Biotechnology player PharmAust has completed enrolment of all 12 patients in Cohorts 1 and 2 in its clinical trial of its drug monepantel (MPL) for Motor Neurone Disease/Amyotrophic Lateral Sclerosis (MND/ALS).

The study aims to determine the tolerability, safety, pharmacokinetics and preliminary efficacy of oral MPL and comprises four cohorts with escalating doses – with the tablets being well tolerated by patients so far.

Once the last patient of Cohort 2 has been dosed, PharmAust (ASX:PAA) will commence an interim analysis to evaluate potential indicatory changes in biomarkers and pharmacodynamics – the biochemical and physiological effect of a drug.

Analysing efficacy and quality of life

The company said it expects to proceed to Phase 2 with favourable efficacy biomarker results under the interim analysis.

The analysis will look at treatment-related changes from baseline in this safety, tolerability, pharmacokinetic and the efficacy study will include an analysis of functional rating scales, quality of life and cognitive assessment.

Indicatory evidence for MND severity and progression during the trial will also be examined.

PharmAust will also continue with the MPL dose escalation for Cohorts 3 and 4 during the interim trial analysis (subject to Safety Committee reviews) to determine the optimum dose level for the Phase 2 trial.

Subject to Safety Committee reviews, the patients from Cohort 1 will move to Cohort 3 and the patients from Cohort 2 will move to Cohort 4. That means the next stages of the trial will move at a rapid pace as there is no need to wait for new patients to be screened and recruited.

A promising treatment for MND

According to the International Alliance of ALS/MND Associations, MND affects over 350,000 people globally and kills more than 100,000 people every year with no cure.

The average life expectancy of someone who has MND is around 27 months, and the disease is progressive – meaning symptoms get worse over time.

The company demonstrated in its preclinical programs that MPL could potentially reduce the rate of degeneration, and notes that patients have been dosed with MPL for up to six months in the clinical trial with no signs of material adverse events and are “stable”.

With success in the clinic, PharmAust hopes that in due course MPL could receive orphan drug designation by the TGA and FDA for motor neurone disease. Such designations come with financial and supportive benefits.

It’s also worth noting the total MND/ALS addressable market is US$3.6 billion per annum with current ALS drug on the market Riluzole already reaching ~US$1 billion in annual sales.

Multiple avenues to a $1Bn drug

Not only does PharmAust have clinical trials underway in MND in humans and cancer in canines, the Company is set for further trials in human cancer to commence this calendar year. This provides PharmAust with multiple ‘shots on goal’ at a +$1Bn drug success.




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.