PharmAust has announced that all 12 patients in Cohort 3 of its Phase-1/2 clinical trial of its lead drug candidate monepantel (MPL) in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS) have now received an initial, escalated dose.

The Phase-1/2 clinical study aims to determine the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL in individuals living with MND.

The trial is open label and comprises four cohorts with escalating MPL doses.

So far, the MPL tablets have been well tolerated by all patients in the trial and no Serious Adverse Events were observed, implying the drug has a good safety profile – with the Safety Monitoring Committee continuing to assess safety and efficacy for each dosage.

PharmAust (ASX:PAA) expects to proceed to Phase-2 of the trial with favourable efficacy biomarker results under the interim analysis.

Biomarkers and pharmacodynamics evaluation

As previously announced, interim analysis of biomarkers and pharmacodynamics through the current Phase-1/2 study is well underway, to explore how MPL is acting on the mTOR signalling pathway in MND patients to slow disease progression.

“It is hoped that biomarker analysis will confirm MPL’s ability to cross the blood-brain barrier and to aid in clearing protein aggregation that is the hallmark of neurodegenerative diseases through the induction of autophagy in nerve cells,” the company said.

Biomarker research in MND has led to the identification of the protein p75 ECD in urine, demonstrating people with MND show significantly higher levels of that protein compared to those without MND.

The p75 ECD study results have now been received from Flinders University in Adelaide and will be formally interpreted by a pharmacologist and will be announced as soon as possible.

An analysis being conducted at the Florey Institute in Melbourne evaluating p-RPS6KB1 and p- EIF4EBP1 is still pending.

Further, highly specialised testing of Neurofilament Light Chain (NfL) is being conducted at the University of Tasmania which is expected in the coming weeks.




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


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