studies, the agent has performed selective growth inhibition in human acute leukemia cell lines that feature KMT2A (MLL) rearrangements or NPM1 mutations.

SMPA is currently conducting a phase 1/2 dose-escalation/dose-expansion study to evaluate the efficacy and safety of enzomenib for relapsed or refractory acute leukemia. As part of the CRADA, enzomenib will be included in the NCI’s Myeloid Malignancies Molecular Analysis for Therapy Choice (MyeloMATCH) precision medicine clinical trial. This trial will evaluate the agent in acute myeloid leukemia (AML) and myelodysplastic syndromes.

Regarding the agent’s regulatory status, in the US, enzomenib received Orphan Drug Designation from the FDA in June 2022 for use in AML and Fast Track Designation in June 2024 for relapsed or refractory AML with an MLL rearrangement or NPM1 mutation. In Japan, it received Orphan Drug Designation in September 2024 from the Pharmaceuticals and Medical Devices Agency (PMDA) for relapsed or refractory AML with MLL rearrangement or NPM1 mutation.

Reference

Sumitomo Pharma America enters into a Cooperative Research and Development Agreement with the National Cancer Institute to advance clinical understanding of enzomenib. Press release. PR Newswire. April 14, 2025. Accessed April 21, 2025. https://www.prnewswire.com/news-releases/sumitomo-pharma-america-enters-into-a-cooperative-research-and-development-agreement-with-the-national-cancer-institute-to-advance-clinical-understanding-of-enzomenib-302426994.html