• PharmAust completes Phase 1 MEND study for MPL in patients with MND/ALS
  • Top line results on track for release in Q1 2024
  • Company has applied for Orphan Drug Designation with the US FDA


Special Report: Excitement is brewing at biotech company PharmAust after it wrapped up the Phase 1 MEND study of the drug monepantel (MPL) in patients with motor neurone disease (MND/ALS).

The company is on track to release top line results from the study in Q1 2024 and has applied for an Orphan Drug Designation (ODD) with the US Food and Drug Administration (FDA), which incentivises the development of drugs or biological products to treat rare diseases or conditions by offering market exclusivity for several years and tax credits.

PharmAust’s (ASX:PAA) Phase 1 MEND study is an open-label, multicentre study involving 12 patients with MND/ALS – a nervous system disease that weakens muscles and impacts physical function.

It affects over 350,000 people globally and is invariably fatal with the average life expectancy of someone with MND/ALS being around 27 months.

The study design involves two cohorts of six patients, each progressively receiving higher dose levels of MPL — which could reduce the rate of degeneration and loss of motor neurons in the brainstem’s anterior horns and motor nuclei — in a staggered design approach over time.

Some patients have been treated for more than 13 months and none have withdrawn from the study with no deaths or significant adverse events to date.

Promisingly, the company noted in August that only one of the 12 patients showed an increase in Neurofilament Light Chain (Nfl) protein concentrations in their plasma.

This is significant as research suggests a correlation between Nfl increases and MND progression.

Success is likely to be hugely lucrative as the total MND/ALS addressable market is US$3.6bn per annum. Riluzole – a treatment that prolongs life on average by just 2-3 months – has already achieved annual peak sales of ~US$1bn.


“Excitement is growing”

With the completion of the Phase 1 MEND study, PAA notes that all patients, who are continuing to receive MPL under a compassionate use program, remain able to swallow and breathe unassisted.

All patients have also be invited to move to a 12-month open-label trial.

“Excitement is growing knowing that we are only months away from releasing top-line data,” chief executive officer Dr Michael Thurn said.

“We have several patients who have now received treatment with monepantel for 13 months or more, with the median treatment duration being 10.6 months for the 12 patients who participated in the study.

“Every month a patient continues on treatment brings enormous satisfaction and excitement around the potential benefit that monepantel may bring to patients, their families and caregivers, and of course the healthcare system as a whole.”

What’s of particular note is that the patients who were diagnosed 24 months before the trial have now survived for 34 months.

“This is longer than the “average survival” of 27 months and nearly matches the current survival-leading drug, which extends life by 9 months (36 months average survival),” Thurn told Stockhead.



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.