PharmAust continues to progress the use of its lead drug candidate monepantel (MPL) in motor neurone disease closer to the Phase 2 clinical trial with the successful completion of the third patient cohort.

With the successful completion of dosing for all six patients in the cohort over 28 days, the company can now mark the milestone of having all participants in the first, second and third dosing levels – with each level having a higher dosage than the previous level – having tolerated MPL well.

Importantly for PharmAust (ASX:PAA), all trial participants – including five participants who have now surpassed the 9-month-mark on MPL without any safety issues – have elected to continue on the MPL treatment.

Subject to Safety Committee approval, the company will continue with MPL dose escalation for Cohort 4 to determine the optimum dose whilst safety and efficacy data from the trial will also guide other human Phase 2 clinical trials such as for the treatment of cancer.

More early efficacy data arriving

Meanwhile, the company has noted that it will soon have another early data point into the efficacy of MPL in treating MND/ALS (Amyotrophic Lateral Sclerosis).

Results from the highly specialised testing of Neurofilament Light Chain (NfL) – a biomarker for ALS – are expected in the coming weeks with PharmAust identifying another laboratory that can process the data as a potential option if the current laboratory is unable to fix a key piece of equipment in a timely fashion.

It recently received urinary biomarker data showing that even participants in Cohort 1 of its Phase 1 trial, who are on the lowest dosage of MPL, have significant reduced levels of p75ECD – a biomarker with predictive value in Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS) progression.

This in turn followed received preliminary data indicating the suppression of inflammation that may be responsible for disease progression.

Five of the seven participants enrolled in Cohort 1 had decreased B1 protein levels in PBMCs whilst six of seven participants have decreased P1 protein levels in PBMCs, which the company said was indicative of successful targeting of the mTOR pathway in the blood of people with MND/ALS.

Promising treatment for MND

It may still be early days, but the signs certainly seem to point to MPL’s potential to significantly improve the quality of life for the more than 350,000 people affected by the disease globally.

According to the International Alliance of ALS/MND Associations, MND is invariably fatal with the average life expectancy of someone who has MND being around 27 months whilst current treatments only prolong survival by up to four months.

More than 100,000 people die from the disease annually.

The disease is progressive, meaning the symptoms get worse over time, and there is no cure or treatment to reverse its progression.

That all patients dosed with MPL have displayed no signs of material adverse events and appear “stable” to date is potentially significant.

Being able to treat MND/ALS opens up a significant market when combined with the potential to secure orphan drug designation from the US Food and Drug Administration that could in turn provide a number of financial and supportive benefits.

The MND/ALS addressable market is US$3.6 billion per annum, with existing third-party drug Riluzole already reaching ~US$1 billion in annual sales.

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




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.