The path is clearer for PharmAust to proceed with the Phase 2 clinical trial of its lead drug candidate monepantel (MPL) in motor neurone disease following the receipt of further positive results.

The company 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.

Importantly for PharmAust (ASX:PAA), the result provides preliminary evidence of MPL activity against motor neurone degradation and does not provide evidence of overall disease progression.

This is not the first set of evidence into the efficacy of MPL.

Earlier this month, the company 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.

Dosage to increase

As such, the company will continue with MPL dose escalation for Cohorts 3 and 4 to determine the optimum dose for Phase 2.

“We are delighted with the results. MND is a disease characterised by progressive, debilitating paralysis due to the loss of motor neurons in the cerebral cortex, brainstem and spinal cord and is fatal,” executive chairman Dr Roger Aston said.

“Therapeutic options are limited to Riluzole, which offers a modest 3-6 month survival extension.

“Baseline urinary p75ECD provides prognostic information and is the only biological fluid–based biomarker of disease progression.”

Results of highly specialised testing for Neurofilament Light Chain – a biomarker for ALS – that is being carried out at the University of Tasmania are expected soon.

The company has also proven the safety of MPL with data showing that all dosages are well tolerated to date.

Much needed treatment

A successful Phase-2 clinical trial has the 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.

For PharmAust, this is 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.




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.