• PharmAust announces first patient dosed in 12-month OLE study for patients with MND/ALS
  • All 12 patients from Phase 1 MEND study expected to be enrolled after meeting minimum requirement
  • Analysis shows a 100% survival rate among the 12 Phase 1 MEND patients without treatment less than 1 in 1000.


Special Report: PharmAust has begun an open-label extension (OLE) study for monepantel (MPL) in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS) at Calvary Health Care Bethlehem in Melbourne as a statistical review shows the drug “defied the odds”.

Clinical-stage biotech PharmAust (ASX:PAA) says the first patient has been dosed in the 12-month OLE study for patients with MND/ALS – with all 12 patients from the Phase 1 MEND Study expected to be enrolled after meeting the minimum requirement of being able to swallow.

The 12-month OLE study is set to explore the prolonged safety, tolerability and effectiveness of MPL in patients with MND/ALS.

The OLE study is being held at two sites in Australia – Calvary Health Care Bethlehem under the supervision of Associate Professor Susan Mathers and Macquarie University under Professor Dominic Rowe.


Building on Phase I MEND study

In December 2023, PAA announced completion of the Phase 1 MEND study, involving two cohorts of six patients, each progressively receiving higher dose levels of MPL in a staggered design approach over time.

The first group of six patients enrolled in the Phase 1 MEND Study have all received treatment with MPL for more than 15 months now.

Top-line results of the Phase 1 trial are on track to be released before the end of this current quarter.

All 12 patients have elected to continue treatment with MPL through a compassionate-use program overseen by their treating physician, either Mathers or Rowe.

Patients will receive a daily dose of 10 mg/kg body weight of MPL for an additional 12 months during the OLE study, to further test if the drug safely reduces disease-
associated protein accumulation in motor neurons and provide therapeutic benefits.


‘Defied the odds’

PAA has also unveiled significant findings from a statistical review indicating a notable survival benefit.

The review, undertaken by statistical experts partner Berry Consultants, found if 12 similar MND patients went untreated, the likelihood of any surviving is less than 0.1%, or 1 in 1000.

To assess the likelihood of a 100% survival rate among the 12 Phase 1 MEND patients without treatment, the statistical experts used baseline survival rates derived from historical control data within the Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database.

The PRO-ACT database is the largest publicly available repository of merged ALS clinical study data.

After adjusting for differing diagnosis durations, Berry’s analysis involved comparing patients in the PRO-ACT database with similar characteristics to those in PAA’s Phase 1 study.

Blue Ocean Equities says MPL is showing great promise in treating a uniformly fatal disease, lacking any cure or effective treatment to halt its advancement.

Figures from the International Alliance of ALS/MND Associations shows MND impacts over 350,000 individuals worldwide, resulting in more than 100,000 annual fatalities.

On average, individuals diagnosed with MND can expect a life expectancy of ~27 months, with independent research indicating a third of patients succumb within the first 12 months after diagnosis.

“Looking at it another way, PAA has defied the odds as not one of the patients involved in their Phase 1 clinical trial for MND has passed away,” Blue Ocean Equities says.

“The fact that all 12 patients have maintained their ability to swallow is a great data point.”

Blue Ocean Equities says to put this into context a recently FDA-approved drug for ALS/MND, called RELYVRIO, showed a survival rate of around 75% in its 48-week trial.


Significant milestone for PAA

Mathers expressed her gratitude to all participants and their families for taking part in the study.

“Everyone is keen to join the OLE study, working with us to understand the longer-term effects and possible benefits of monepantel in MND/ALS,” she says.

PAA chief executive Dr Michael Thurn says initiation of the OLE study is a significant milestone for PAA and the 12 patients who began their treatment journey with monepantel in October 2022.

“It is remarkable and satisfying to know that all 12 patients are still alive  and capable of participating in this important extension study,” he says.

“The survival statistics based on these 12 patients are extremely encouraging for the company and the wider patient population with MND/ALS.

“This provides an exciting backdrop ahead of the release of the top-line data from the recently completed Phase 1 MEND study, due by the end of February 2024.”


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.