Drug intelligence / Profile preview

AU-409

Development stage
Phase 1
Lead developer
Auransa
Modality
Nucleic Acid Therapeutics, Small Molecules
Administration
Oral
01

Overview

AU-409 is a novel, orally bioavailable small molecule drug candidate developed for the treatment of hepatocellular carcinoma (HCC) and other cancers. It functions as an RNA transcription modulator, specifically altering the transcription of certain genes involved in cancer cell proliferation. The mechanism of action is distinct from currently approved drugs for HCC, such as tyrosine kinase inhibitors. AU-409 was discovered using Auransa's proprietary AI-driven SMarTR Engine platform and has demonstrated promising antitumor activity in preclinical models and patient-derived xenografts. The drug is currently being evaluated in Phase 1 clinical trials for advanced primary liver cancers and solid tumors with liver-predominant metastases[1][2][3][4][5][6][7].

Other names
RNA transcription modulator AU-409
02

Targets

DNA

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