Monepantel (MPL) is an oral aminoacetonitrile (AAD) compound that modulates the mTOR pathway which is recognised as a key driver of many cancers.  The mTOR pathway also recognised as playing a role in the pathogenesis of diseases such as Alzheimer’s, Parkinson’s and various ageing disorders.

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/year.

MPL has been evaluated in a Phase 1 clinical trial in humans.  The principal endpoint was to assess the safety of 5mg/kg bw of liquid MPL as an anticancer drug in four patients with refractory metastatic cancer.

Analysis showed that MPL 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.