Ann: PharmAust receives ethics approval for Phase I trial in MND, page-3

PharmAust receives ethics approval for Phase I trial ofmonepantel in motor neurone disease
● 12 trial subjects to test safety and some efficacy measures● Special tablets to be developed and manufactured for the tria
l● Trial to commence in CYQ4 2021
● Trial funded by FightMND Australia ($881,085)
● Protocol accepted and accessible on US NIH National Library of MedicineClincialTrials.gov website2

June 2021 – Perth, Australia: PharmAust Limited (ASXAA), a clinical-stage biotechnologycompany, 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 HealthCareBethlehem (Melbourne) and Macquarie University (Sydney).ALS/MND is a group of diseases that affect nerve cells controlling our vital everyday functionsincluding movement and breathing. ALS/MND is rare and invariably fatal (~140,000 newpatients worldwide per annum) and imposes a high burden on patients, their families andcarers, and carries substantial socio-economic costs. Two Australians will be diagnosed withMND every day with a life-time risk of developing MND of 1 in 300. The average life expectancywith MND is just 27 months. There is a great need for better treatments to prolong life andimprove therapy.PharmAust has already shown in its preclinical programs that MPL has the potential to activatemolecular pathways relevant to the treatment of MND. If effective, MPL would reduce the rateof degeneration and loss of motor neurons in the anterior horns and motor nuclei of thebrainstem. Furthermore, there are a number of surrogate clinical endpoints that will also bedetermined during the trial. For the purpose, PharmAust is developing and manufacturing abespoke monepantel tablet for the trial.Monash Health Human Research Ethics Committee (HREC) has approved the monepantel(MPL) clinical trial protocol.The study is a multi-centre open label trial entitled A Phase I Tolerability, Safety,Pharmacokinetics and Preliminary Efficacy Study of Oral Monepantel in Individuals with MotorNeurone Disease. It is designed to first test safety in 12 individuals living with ALS/MNDaccording to a conventional dose escalation design, with each level of the dose escalationlasting 28 days. Measures of efficacy are included in the trial design so that where appropriate,this trial can be extended into a Phase II setting. Details of the trial can be found atclinicaltrials.gov using the Identifier code: NCT04894240.PharmAust expects that in due course MPL will receive orphan drug designation by the FDAfor the indication of motor neurone disease. Such designations come with a number of financialand supportive benefits. The Orphan Drug Act provides for granting special status to a drug orbiological product to treat a rare disease or condition upon request of a sponsor.