PharmAust Limited will proceed with a new Phase I clinical trial in humans to assess monepantel (MPL) in patients with amyotrophic lateral sclerosis/motor neurone disease (ALS/MND). This trial will be funded and conducted in collaboration with FightMND, Calvary HealthCare Bethlehem (Melbourne) and Macquarie University (Sydney). ALS/MND is a group of diseases that affect nerve cells controlling vital everyday functions including movement and breathing. ALS/MND is rare and invariably fatal (~140,000 new patients worldwide per annum) and imposes a high burden on patients, their families and carers, and carries substantial socio-economic costs. Two Australians will be diagnosed with MND every day with a life-time risk of developing MND of 1 in 300. The average life expectancy with MND is just 27 months. There is a great need for better treatments to prolong life and improve therapy. PharmAust has already shown in its preclinical programs that MPL has the potential to activate molecular pathways relevant to the treatment of MND. If effective, MPL would reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem. Furthermore, there are a number of surrogate clinical endpoints that will also be determined during the trial. For the purpose, PharmAust is developing and manufacturing a bespoke monepantel tablet for the trial. Monash Health Human Research Ethics Committee (HREC) has approved the monepantel (MPL) clinical trial protocol.