PharmAust has dosed the first patient of the second cohort in its Phase 1/2 Motor Neurone Disease (MND) clinical trial mere weeks after securing approval to proceed with an escalated dose.

The patient will receive 4mg/kg of the company’s lead drug monepantel, which has thus far proved safe for human consumption and has been shown to slow disease progression in MND models in pre-clinical studies.

This is twice the dosage received by patients in the first cohort.

PharmAust (ASX:PAA) noted that the outcome of this rising dose study will be a key determinant in proceeding to a Phase 2 clinical trial.

Additionally, all six patients in Cohort 1 have elected to continue on MPL with the first patient now surpassing 140 days of treatment.

This continuation of dosing is essential as Continued Safety, pharmacokinetics and efficacy data from the trial will also be used to facilitate a Phase 2 study into the use of MPL in COVID 19 patients.

The company will now focus on enrolling a further five patients to complete Cohort 2. The outcome of this rising dose study will be a key determinant in proceeding to a phase 2 trial in MND.

PharmAust director, Sam Wright told Stockhead: “If we get the right effect on the surrogate markers, the Safety Committee will be proposing to move into Phase 2. As the markers in our previous human cancer trial at the Royal Adelaide Hospital changed favourably in a few days, we are hopeful this approach will accelerate the trial outcomes.”

Study objectives

The phase 1/2 clinical study is determining the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in MND sufferers.

The open label trial comprises a four week escalating dose of MPL and is overseen by Dr Susan Mathers of Calvary Health Care, Bethlehem, Statewide Progressive Neurological Disease Service, Melbourne.

The trial also includes a second site headed by Professor Dominic Rowe of the Centre for Motor Neurone Disease Research Faculty of Medicine and Health Research at Macquarie University, Sydney.

MND a multi-billion dollar market

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 is invariably fatal, with the average life expectancy of someone who has MND being just 27 months, and has no known cure.

The first approved drug treatment for the disease, called Riluzole, became available in 1995. Clinical trials with MND patients showed that Riluzole prolongs survival by around 3-4 months, mainly in those with difficulty swallowing. Riluzole does not reverse the damage already done to motor neurons, and persons taking the drug must be monitored for liver damage and other possible side effects.

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

Funding and orphan drug hopes

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

Should the trial be successful, PharmAust is hopeful of receiving orphan drug designation by the US FDA for the indication of motor neurone disease.

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, which will provide a number of financial and supportive benefits.




This article was developed in collaboration with PharmAust (ASX:PAA), 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.