AUS_001

AUS_001 is a preclinical small molecule anticancer agent being developed by Australis Pharmaceuticals (also known as Terra Australis Pharmaceuticals PTY LTD). Discovered in honeybee propolis from Kangaroo Island, South Australia, it functions as a microtubule-destabilizing agent that disrupts the structural support required for cell division and growth, triggering programmed cell death (apoptosis) in cancer cells. AUS_001 has demonstrated high potency against 24 different types of cancer in vitro and has confirmed efficacy in seven in vivo cancer models, including glioblastoma, pancreatic cancer, breast cancer, bladder cancer, kidney cancer, lung cancer, and melanoma. The drug is orally bioavailable, crosses the blood-brain barrier, and exhibits approximately 20-fold selectivity for cancer cells over healthy cells. In 2025, it received FDA Orphan Drug Designation for malignant gliomas and Rare Pediatric Disease Designation for pediatric-type diffuse high-grade gliomas. IND-enabling studies are currently underway as of 2025-2026.