PharmAust’s Phase 1/2 trial into the safety and efficacy of monepantel in treating people with MND or Lou Gehrig’s disease is well underway with recruitment of the first cohort of patients now completed. 

First treatment has thus far demonstrated that the company’s lead drug monepantel (MPL) is well tolerated by the six patients in the cohort.

PharmAust (ASX:PAA) added that subject to satisfying safety, progressive cohorts will receive escalating oral treatment doses of MPL tablets.

It expects to recruit patients at the next dosing level in the new year.

Evaluation of the safety and efficacy of MND (motor neurone disease) patients treated with rising doses of MPL will continue to enable the company to progress to a Phase 2 study.

This data will also facilitate Phase 2 trials of the drug in cancer and COVID 19 patients.

Phase 1/2 trial

The trial is being carried out at Calvary Health Care Bethlehem, Parkdale, Victoria and the Centre for Motor Neurone Disease Research, Faculty of Medicine and Health Research Macquarie University, Sydney, New South Wales.

It is aimed at determining the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in individuals living with MND.

The open label trial comprises a four week escalating dose of MPL.

Patients will be assessed for serious adverse effects, adverse effects, safety blood results, ECG, vital signs and pharmacokinetic results.

Additionally, the progressive elevation of MPL levels to determine safe dosing levels will be determined in serum after dosing.

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.

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.

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.




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.