An investigational epigenetic therapy, NTX-301, has shown encouraging results against treatment-resistant acute myeloid leukemia (AML) in preclinical studies from The University of Texas MD Anderson Cancer Center, according to a press release dated July 15, 2026.
The research, published in Nature Communications, discovered that NTX-301 targets the Hippo signaling pathway, which is often dysregulated in AML. The drug is a DNMT1 inhibitor that reactivates tumor suppressor genes.
In laboratory models, NTX-301 demonstrated significant anti-leukemic activity, particularly in AML cells resistant to standard therapies like venetoclax. The study suggests a potential new treatment avenue for patients with limited options.
Further clinical trials are needed to confirm these findings in humans. The research was led by Dr. Marina Konopleva and colleagues at MD Anderson.
