Every day, two people are diagnosed in Australia with a form of motor neurone disease (MND). MND entails a group of diseases that attack your motor nerves, leaving your muscles to become weak and degenerate. According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills over 100,000 people every year.
The average life expectancy of someone who has an MND is just around two years. The addressable market for this disease is $5.33 billion per annum, with the drug Riluzole (also targeting motor neuron diseases) already reaching about $1.5 billion in annual sales.
Biotech company PharmAust (ASX: PAA) is aiming to tap into that market, too. The Company has completed dosing its first cohort of six patients in its Phase 1/2 clinical trial of its lead drug candidate monepantel (MPL) to treat Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).
After completing patient recruitment for treatment level 1 in December last year, the Company has reached day 29 of dosing the final patient in the first cohort. These patients were enrolled at two sites: Calvary Health Care Bethlehem, Victoria and the Centre for Motor Neurone Disease Research, Faculty of Medicine and Health Research at Macquarie University, Sydney.
In its preclinical programs, MPL showed the potential to activate molecular pathways that could aid in the treatment of MND. The drug could reduce the rate of degeneration and loss of motor neurons of the brainstem, thus slowing the onset of an MND. Still, there are many clinical outcomes yet to be discovered.
Things are looking up for the drug following the completion, given that all six patients from the first cohort have elected to continue on the MPL treatment by PharmAust.
The ongoing study is currently being funded by a commitment of $881,085 made by FightMND, an Australian MND foundation and the largest independent funder of MND research in Australia. The phase 1/2 clinical study is determining the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in MND sufferers.
It’s an open-label trial, meaning that both patients and researchers know what drug has been administered. The trial entails a four-week escalating dose of MPL. As of now, PharmAust has found that the tablets are well-tolerated by patients; the Safety Monitoring Committee will review data from each dosage level for safety and pharmacokinetics.
Researchers will now begin increasing MPL dosage to evaluate the efficacy of the drug. It will help understand safe dosing levels for the planned COVID trial in Phase 2. Currently, safe levels are determined in serum after dosing over 28 days.
Later this month, PharmAust will start recruiting patients for the next dosing level.
If all goes to plan, the Company hopes that MPL would receive the orphan drug designation by the FDA for MND. Since the approval is for drugs that treat rare diseases or conditions, FDA provides tax and other incentives that could benefit PharmAust financially.
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