The Phase I clinical trial of lead drug Monenpantel in humans is an exciting development after earlier announced encouraging pre-clinical studies.

Clinical stage oncology company, PharmAust Limited (ASX:PAA), is set to commence the Phase I clinical trial on its lead drug Monepantel, for  people with motor neurone diseases (MND).

The study will be conducted following a trial protocol approval from the Monash Health Human Research Ethics Committee, and a $900k funding from FightMND.

Founded in 2014 by former AFL player and coach Neale Daniher,  FightMND is a charity established with the purpose of finding effective treatments and a cure for MND.

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 Motor Neurone Disease.”

It will be conducted in collaboration with the Calvary HealthCare Bethlehem in Melbourne, and Macquarie University in Sydney.

It will look specifically into patients with Amyotrophic Lateral Sclerosis/ Motor Neurone Disease (ALS/MND) – a condition that became widely known following the affliction of renowned physicist, Stephen Hawking.

The ALS/MND disease

ALS/MND is the name given to a group of diseases that affect nerve cells controlling our everyday movements including breathing.

Two Australians will be diagnosed with MND every day with a life-time risk of developing MND of 1 in 300.

The disease is rare and invariably fatal, with the average life expectancy of someone who has MND being just 27 months.

It has a terribly high burden on patients, family and carers, along with socioeconomic consequences.​

Current best treatment options attempt to control the disease progression and manage symptoms, but offer no cure and are very expensive at the same time.

The aim and use of Monepantel (MPL) is to provide a better treatment with positive therapeutic impact, by ameliorating the ALS/MNS disease progression.

In pre-clinical programs, PharmAust has already shown that MPL could activate molecular pathways relevant to its development as a MND therapeutic drug. If effective, MPL would reduce the rate of degeneration and loss of motor neurons in the anterior horns and motor nuclei of the brainstem.

The Phase I clinical trial

The Phase I clinical trial is designed to test the safety of using MPL in 12 individuals living with ALS/MND.

The patients will undergo dosing according to a conventional dose escalation design, with each level of the dose escalation lasting 28 days.

Measures of efficacy will also be included in the trial, so it can be extended into a Phase II setting when appropriate.

As announced in September last year, FightMND awarded PharmAust, along with Principal Investigators Dr Susan Mathers of Calvary HealthCare Bethlehem and Professor Dominic Rowe of Macquarie University, a total sum of  $881,085 to undertake this trial.

To promote the partnership and spread public awareness about the disease, FlightMND will once again launch its Big Freeze campaign during the AFL game between Melbourne and Collingwood at the MCG on the Queen’s Birthday June 14 holiday.

This is where celebrities and ex AFL players slide into an ice bath, which usually raises around $10 million every year.

Since 2014, the charity has  committed more than $48m to help find a cure for MND.

Dr Bec Sheean, Research Director at FightMND commented, “FightMND is pleased to be supporting this trial for MND patients in Australia. We look forward to the results and outcomes of this Phase I trial, in the hope that promising results will support the progression of monepantel to a Phase II trial and beyond.”

Ethical approval for the trial has been given by the Monash Health HREC, and is currently awaiting the finalisation of the availability and location of laboratories.

The trial is anticipated to commence in Q4 2021.

Orphan Drug Designation

PharmAust stated that it expects 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.

Major health authorities have long recognised the need to support organisations to develop interventions for the unmet needs under orphan or rare disease programs. Incentives include:

  • R&D grants;
  • reduced fees for scientific advice;
  • protocol assistance and regulatory activities and licences;
  • market exclusivity; and
  • provisions for accelerating authorisation.

In the US and Europe for instance, when an orphan drug designation (ODD) is granted, a medicine can have market exclusivity for up to 7 years and 10 years, respectively.

Over the past 20 years or so in Australia, the application fees for initial registration (via the TGA) and government reimbursement (via the PBS) for ODD medicines, whether new molecules or established molecules to treat rare conditions, have been waived. There is also the prospect that the benefit-risk profile promotes earlier and expedited authorisation.

This article was developed in collaboration with PharmAust, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.