Motor neurone disease (MND) charity FightMND has awarded oncology company PharmAust (ASX: PAA) a grant for a Phase 1 trial to examine the effects of its flagship drug candidate monepantel on the disease.
PharmAust will receive $881,085 for the human trial, which is anticipated to begin next year with patients to be recruited at Calvary Health Care Bethlehem in Melbourne and Macquarie University in Sydney.
The company has a three-pronged strategy to develop monepantel including treating cancer in dogs, a repurposed treatment for COVID-19 and as an anti-neurodegenerative disease drug.
In the last month, PharmAust outlined its research and development targets for the next year, which included ambitions to secure agreements to commercially develop monepantel-based products to treat MND and other central nervous system conditions.
MND, also known as Lou Gehrig Syndrome or Amyotrophic Lateral Sclerosis (ALS), is a rare and invariably fatal neurological disease that not only puts a terribly high burden on patients, families and carers, but carries a great socio-economic cost.
Current treatment options are expensive and can only attempt to control disease progression and manage symptoms, offering no cure.
How monepantel could help fight MND
According to PharmAust, monepantel is an MTOR inhibitor and induces autophagy, which is a mechanism relevant to the clearing of misprocessed and excessive intracellular protein in neural cells associated with the cause of MND/ALS.
The company has hypothesised that monepantel will benefit the health of patients’ neural cells by regulating problems associated with these proteins and thereby reduce MND-associated symptoms.
PharmAust noted a recent demonstration that mTOR inhibition through autophagy “synergises therapeutically with Riluzole”, the current ALS/MND standard-of-care.
“This means that success in this trial may provide greater options to patients through the use of either stand-alone or combinational therapeutics,” it stated.
FightMND is the leading independent funder of MND research in Australia. It was founded in 2014 by former AFL football player and coach Neale Daniher, who was diagnosed with the disease a year prior.
Its major annual fundraising campaign The Big Freeze, involving the sale of its iconic blue beanies, raised a record-breaking $11.9 million this year to help fund research into more effective treatments and a cure for MND.
FightMND’s grant will fund a dedicated monepantel tablet manufacturing program, trial participant assessment and monitoring during the trial, and an independent clinical research organisation to assist in trial management.
Phase 1 trial preparations
The Melbourne trial will be overseen by Dr Susan Mathers, while the Sydney site will be headed by Professor Dominic Rowe from the university’s Centre for Motor Neurone Disease Research Faculty of Medicine and Health Research.
PharmAust said preparations for the Phase 1 trial have already commenced and while remaining subject to approval from the Institutional Human Research Ethics Committees, recruitment is due to begin “as soon as possible in 2021”.
FightMND’s funding agreement enables PharmAust to own all intellectual property generated from the study.
PharmAust chief scientific officer Dr Richard Mollard said PharmAust is looking forward to assisting Dr Mathers and Dr Rowe in the trial.
“PharmAust acknowledges FightMND and its donors, in particular the patients, their families and friends and their support network,” he said.