PharmAust (ASX:PAA) is a clinical-stage biotechnology company developing new therapeutics for human and animal health applications. The company is repurposing monepantel for the treatment of cancer in dogs and neurodegenerative diseases in humans.

Monepantel is an approved veterinary product for the treatment of worm infestations in sheep. In mammalian cells, monepantel is a potent inhibitor of the mTOR pathway. The mTOR pathway plays a central role in cell growth and proliferation of cancer cells and in degenerating neurons. The mTOR pathway regulates the cellular “cleaning process”, where toxic protein is broken down into macromolecules to be reused. This autophagic process is disrupted in in most neurodegenerative diseases, including motor neurone disease.

PharmAust has an extensive patent portfolio with multiple ‘methods of use’ patents granted and filed covering the use of monepantel as a novel anti-cancer agent and for the treatment of neurodegenerative diseases.