Our lead clinical candidate, BTX-A51, is an oral small molecule, multi-kinase inhibitor designed to block a specific leukemic stem cell target (CK1α) as well as super enhancer targets (CDK7/CDK9) preventing transcription of key oncogenic genes. This therapeutic mechanism entails activation of p53 (an important tumor suppressor) and its sustained stabilization by super-enhancer shutdown of Mdm2 (a protein degrader of p53), in combination with transcriptional shutdown of leukemia oncogenes, such as Myc and Mcl1.
Blocking CKIα, CDK7, and CDK9 augments and synergistically stabilizes p53 promoting the rapid killing of leukemia cells as well as leukemic stem cells. Pre-clinical results have been published in a peer-reviewed Cell journal, demonstrating BTX-A51’s favorable efficacy in animals.
Our Phase 1 clinical trial of BTX-A51 is currently ongoing and recruiting patients.