PharmAust doses first patient with MPL in motor neurone disease trial

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By Lorna Nicholas - 
PharmAust ASX PAA FightMND Motor Neurone Disease Monepantel MND biotechnology

The phase 1/2 trial will test the safety and tolerability of MPL and look for signs that it can slow the progression of MND.


PharmAust (ASX: PAA) has dosed the first patient of its phase 1/2 trial evaluating its lead drug monepantel (MPL) in people suffering from motor neurone disease (MND).

The company has also secured ethics approval to make “small amendments” to the study, which will refine its design and accelerate recruitment.

With the first patient recruited and dosed for the trial, PharmAust is eligible for the third instalment of FightMND’s funding commitment.

FightMND is Australia’s largest independent funder of MND and previously committed $881,085 towards PharmAust’s phase 1/2 trial.

As part of the patient dosing milestone, FightMND will pay PharmAust $173,034 to assist with study costs under its commitment.

Calvary Health Care’s Dr Susan Mathers is overseeing PharmAust’s trial in Melbourne, Prof Dominic Rowe will head up a second trial site.

Prof Rowe is from the Centre for Motor Neurone Disease Research Faculty of Medicine and Research at Macquarie University in Sydney.

The trial will test the safety and tolerability of MPL and look for signs that it can slow the progression of MND.

Tablets made to current good manufacturing practices were shipped to Sydney and Melbourne for the trial in May.

The tablets were designed with a different shape and size to aid swallowing in MND sufferers.


PharmAust describes MPL as a novel, potent and safe inhibitor of the mTOR pathway. The mTOR pathway has “key influences” in cancer growth and neurodegenerative diseases.

In addition to evaluating MPL’s impact in MND, PharmAust is testing its anti-cancer effect in humans and canines.

Latest results from canine research have shown it can more than double the life expectancy of dogs with cancer.

At veterinary centres in Australia, New Zealand and the US, MPL is being evaluated in dogs with newly diagnosed B-cell lymphoma and have not undergone any prior cancer treatment.

To-date, results show MPL has effective anti-cancer activity during phase 2a and 2b trials, with minimal side effects.

“The combination of MPL with prednisolone, which has provided average extension of survival to these pet dogs of 16-24 weeks, more than doubles the life expectancy than standard of care (palliative steroid therapy) that typically provides for six-to-eight-week survival in association with a range of adverse events,” principal investigator Dr Kim Agnew noted.