Motor neurone disease (MND) is a severely debilitating, incurable and fatal condition that progressively damages parts of the nervous system.

Amyotrophic lateral sclerosis (ALS) is the most common form of MND. MND/ALS is characterised by progressive degeneration of nerve cells in the spinal cord and brain, affecting voluntary control of the arms and legs and eventually leading to difficulty breathing.

MND/ALS has an extremely poor prognosis and has no approved cure or effective treatment to reverse progression. Independent studies have shown that one-third of patients die within 12 months of diagnosis, with an average life expectancy of 27.5 months.

The International Alliance of MND/ALS Associations estimate MND/ALS affects over 350,000 people globally and kills more than 100,000 people annually. While there is currently no cure for MND/ALS, there are limited treatment options. The standard-of-care treatment Riluzole prolongs life by an estimated 3 months.

Monepantel MEND Study

PharmAust completed a Phase 1 study of monepantel to treat MND/ALS in December 2023, delivering promising safety and preliminary efficacy results. The MEND study aimed to determine the recommended Phase 2 dose based on safety and preliminary efficacy data

The MEND study indicated that when compared to matched controls from the PRO-ACT natural history database, monepantel slowed the progression of disease by 39%. These findings were independently verified and presented at the premier European ALS conference in 2024.

Participating patients are also eligible to roll-over into an open-label extension (OLE) study which began in early 2024. The OLE study will generate valuable safety and efficacy data for a further 12 months.

PharmAust has leveraged the positive data from the Phase 1 MEND study to design a Phase 2 study (see below), and to support applications for an orphan drug designation and investigational new drug application with the US FDA.

PharmAust’s Phase 1 MEND study was supported by a grant of $881,085 from FightMND, Australia’s largest independent not-for-profit organisation for MND/ALS research.

HEALEY ALS Platform Trial

Monepantel has been accepted into the prestigious HEALEY ALS Platform Trial conducted by the Massachusetts General Hospital in partnership with the Northeast ALS (NEALS) consortium in the US.

Inclusion in the HEALEY trial provides independent validation by global ALS experts of monepantel’s potential as an ALS treatment. The innovative platform trial is a large-scale collaboration across 70+ clinical sites in the US to evaluate several drug candidates simultaneously.

Monepantel’s inclusion is a significant step towards FDA approval, enhances PharmAust’s visibility within the ALS research community and is a critical advancement in our mission to offer a viable treatment for ALS. Not only does it enhance patient access to the trial, but also reduces trial costs and accelerates timelines, supporting our commitment to advancing monepantel through the clinical pipeline.

The trial is set to commence in the second half of 2024.

Learn More