Key Facts:
- An Australian biotechnology company, NuNerve, is advancing its investigational therapy NUN-004 into a national Phase 2 clinical trial aimed at halting the progression of motor neurone disease (MND) and restoring function by supporting the repair of damaged neural connections.
- NUN-004 is a long-acting, protein-based therapy designed to inhibit EphA4, a receptor whose excessive signalling has been linked to motor neuron degeneration, with the goal of protecting motor neurons and enabling nerve repair - going beyond current treatments that largely focus on slowing functional decline.
- The trial will recruit 75 participants across six Australian clinical sites, with participants randomised to receive either NUN-004 or a placebo over 24 weeks, followed by a 24-week open-label extension in which all participants receive NUN-004.
- The Phase 2 trial follows an earlier Phase I/Ib study involving 20 healthy volunteers and eight people living with MND, which showed NUN-004 was well tolerated, with disease stabilisation observed during treatment and participants reporting improvements in motor tasks including grip, balance, walking, strength, and reduced fatigue.
- NuNerve, which was established to advance over 25 years of research originating at the University of Queensland's Queensland Brain Institute, intends to seek international investment and partnerships to progress NUN-004 into Phase 3 and towards potential regulatory approval in major global markets.
Video interviews, overlay and photos here BRISBANE, AUSTRALIA - 10 November 2026 - An Australian-developed therapy aimed at halting the progression of motor neurone disease (MND) and restoring function by supporting the repair of damaged neural connections is advancing into a national Phase 2 clinical trial. The 75-participant trial follows encouraging early signs of disease stabilisation and functional improvement in a Phase I/Ib study of NUN-004, the lead investigational therapy from Queensland-based clinical-stage CNS (central nervous system) biotechnology company, NuNerve. Motor neurone disease (MND), also known as amyotrophic lateral sclerosis (ALS), is a progressive neurological condition that affects the nerve cells in the brain and spinal cord responsible for movement. As these motor neurons deteriorate and die, the brain can no longer send signals to the muscles, leading to muscle weakness, wasting, and loss of function over time.
NUN-004 is a long-acting, protein-based therapy designed to inhibit EphA4, with the aim of protecting motor neurons and supporting nerve repair. EphA4 is a receptor whose excessive signalling has been linked to motor neuron degeneration and impaired recovery of nerve connections.
NuNerve Chair Emeritus Professor Perry Bartlett AO said the therapy was developed to pursue a more ambitious goal than slowing decline alone. "No approved treatment has been shown to halt or reverse progression across the broad MND population," he said.
"Current treatments largely focus on slowing the loss of function.
"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 MND." The trial will recruit 75 participants across six Australian clinical sites on the Sunshine Coast, Sydney, Newcastle, Brisbane, and Perth.
Participants will be randomised two-to-one to NUN-004 or placebo for a 24-week double-blind study, followed by a 24-week open-label extension in which all participants receive NUN-004. The study will assess change in the ALS Functional Rating Scale-Revised, safety and tolerability, together with respiratory, neurophysiological, quality-of-life and biomarker measures. NuNerve Clinical Lead Associate Professor Antony Winkel said the study had been carefully designed to determine whether NUN-004 meaningfully changes the course of MND. "People living with MND 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 I signal is a real treatment effect. "If those findings are confirmed, they would represent an important result for people living with MND and place Australian MND research firmly on the international stage." The new trial builds on a published Phase I/Ib study involving 20 healthy volunteers and eight people living with MND. The results showed NUN-004 was well tolerated, with stabilisation of the disease during treatment and participants reporting improvements in fine and gross motor tasks including grip, balance, walking, strength and reduced fatigue. 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 FightMND, spanning early clinical development through to Phase 2.
NuNerve intends to seek international investment and partnerships to progress NUN-004 into Phase 3 and towards potential regulatory approval in major global markets.
