Monepantel for Human Cancer

mTOR inhibitors have been approve to treat various human indications including renal cell carcinoma, breast cancer, mantle cell lymphoma and pancreatic neuroendocrine tumours.

Monepantel inhibits the mTOR pathway – a key driver of many cancers. mTOR inhibitors have been approved to treat various human indications including renal cell carcinoma, breast cancer, mantle cell lymphoma and pancreatic neuroendocrine tumours. The market for human mTOR inhibitors is valued at over $2 billion per year.

PharmAust has conducted a Phase 1 study of monepantel in human cancers. The principal endpoint was to assess the safety of 5mg/kg bw of liquid monepantel as an anticancer drug in four patients with refractory metastatic cancer.

Monepantel treatment was well-tolerated with only minor adverse events reported.  Pharmacodynamic analysis revealed treatment was associated a significant reduction in two key biomarkers associated with the disease.  Clinical assessment using Response Evaluation Criteria in Solid Tumours (RECIST 1.1) showed that of the four patients who completed the trial, three were categorised as having stable disease and one patient as having progressive disease.