BTSA1

BTSA1 is a pharmacologically optimized, orally active small-molecule direct activator of the proapoptotic BCL-2 family protein BAX, designed to bind with high affinity and specificity to the BAX N‑terminal “trigger site” and induce conformational activation, mitochondrial outer membrane permeabilization, and caspase-dependent apoptosis in primed cells. It was developed in academic collaboration led by researchers at Albert Einstein College of Medicine as a tool and preclinical candidate for targeting apoptosis-resistant cancers, particularly acute myeloid leukemia, where it potently induces BAX-mediated apoptosis in leukemia cell lines, patient samples, and xenograft models while sparing normal hematopoietic cells.[1][4][13] More recently, BTSA1 has also been characterized preclinically as a senolytic agent that selectively eliminates senescent myofibroblasts, converting minority mitochondrial outer membrane permeabilization to complete permeabilization to alleviate experimental pulmonary fibrosis, suggesting broader potential where apoptosis resistance and BAX inactivation underlie disease biology.[2][3]

Other names

BTSA1BTSA-1BTSA 1BAX Trigger Site Activator 1