Australian ALS therapy aimed at halting disease progression and restoring function advances to Phase 2 trial

Video interviews, overlay and photos here

BRISBANE, Australia, Nov. 10, 2026 (GLOBE NEWSWIRE) -- An Australian-developed therapy aimed at halting the progression of amyotrophic lateral sclerosis (ALS) and restoring function is advancing to a Phase 2 clinical trial across Australia.

The 75-participant trial follows encouraging early signs of disease stabilisation and functional improvement in a small Phase I/Ib study of NUN-004, the lead investigational therapy from Queensland-based clinical-stage CNS (central nervous system) biotechnology company, NuNerve.

ALS, also known as Lou Gehrig’s disease, is the most common form of motor neuron disease. It damages the nerve cells that control movement, progressively affecting a person’s ability to walk, speak, swallow and breathe.

NUN-004 is a long-acting, protein-based therapy designed to inhibit EphA4, a receptor involved in neural connectivity and repair.

NuNerve’s approach aims to protect motor neurons while creating conditions in which damaged neural connections may recover.

NuNerve Chair Emeritus Professor Perry Bartlett AO, whose research helped lay the foundation for the therapy, said NUN-004 was developed with a more ambitious goal than slowing decline alone.

“No approved treatment has been shown to halt or reverse progression across the broad ALS population,” Professor Bartlett said.

“NUN-004 was designed to go further to protect motor neurons and create the conditions for damaged neural connections to repair.

“We have spent more than 25 years following the biology of EphA4, from understanding its role in motor neuron connections to developing a therapy that can now be tested rigorously in people living with ALS.”

The Phase 2 trial will recruit 75 participants at six Australian sites in the Sunshine Coast, Brisbane, Sydney, Newcastle, Melbourne and Perth.

Participants will be randomly assigned in a two-to-one ratio to receive NUN-004 or a placebo during a 24-week blinded study. A further 24-week open-label period will allow all participants to receive NUN-004.

Researchers will assess changes in everyday function using the ALS Functional Rating Scale–Revised, alongside safety, breathing, nerve and muscle function, quality-of-life and biomarker measures.

NuNerve Clinical Lead Associate Professor Antony Winkel said the study was designed to put the early findings to a rigorous test.

“People living with ALS urgently need better treatment options grounded in high-quality evidence,” Associate Professor Winkel said.

“This trial combines measures of everyday function with respiratory, neurophysiological and biomarker assessments to determine whether the early Phase 1 signal is a real treatment effect.”

The published Phase 1/Ib study involved 20 healthy volunteers and eight people living with ALS.

NuNerve was established by the Estate Peter Goodenough Unit Trust and UniQuest to advance more than 25 years of research by Emeritus Professor Perry Bartlett and Emeritus Professor Andrew Boyd originating at The University of Queensland’s Queensland Brain Institute.

The program has received three consecutive grants from Australian nonprofit FightMND, spanning preclinical development through to Phase 2.

NuNerve intends to seek international investment and partnerships to advance NUN-004 toward a Phase 3 trial and potential regulatory approval in major markets.

About NuNerve

NuNerve is a clinical-stage biotechnology company developing therapies designed to protect, reconnect and restore function in the central nervous system.

Our lead candidate, NUN-004, is advancing into Phase 2 development for ALS. This clinical program is the first step in a broader strategy to translate our expertise in neuronal connectivity, survival and repair into treatments for neurological diseases.

Notes for editors

Published Phase 1/Ib study: https://doi.org/10.1007/s40261-024-01410-x

Available for interview: NuNerve Chair Emeritus Professor Perry Bartlett AO FAA, NuNerve CEO Dr Anthony Raphael and NuNerve Clinical Lead Dr Antony Winkel.

This is a corporate and scientific announcement, not participant recruitment material.

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