PharmAust trial heralds new era of hope for motor neurone disease patients

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By Colin Hay - 
PharmAust ASX PAA biotechnology motor neurone disease Orphan Drug Designation

Perth-headquartered clinical-stage biotechnology company, PharmAust (ASX: PAA), has hit another milestone in the assessment of its potential motor neurone disease (MND) treatment monepantel (MPL).

The company reports that all patients have now completed its Phase 1 MEND study of MPL, with top-line results expected to be released in the first quarter of 2024.

Chief executive officer Dr Michael Thurn said that, to date, there have been no seriously adverse treatment-related events.

“Excitement is growing, knowing that we are only months away from releasing top-line data.”

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

“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.”

Major global impact

MND – also known by the term amyotrophic lateral sclerosis (ALS) – is a rare condition that progressively damages parts of the nervous system. This leads to muscle weakness, often with visible wasting.

At this stage, the disease has no cure and no effective treatment to reverse its progression. Independent studies have shown that one-third of patients die within 12 months of initial diagnosis.

According to the International Alliance of ALS/MND Associations, the disease affects more than 350,000 people globally and kills more than 100,000 people yearly.

The disease is invariably fatal, with the average life expectancy of someone with MND being around 27 months.

The MND/ALS addressable market is estimated to be around $5.4 billion per annum.

Phase 1 study making progress

PharmAust’s Phase 1 MEND study involves two cohorts of six patients each, progressively receiving higher dose levels of MPL in a staggered design approach over time.

The open-label, multicentre study involved 12 patients with MND/ALS to determine the recommended Phase 2 dose, based on safety and preliminary efficacy.

Each dosage level of MPL is subject to meeting set safety criteria, governed by a safety monitoring committee. The highest dose evaluated was 10mg/kg.

To date, only three adverse events possibly related to treatment with MPL have been recorded, while there have been no reports of difficulty in swallowing tablets or patients needing assistance with breathing, which are common clinical signs of late-stage disease progression.

Ongoing trial plans

PharmAust says all patients are continuing treatment with MPL under a compassionate-use program and will be invited to participate in a 12-month open-label extension (OLE) study due to commence in or around January 2024.

PharmAust has now successfully submitted for approval the protocol for the 12-month OLE study to the Human Research Ethics Committee.

The company has also applied for an orphan drug designation with the US Food and Drug Administration and remains on track to commence a Phase 2 study for MND/ALS in H1 CY24.

The study is supported by a drug development grant of $881,085 from the independent not-for-profit, MND research organisation FightMND.