Sumitomo Pharma America Enters into Agreement with NCI to Advance Enzomenib in Hard-to-Treat Cancers
Tuesday, April 15, 2025
Sumitomo Pharma America has entered into a Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI).
Enzomenib is designed to block the interaction between menin and KMT2A (also known as MLL), a key driver in the development and growth of certain acute leukaemias. This interaction has been identified as a target for therapeutic intervention, particularly in cancers associated with KMT2A rearrangements or NPM1 mutations, which are known for their poor outcomes and limited treatment options.
Enzomenib has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of acute myeloid leukaemia, along with Fast Track Designation for use in patients with relapsed or refractory AML with MLL rearrangements or NPM1 mutations. In Japan, the Pharmaceuticals and Medical Devices Agency (PMDA) has also awarded Orphan Drug Designation for the same indication.
The collaboration will support further clinical research into enzomenib, an investigational oral small molecule, to better understand its potential in treating cancers that are currently difficult to manage.
Currently, enzomenib is being tested by SMPA in an ongoing Phase 1/2 clinical trial for patients with relapsed or refractory acute leukaemia. Preclinical data have shown that enzomenib can selectively inhibit the growth of human acute leukaemia cell lines with KMT2A rearrangements or NPM1 mutations. The molecule has also been found to influence gene expression by lowering levels of HOXA9 and MEIS1, while increasing CD11b, a marker linked to cell differentiation.
Through the CRADA, enzomenib will be studied further as part of the NCI's MyeloMATCH initiative, a precision medicine clinical trial aimed at improving treatments for acute myeloid leukaemia (AML) and myelodysplastic syndromes (MDS). The collaboration may also extend to other NCI programmes, expanding the investigation into additional cancer types.
This partnership brings together SMPA’s research efforts and the NCI’s scientific expertise, aiming to accelerate the development of new treatment options for patients facing urgent and unmet medical needs.
Source: sumitomo-pharma.com
