Before the Big Freeze, Neurizon delivers warm hope by advancing MND treatment

Ahead of FightMND’s Big Freeze read all about the biotech developing a potentially effective treatment for a common form of motor neurone disease.

Neurizon is advancing its potential ALS/MND therapy, as Australia's turns its attention to the debilitating illness at Monday's Big Freeze. Pic: Getty Images

• FightMND will hold its Big Freeze fundraiser for motor neurone disease on June 9

• Neurizon working to develop a potentially effective treatment for common form of motor neurone disease

• Recent data shows Neurizon's NUZ-001 drug well-tolerated and may extend life in ALS/MND patients

Ever been in an ice bath? It’s cold...really, really cold.

On June 9 celebrities will plunge into icy waters in front of the MCG crowd as the Melbourne Demons take on Collingwood for this year’s Big Freeze.

Blue beanies will fill the crowd with events also being held around the nation as the annual major fundraiser for FightMND.

The Australian charity is dedicated to raising awareness, funding groundbreaking research, and accelerating development of effective treatments for motor neurone disease (MND).

FightMND was co-founded in 2014 by 2025 Australian of the Year, former AFL player and coach Neale Daniher, following his diagnosis with MND in 2013.

Daniher, who led the Demons to the 2000 Grand Final, co-founded the organisation with the late Dr Ian Davis – a haematologist and fellow MND patient – and Pat Cunningham, whose late wife also had the disease.

MND is a group of progressive, fatal neurological disorders in which the nerve cells that control movement, speech, breathing and swallowing gradually degenerate and die.

"Motor neurone disease is a terrible disease that takes and takes," FightMND director of programs Dr Bec Sheean told Stockhead.

"Your movement, your voice, your breath and ultimately, your life.

"The support we receive from the Australian community during the Big Freeze and throughout the year means that we can invest in research in Australia, and around the world, that improves our understanding of the Beast and makes strides towards finding effective treatments and, one day, a cure."

Neurizon advances potential MND breakthrough

One ASX-listed company working to develop a potentially effective treatment and extend patient life expectancy for the most common form of MND called amyotrophic lateral sclerosis (ALS) is Neurizon Therapeutics (ASX:NUZ).

Managing director and CEO Dr Michael Thurn told Stockhead it was the only company on the ASX specifically focused on developing an effective therapy for MND with others exploring the space as part of programs tackling neurodegenerative diseases.

He said private Australian biotechs and organisations including the Queensland Brain Institute (QBI) – which is part of the University of Queensland – and Macquarie University MND Research Centre were also doing research in the space. Both have received funding support from FightMND.

Neurizon's lead drug shows promise

Neurizon's lead drug candidate NUZ-001 is a veterinary drug known as monepantel which is being repurposed in treating ALS.

Recent data from its open-label extension study shows the drug continues to be well-tolerated and is showing promise in extending the life expectancy of patients with ALS/MND, who live on average for just over two years.

He said data showed NUZ-001 has capacity to prevent toxic build up of cellular waste through a process known as autophagy.

"In patients with ALS there is toxic buildup of a protein called TDP43, which accumulates in the motor neurone and ultimately leads to the death of that motor neurone," he said.

Thurn said NUZ-001 was being investigated for its potential to prevent the toxic buildup of TDP43 in patients with ALS.

Analysis of patients treated with NUZ-001 by Neurizon's statistical partners Berry Consultants showed the drug significantly increased survival and reduced the risk of death by nearly 80% when compared to matched controls from a natural history database.

"Both those results were considered highly statistically significant," Thurn said.

Out of the 12 patients who started under the original phase I MEND study in October 2022 six are continuing treatment on NUZ-001 under a compassionate use program after completion of the 12-month open label extension study.

"Based on this small cohort of patients it looks like treatment with NUZ-001 potentially increases life expectancy by at least 11 months but more data is obviously pending to confirm that result," Thurn said.

What's next for Neurizon?

Full results from Neurizon's open-label extension study are forecast for Q3 CY25.

Meanwhile, the company is undertaking two pharmacokinetic (PK) studies necessary to lift a clinical hold on its US Food and Drug Administration (FDA) investigational new drug (IND) application for NUZ-001.

The studies are expected to be completed and submitted back to the FDA in coming months to request a lift on the hold in Q3 CY25.

Neurizon then hopes to enter the HEALEY ALS Platform Trial, a significant ongoing trial in the US aimed at accelerating ALS treatment development.

"We're one of eight companies from about 50 accepted into this prestigious trial," Thurn said.

The trial based out of Massachusetts General Hospital aims to accelerate ALS drug development by testing multiple drugs simultaneously in a flexible, adaptive trial design.

"The HEALEY ALS Platform Trial is a game-changing initiative that’s transforming the way we test potential treatments, offering hope and accelerating progress for everyone affected by MND/ALS," Thurn added.

"Despite the hurdles and heartbreak of past MND/ALS clinical trials, each setback deepens our understanding and fuels our drive.

"NUZ-001 stands at the forefront of this journey, offering a new horizon of possibility for patients and families."

READ about the Aussie Medtechs stepping up to help improve quality of life for those living with MND:
Big Freeze puts MND in focus – and the Aussie tech helping sufferers take back control

At Stockhead we tell it like it is. While Neurizon Therapeutics is a Stockhead advertisers, the company did not sponsor this article.

Originally published as Before the Big Freeze, Neurizon delivers warm hope by advancing MND treatment