Known as the ‘powerhouse of the cell’, mitochondria are becoming a focus of research into ageing, health and disease
ASX healthcare companies are focusing on supplements supporting cell function and therapies targeting dysfunction linked to disease
EZZ Life Science co-funded research proposing that three compounds associated with healthy ageing may act more effectively in combination to prevent mitochondrial decline
For decades, the pursuit of better health and ageing well has focused on familiar strategies including good nutrition, regular exercise, managing disease risk and maintaining overall wellbeing.
However, a growing area of biomedical research is looking deeper inside the body, focusing on the tiny structures within our cells known as mitochondria.
First identified by Swiss anatomist and physiologist Albert von Kölliker in the mid-1800s, mitochondria were later named by German anatomist Carl Benda in 1898.
In a 1957 Scientific American article, biochemist Philip Siekevitz described mitochondria as the “Powerhouse of the cell”, a phrase that remains widely used today.
The description reflects mitochondria’s critical role in converting nutrients into adenosine triphosphate (ATP), the chemical energy that fuels everything from muscle contraction to brain activity.
But research over recent decades has revealed mitochondria play a much broader role beyond energy production.
They are involved in regulating cellular signalling, calcium balance, immune responses and apoptosis — an orderly, programmed process of cell self-destruction that enables damaged or unnecessary cells to be safely removed.
As people age, mitochondria can become less efficient, a process known as mitochondrial dysfunction.
It is recognised as one of the hallmarks of ageing and has been associated with declining cellular energy production and impaired cell function.
Mitochondria dysfunction has also been associated with increased oxidative stress, which is associated with heart disease, neurodegenerative disorders, diabetes and cancer.
The quest to improve mitochondrial function
With mitochondria present in almost every cell in the body, researchers are investigating whether supporting mitochondrial function could help maintain healthy cellular processes across tissues including the brain, muscles and cardiovascular system.
The growing interest in mitochondrial biology is creating opportunities across both the consumer healthcare market and clinical drug development, including within the ASX healthcare sector.
On the ASX some companies are developing supplements aimed at supporting cellular health.
Others are pursuing therapeutic approaches designed to target mitochondrial dysfunction and the biological processes associated with disease.
EZZ-backed research targets reducing mitochondrial decline
A focus area for health and wellness company EZZ Life Science (ASX:EZZ) is healthy ageing and prevention of mitochondrial decline.
Research by the University of Auckland, co-funded by EZZ, proposes that three compounds associated with healthy ageing may act more effectively in combination.
Published in the peer-reviewed journal Redox Biology, the research examines nicotinamide mononucleotide (NMN)/nicotinamide riboside (NR), pyrroloquinoline quinone (PQQ), and ergothioneine (EGT).
NMN/NR is thought to boost levels of nicotinamide adenine dinucleotide (NAD+), a coenzyme essential for cellular energy production and repair processes.
PQQ promotes the growth of new mitochondria, which produce chemical energy. EGT acts as a targeted antioxidant, neutralising harmful free radicals inside mitochondria.
The researchers propose the three compounds form a complementary loop, though they acknowledge direct human trial evidence for the combination remains limited.
Chief strategy officer Glenn Cross said the company’s focus was on developing health and wellness products supported by scientific research into healthy ageing.
In September 2025, the US Food and Drug Administration (FDA) confirmed NMN as lawful for use in dietary supplements.
Australia’s Therapeutic Goods Administration (TGA) followed suit in December 2025, adding NMN to its list of permissible ingredients for listed complementary medicines.
“Our focus at EZZ was on NMN and the research summary said the three compounds had potentially extensive protective effects,” EZZ Chief Strategy Officer Glenn Cross said.
“We were one of the first companies to come out with an NMN product. “
“Further research is still needed, but this study may lead to longer term validation and safety assessments.”
Vita Life taps into healthy aging golden opportunity
Vita Life Sciences (ASX:VLS) operates established consumer health brands including Herbs of Gold, VitaHealth and VitaScience selling a range of vitamins, supplements and wellness products across Australia and Asia.
The company provides exposure to the growing consumer trend towards preventative health, with demand increasing for products targeting areas such as energy, immunity, metabolic health, healthy ageing, including mitochondrial health.
‘Tough nut to crack’ but Alterity showing promise
Late-stage clinical biotechnology companies Alterity Therapeutics (ASX:ATH) and Neurizon Therapeutics (ASX:NUZ) are working to develop therapies that modify disease processes for neurodegenerative diseases.
Research suggests mitochondrial dysfunction and oxidative stress may be involved in the development and progression of several neurodegenerative diseases.
Alterity’s approach is focused on developing potential disease-modifying treatments for neurodegenerative disorders where cellular dysfunction may contribute to disease progression.
The company is preparing for a Phase III trial of its lead candidate ATH434 in multiple system atrophy (MSA), a rare and aggressive Parkinsonian disorder.
Affecting up to 50,000 patients in the US, MSA is driven by excess iron and aggregation of a protein called alpha-synuclein in the central nervous system (CNS).
Chief medical Advisor and Professor of Neurology at the Vanderbilt University Medical Center Dr Daniel O Claassen was principal investigator of Alterity’s biomarkers of progression in multiple system atrophy (bioMUSE) natural history study.
Claassen was also co-ordinating investigator of Alterity’s phase II trial ATH434-201, a double-blind study in patients with early-stage disease that has delivered positive top-line results.
He said ATH434 takes a novel approach acting as an “iron chaperone”, redirecting excess reactive iron to help protect neurons, curb protein accumulation and maintain or enhance neuronal function to offer what could be the first disease-modifying therapy.
“Iron dysfunction is a problem in MSA and what ATH-434 does is redistributes excess reactive iron in the cell to slow the pathologic progress without interfering with normal cellular function” he said.
“There is a large body of literature linking iron to neurodegeneration and that is through cellular and models along with autopsy.
“Neurodegeneration is a tough nut to crack, and this really moves the needle.”
He said many Parkinsonian disorders, and even, to some extent, Alzheimer’s, were linked to iron dysregulation, making them potential targets for exploration with ATH434.
Neurizon targets cellular maintenance pathways in neurodegeneration
Neurizon is developing its investigational small molecule NUZ-001 as a potential treatment for amyotrophic lateral sclerosis (ALS), the most common form of motor neurone disease (MND).
Taken as an oral tablet, NUZ-001 has a growing body of preclinical evidence indicating activity across biological pathways implicated in ALS pathology, including mechanisms involved in maintaining protein balance within neurons.
NUZ-001 has progressed to Regimen I of the HEALEY ALS Platform Trial, a Phase 2/3 registrational study and one of the largest late-stage clinical trials currently underway in ALS.
In April, Neurizon announced new preclinical findings on the biological mechanism of action (MOA) for NUZ-001, providing further insight into how the drug candidate may work in neurodegenerative diseases.
The data suggest NUZ-001 increases activity across multiple protein clearance pathways in neuronal systems.
The findings provide further insight into how NUZ-001 may influence neuronal pathways involved in managing misfolded and aggregated proteins, a key feature of neurodegenerative diseases such as ALS.
In these diseases, the progressive build-up of abnormal proteins can overwhelm the cell’s natural protein quality-control systems, contributing to neuronal dysfunction and cell death.
The research suggests NUZ-001 may help neurons better manage this burden by enhancing two important cellular clearance mechanisms — autophagy and its mitochondria subtype mitophagy, and the ubiquitin-proteasome system.
Autophagy acts as the cell’s bulk recycling system, helping break down larger damaged or aggregated proteins, while the ubiquitin-proteasome system selectively removes smaller unwanted proteins.
Together, these pathways play a critical role in maintaining protein balance, a process known as protein homeostasis.
“Our research is building an increasingly coherent picture of NUZ-001’s biological activity, including modulation of multiple pathways involved in maintaining protein homeostasis in neurons,” interim executive chairman Sergio Duchini said.
“That growing mechanistic understanding strengthens the scientific rationale for its advanced clinical evaluation in ALS.”
This story does not constitute financial product advice. You should consider obtaining independent advice before making any financial decision
At Stockhead we tell it like it is. While EZZ Life Science, Alterity Therapeutics and Neurizon Therapeutics are Stockhead advertisers, they did not sponsor this article.
Originally published as Powerhouse of the cell: ASX biotechs target the next frontier in healthy ageing