PharmAust is a clinical-stage biotechnology company developing new therapeutics for human health. The company is repurposing monepantel for the treatment of neurodegenerative diseases, starting with amyotrophic lateral sclerosis which is the most common form of motor neurone disease. ALS is a rare and incurable disease.
Monepantel is a potent inhibitor of the mTOR pathway which plays a central role in cell growth and proliferation 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 for the treatment of neurodegenerative diseases.
The company successfully completed a Phase 1 study in monepantel as a treatment for motor neurone disease (MND/ALS) with encouraging results, and anticipates starting a Phase 2 study in H2 2024.