PharmAust (ASX: PAA) has achieved a major breakthrough in plans to commercialise its monepantel (MPL) treatment for motor neurone disease with the receipt of an orphan drug designation (ODD) from the US Food and Drug Administration (FDA).

The ODD status assists companies in the development of safe and effective treatments for rare diseases and disorders.

It also provides incentives including tax credits, grants, waiver of some administrative fees for clinical trials and seven years of market exclusivity following drug approval.

Treating MND

Motor neurone disease (MND) is the name given to a group of diseases in which the nerve cells that control muscles undergo degeneration and die.

Amyotrophic lateral sclerosis (ALS) is a subtype of MND.

MND is the widely used generic term in the United Kingdom, Australia and parts of Europe, whereas ALS is more commonly used in the United States, Canada and South America.

It is also known in the US as “Lou Gehrig’s disease” after a famous New York Yankees baseball player who lost his life to the condition.

Widespread reach

Although classified as a rare disease based on its prevalence, ALS/MND is in fact quite common with approximately 140,000 new cases diagnosed worldwide each year.

The disease affects each individual differently and can have a devastating impact on family, carers and friends.

The rapidly progressive nature of the disease requires constant adaptation to increasing and changing levels of disability, which in turn require increased levels of support.

Strong pathway

PharmAust’s managing director John Clark said the ODD grant is an outstanding milestone for PharmAust and MPL.

“It provides an even stronger pathway forward for the drug, particularly in light of recent failures of other MND treatments,” he said.

“We are now increasingly optimistic as we progress to our pivotal registration adaptive Phase 2/3 study, which will commence in H2 CY2024.”

Funding support

The fight against MND attracts major attention in Australia, with the Big Freeze fundraising campaign currently in its tenth consecutive year of operation for FightMND.

Legendary AFL footballer and MND sufferer Neale Daniher is the patron for the campaign aimed at raising funds for research into the disease, while providing support for sufferers of what is known locally as “the Beast”.

With the ongoing support of the Australian public and the FightMND Army, FightMND has invested more than $55.9 million into MND research since 2014.

Promising results

PharmAust’s application to the FDA was based on preclinical mechanistic data that demonstrates MPL can induce autophagy in diseased cells, considering the pathology of the disease.

Positive top-line data from the Phase 1 MEND Study showed that daily administration of MPL over a 7- to 12-month period was well-tolerated and did not result in any dose-limiting toxicities or serious adverse effects.

It was found that daily administration of MPL resulted in a clinically meaningful therapeutic effect, as evidenced by a small and non-significant numerical reduction in ALS Functional Rating Scale-Revised scores from baseline to end of treatment.

Quality of life, cognitive and behavioural function were not significantly impacted and there was no change in respiratory function.

PharmAust ascertained that the effects of treatment with MPL on biomarkers demonstrated a large reduction in cerebrospinal fluid, which also supported a meaningful clinical effect.