CNC-01

CNC-01 is a novel, orally available small molecule dual inhibitor of Signal Transducer and Activator of Transcription 5 (STAT5) and Signal Transducer and Activator of Transcription 3 (STAT3). Developed by C&C Research Laboratories, it is being investigated for the treatment of acute myeloid leukemia (AML), particularly cases resistant to FLT3 inhibitors and venetoclax. CNC-01 directly binds to STAT5 and STAT3, inhibiting their phosphorylation (pY694-STAT5 and pY705-STAT3) and subsequent pro-tumorigenic transcriptional activity. Preclinical studies have demonstrated its ability to overcome resistance mediated by FLT3-TKD mutations, such as the F691L gatekeeper mutation, and show synergy with venetoclax.