Clinical-stage biotechnology company PharmAust Limited (ASX:PAA) has elected to 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 our 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.
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.
PharmAust’s Chief Scientific Officer, Dr Richard Mollard, stated that the company expects that in due course MPL will receive orphan drug designation by the FDA for the indication of motor neurone disease. Such designations come with a number of financial and supportive benefits. The Orphan Drug Act provides for granting special status to a drug or biological product to treat a rare disease or condition upon request of a sponsor.
PharmAust is privileged and delighted to be working with Dr Mathers, Professor Rowe, FightMND, Monash Health and Macquarie University with the common aim of finding positive outcomes for people living with ALS/MND. PharmAust is especially thankful to FightMND and all its supporters who make this type of work possible,” Dr Mollard said.
Founded in 2014, FightMND was established in Australia with the purpose of finding effective treatments and ultimately a cure for Motor Neuron Disease (MND), also referred to as ALS or Lou Gehrig’s Disease. FightMND, with its vision of a world without MND, is the largest independent funder of MND research in Australia. What FightMND has done since 2014, is be the voice and the guiding star for Australians who want to fight “The Beast”. Integral to this vision is the determination to help facilitate the translation of the growing body of new knowledge about the disease into a cure for MND patients in Australia and abroad.