PharmAust begins extended study on promising MND treatment following successful trial

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By Colin Hay - 
PharmAust ASX PAA biotech ALS MND motor neurone disease

Biotechnology company PharmAust (ASX: PAA) has dosed the first patient in an open-label extension (OLE) study follow-up to a program which has obtained promising initial results.

The OLE study will investigate the long-term safety, tolerability and efficacy of monepantel (MPL) in patients with motor neurone disease (MND) / amyotrophic lateral sclerosis (ALS) who previously completed the Phase 1 MEND Study.

MPL is described as a novel, potent and safe inhibitor of the mTOR pathway that has key influences in cancer growth and neurodegenerative diseases.

In December 2023, PharmAust completed its 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.

All 12 patients have now elected to continue treatment with MPL through a compassionate-use program and will be able to participate in the 12-month OLE study.

In this next stage, patients will receive a daily dose of 10 milligrams of MPL per kilogram of body weight to further test if it safely reduces disease-associated protein accumulation in motor neurons and provides therapeutic benefits for patients with MND/ALS.

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.

Promising early results

Initial studies by PharmAust partner Berry Consultants have highlighted the potential benefits MPL may provide.

Berry is now assessing the likelihood of a 100% survival rate among the 12 Phase 1 MEND patients without treatment.

The statistical consultant specialist is utilising baseline survival rates derived from historical control data within the pooled resource open-access ALS clinical trials (PRO-ACT) database.

After adjusting for differing diagnosis durations, Berry’s conservative sensitivity analysis has identified a one-year study survival rate estimate of 67.7% with a 95% confidence interval.

In comparison and considering differential diagnosis durations, the probability estimates of all 12 Phase 1 MEND patients surviving today without treatment are less than 0.1% (less than 1 in 1,000).

The PRO-ACT database is the largest publicly-available repository of merged ALS clinical study data pooled from 16 completed Phase 2/3 ALS/MND clinical studies and one observational study.

Over 8 million de-identified longitudinally-collected data points from more than 8,600 people with ALS were standardised across studies and merged to create the database, which includes demographics, family histories and longitudinal clinical and laboratory data.

MND costly and painful

MND is an umbrella term to describe the different types of motor neurone diseases, of which ALS is the most common.

These diseases affect nerves known as motor nerves, or motor neurons.

In MND, these neurons degenerate and die, causing the muscles to become weaker and weaker, eventually leading to paralysis.

Studies have found that for each year they live, every MND patient will on average require $31,598 worth of equipment.

The average lifespan is between 2-5 years so this equates to an average lifetime cost of between $63,196 and $157,990 in equipment needs alone.