AIT-102

AIT-102 is a proprietary small molecule analogue of the natural product mithramycin, currently under development by OrphAI Therapeutics (formerly AI Therapeutics). It is designed to treat tumors characterized by SWI/SNF mutations or transcriptional dysregulation. AIT-102 acts as an Sp1 transcription factor inhibitor by binding to the minor groove of DNA at GC-rich regions. This mechanism interferes with the binding of several key oncogenic transcription factors, including MYC, SP1, HIF-1, and AP-1, thereby disrupting the "transcriptional addiction" that fuels cancer cell survival and proliferation. Preclinical data indicate that AIT-102 is significantly less toxic (25-40 times) than its parent compound, mithramycin, while maintaining potent anti-tumor activity across various indications such as colorectal cancer, triple-negative breast cancer, head and neck cancer, and melanoma.

Other names

EC-8042EC8042EC 8042