The Phase 1/2 trial will study the safety and efficacy of PharmAust’s lead drug monepantel in treating people with MDS or Lou Gehrig’s disease.

Clinical stage biotech company PharmAust (ASX:PAA) has made the first clinical step towards progressing its Motor Neurone Disease (MND) Phase 1/2 Trial.

The company has  dosed its first patient in the trial, which will be studying the effects of monepantel (MPL) in people living with the MND disease.

In addition, ethics approval has been granted to make a few small amendments to the Clinical Study Protocol, in order to refine the study design and accelerate recruitment.

Apart from  testing the safety and tolerability of MPL, the Phase 1/2 trial will also look for signs that MPL can slow the progression of MND.

This data, with concurrent animal studies, will determine whether MPL should proceed to larger Phase 2 studies.

The trial is being overseen by Dr Susan Mathers of Calvary Health Care, Bethlehem, Melbourne and includes a second trial site headed by Professor Dominic Rowe of the Centre for MND Research Faculty of Medicine and Health Research at Macquarie University, Sydney.


Fighting a global problem

Motor neurone disease (MND), sometimes called amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, is a global problem.

The ALS/MND addressable market is US$3.6Bn with Riluzole already reaching ~US$1Bn in annual sales.

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 of the world’s population, and kills more than 100,000 people every year.

It became widely known following the affliction of the late renowned physicist, Stephen Hawking.

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

Pre-clinical studies have shown that MPL can slow disease progression in MND models by clearing harmful materials in a motor neuron that stick together and make them unwell.

This Phase1/2 study is being funded by a commitment of $881,085 made by FightMND, the largest independent funder of MND research in Australia.

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

The funding agreement provides that PharmAust shall own all intellectual property generated from the trial.

The third instalment of $173k is now payable upon recruitment of the first patient.

Further instalments for a total of $881k will be paid by FightMND to PharmAust as clinical trial milestones are met.


Orphan Drug Designation

With success in the clinic, PharmAust expects that in due course MPL could 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.


Expedited study into Phase 2 COVID trial

PharmAust is also relying on the MND trial to provide the important Phase 1 pharmacokinetic (PK) data for both the MND and COVID-19 trials.

This will allow PharmAust to undertake a Phase 2 trial in COVID-19, rather than a Phase 1 study, which will facilitate faster recruitment as the company has been advised by the CRO that COVID-19 infected patients generally prefer participating in a Phase 2 study.

From a timing viewpoint, the Phase 2 will now take place 6-9 months earlier than it would have had PharmAust conducted a Phase 1 prior. From a financial viewpoint, an expedited study into Phase 2 will also benefit PharmAust, saving it of around $1.5 million.


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.