After securing ethics approval, PharmAust (ASX: PAA) is gearing up to begin a phase I clinical trial in the December quarter, which will assess its monepantel drug in patients with motor neurone disease.
Ethics approval from Monash Health gives PharmAust the ability to proceed with phase I trials assessing monepantel (MPL) in people suffering from amyotrophic lateral sclerosis/motor neurone disease (ALS/MND).
FightMND, Calvary HealthCare Bethlehem (Melbourne) and Macquarie University (Sydney) will collaborate on the trial, with FightMND kicking in $881,085 to facilitate the research.
Leading the trial are principal investigators Dr Susan Mathers from Calvary and Professor Dominic Rowe from Macquarie University.
The multi-centre open label trial will test the MPL’s safety in 12 people with ALS/MND.
Measures of efficacy are also included so the research can be extended into a phase II setting.
PharmAust’s chief scientific officer Dr Richard Mollard said the company was “privileged” and “delighted” to be working with experts 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 added.
Orphan drug designation
PharmAust is anticipating orphan drug designation “in due course” from the United States Food and Drug Administration for MPL in treating MND.
According to PharmAust the orphan designation will provide a number of financial and supportive benefits including tax credits, fee waivers and market exclusivity.
If granted, a benefit for PharmAust is it will be able to use MPL to treat MND upon a sponsor’s request.
ALS/MND diseases and preclinical research
ALS/MND is a group of diseases that affect the nerve cells controlling the human body’s every day vital functions including movement and breathing.
The disease is rare with about 140,000 people worldwide diagnosed each year. It imposes a high burden on patients and their families and is invariably fatal. The average life expectancy with MND is 27 months.
In Australia, two people will be diagnosed with MND every day. There remains a need for better treatments to prolong life and improve therapy.
PharmAust’s preclinical work with MPL has shown it has the potential to activate molecular pathways relevant to treatment of MND.
The company says if it is proved effective in clinical trials, MPL could reduce the rate of degeneration and loss of motor neurons.
In readiness for the upcoming phase I trial, PharmAust is developing and manufacturing a bespoke MPL tablet.