• PharmAust reports promising updates on MPL’s efficacy in treating MND/ALS
  • New data suggests MPL significantly reduces the risk of death by 91% in MND/ALS patients
  • The next step is the Open-Label Extension (OLE) study, where enrolment has just been completed


Special Report: New data suggests PharmAust’s MPL drug could potentially extend life expectancy and improve quality of life for those with MND/ALS.

PharmAust (ASX:PAA) jumped by almost +20% this morning after sharing some exciting updates about its ongoing research into Motor Neurone Disease (MND), also called Amyotrophic Lateral Sclerosis (ALS).

PAA announced that enrollment for the Open-Label Extension (OLE) study has now been completed.

This study looks at the long-term safety, tolerability, and effectiveness of PAA’s lead drug, monepantel (MPL), for people with MND/ALS.

PAA says that new and updated data provided by Berry Consultants reveals some promising news — patients who took MPL had a 91% lower risk of death from MND/ALS compared to ‘untreated matched-controls’.

This is significant and suggests that MPL could be a really effective treatment for MND/ALS.

PharmAust Managing Director, Dr Michael Thurn, said this result is a significant milestone for PharmAust.

The updated survival analysis conducted by Berry Consultants is extremely encouraging, as is the updated efficacy analysis that indicates MPL continued to slow the rate of disease progression in patients with MND/ALS.

“These results provide an exciting backdrop ahead of the anticipated commencement of the pivotal adaptive Phase 2/3 STRIKE study in H2 2024,” Dr. Thurn said.


More about the OLE Study

The OLE study is looking into how safe and effective MPL is for people who have MND/ALS.

This study was designed specifically for people who finished the earlier PharmAust Phase 1 MEND Study.

In the OLE study, those patients will take a daily dose of MPL for an extra 12 months to see how it affects them in the long run.

PAA has now completed enrolling patients for the study, and out of the 12 who finished the Phase 1 MEND study, 10 of them are now part of the OLE study.

Of the remaining two one passed away and the other couldn’t join the study because of issues with their liver enzymes. These liver enzyme problems weren’t caused by taking MPL, says PharmAust.

The OLE study involves two sites in Australia, Calvary Health Care Bethlehem, led by Associate Professor Susan Mathers, and Macquarie University, led by Professor Dominic Rowe.


Survival analysis: making sense of the data

A group of experts, including statistical consultants and PharmAust’s partner Berry Consultants, took a deep dive into the data to understand how MPL affects the survival of patients with MND/ALS.

They looked at data from people treated with MPL for different lengths of time, ranging from 11.3 to 19.7 months, with a median of 16.4 months.

To compare this with previous data, they used information from the PRO-ACT database, which is the largest publicly available repository of merged ALS clinical study data.

They made sure to match each MPL-treated patient with similar patients from the database based on factors such as where their disease started, how quickly their condition worsened before starting MPL, their condition at the start of the study, and how long they’ve had the disease.

To make the analysis stronger, Berry Consultants created two different sets of comparison data: one that assumed patients who hadn’t died were still alive, and one that only used data where they knew for sure if patients had died or not.

They also tried different ways to estimate the time of death for patients where this information was missing.

When they looked at all this data, they found interesting patterns.

Even when they were very careful with their analysis, they still saw big differences between patients treated with MPL and those from the PRO-ACT database.

In fact, using the most cautious approach, they found that MPL reduced the risk of death by a whopping 91%.

This means that MPL could be a game-changer for people with MND/ALS, potentially helping them live longer and better lives.


Understanding how MPL affects disease progression

Berry Consultants also took a closer look at how MPL impacts the rate at which ALS Functional Rating Score-Revised (ALSFRS-R) scores decline over time.

The ALSFRS-R is a tool used to measure the functional status of individuals with MND/ALS, assessing various aspects of daily functioning, including mobility, self-care, speech, and swallowing.

The consultants updated their analysis using data from the Phase 1 MEND study, including the baseline ALSFRS-R scores for patients who continued to the OLE Study.

Using a similar method to their survival analysis, they compared the change in ALSFRS-R scores over time between patients treated with MPL and those from the PRO-ACT database.

They found that the MPL treatment slowed down the rate of decline in ALSFRS-R scores.

Specifically, the change in slope was 0.40 ALSFRS-R points per month, resulting in an estimated rate of decline of -0.81 ALSFRS-R points per month.

This means that MPL slowed down the decline in ALSFRS-R scores by about 34%.

When they looked at different groups of patients separately, they found that MPL had varying effects.

For Cohort 1, disease progression was slowed by 23%, while for Cohort 2, it was slowed by 44%.

This suggests that MPL might have different effects on different groups of people with MND/ALS, but overall, it seems to slow down the progression of the disease, which is really promising news.

The new development today comes after PharmAust recently reappointed Dr Michael Thurn as managing director and CEO to lead its push in human neurodegenerative diseases.

At the same time, the company also appointed neuroscientist Dr Nicky Wallis as its chief scientific officer.

These appointments signify a strategic move towards advancing the company’s Phase 2/3 clinical trial of MPL in the treatment of MND/ALS, and positioning itself as a leader in neurodegenerative disease treatment.

NOW READ: PharmAust puts experienced hands back on its rudder



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.