# NuNerve's MND drug NUN-004 faces its first placebo-controlled test after early signs of stabilising disease - Published: 2026-11-10 - Author: [Healthcare News](https://healthcarenews.io/author/healthcarenews-io.md) - Topics: [News](https://healthcarenews.io/news.md) - About: [NuNerve](https://nunerve.com/), [NUN-004](https://nunerve.com/clinical-program/) - Based on: [Australian therapy aimed at halting MND progression and restoring function advances to national Phase 2 trial](https://www.globenewswire.com/news-release/2026/11/10/3399135/0/en/australian-therapy-aimed-at-halting-mnd-progression-and-restoring-function-advances-to-national-phase-2-trial.html), GlobeNewswire, 2026-11-10 - Based on: [Evaluation of NUN-004, a Novel Engineered Ephrin Antagonist, in Healthy Volunteers and Patients with Amyotrophic Lateral Sclerosis: A Phase I/Ib, Open-Label, Escalating Dose and Extended Access Study](https://doi.org/10.1007/s40261-024-01410-x), Clinical Drug Investigation (Springer), 2024-12-02 - Based on: [Clinical Program](https://nunerve.com/clinical-program/), NuNerve - Based on: [Amyotrophic lateral sclerosis](https://medlineplus.gov/genetics/condition/amyotrophic-lateral-sclerosis/), MedlinePlus Genetics, US National Library of Medicine - Source: https://healthcarenews.io/news/nun-004-phase-2-trial-mnd-placebo-test/ - Publication: Healthcare News - Publisher: Connectively (Terkel, Inc.) - Editorial policy: https://healthcarenews.io/editorial-policy/ - AI use: Connectively's automated system wrote this article with AI from a press release issued by NuNerve on November 10, 2026, linked under Sources. Statements about the product are the company's. The illustration was generated with AI. The article was assembled and published automatically, without an editor's review. Report errors to corrections@connectively.us. The NUN-004 Phase 2 trial will test NuNerve's motor neurone disease drug against placebo in 75 people at six Australian sites, the Brisbane company said on November 10, 2026. It is the first controlled test of a therapy whose small, open-label first study suggested it might stabilise the disease. ## Key points - Participants will be assigned two to one between NUN-004 and a placebo for 24 weeks, with neither they nor the investigators knowing who receives which, and then everyone receives NUN-004 for a further 24 weeks. - The trial's measure of function is change on the ALS Functional Rating Scale-Revised (ALSFRS-R), alongside safety, breathing, nerve-signal, quality-of-life and biomarker results. - The earlier study gave the drug to 20 healthy volunteers and eight people with MND, and had no placebo group: its published title describes it as open-label ([Clinical Drug Investigation](https://doi.org/10.1007/s40261-024-01410-x)). - The release names five places for its six sites, the Sunshine Coast, Sydney, Newcastle, Brisbane and Perth, without saying which has two, and gives no start date or date for results. - FightMND has funded the program with three grants in a row, and NuNerve says it will look for international investors and partners to reach Phase 3. ## What is NUN-004 and how is it meant to work? NUN-004 is an investigational, long-acting protein drug that blocks EphA4, a receptor on nerve cells. NuNerve says too much EphA4 signalling has been tied to the loss of motor neurons and to nerves failing to reconnect, so blocking it is meant to protect those cells and help damaged connections repair. The drug comes out of research at the University of Queensland's Queensland Brain Institute, led by Emeritus Professors Perry Bartlett and Andrew Boyd. MND, called amyotrophic lateral sclerosis (ALS) in the United States, destroys the nerve cells that control movement, so muscles weaken and waste. About 5,000 people in the United States are diagnosed with it each year, and worldwide it affects 2 to 5 in every 100,000 people, according to [MedlinePlus Genetics](https://medlineplus.gov/genetics/condition/amyotrophic-lateral-sclerosis/) at the US National Library of Medicine. > "No approved treatment has been shown to halt or reverse progression across the broad MND population." > > — Emeritus Professor Perry Bartlett AO, Chair, NuNerve ## What did the first study show, and what did it not? The first study showed the drug was well tolerated, the company says, and its participants with MND held steady on the disease while being treated. They also reported better grip, balance, walking and strength and less fatigue, NuNerve says. On its [clinical program page](https://nunerve.com/clinical-program/), NuNerve adds that no serious adverse events or withdrawals were reported in that study. What it could not show is whether the drug caused any of that. With eight patients, all on the drug and all aware of it, there was nothing to compare against, and progression varies widely between people with the disease ([MedlinePlus Genetics](https://medlineplus.gov/genetics/condition/amyotrophic-lateral-sclerosis/)). The announcement gives no figures for how much scores changed. That is the question the placebo arm is there to answer. > "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." > > — Associate Professor Antony Winkel, Clinical Lead, NuNerve ## What does the NUN-004 Phase 2 trial measure? It measures whether people on the drug lose less function than people on placebo, scored on the ALSFRS-R, a rating of everyday physical function. Breathing tests, nerve-conduction measures, quality of life and blood or fluid biomarkers sit alongside it. For background on how controlled trials like this one are run, see our explainer on [how clinical trials shape future healthcare](https://healthcarenews.io/insight/your-journey-in-clinical-research-how-clinical-trials-shape-future-healthcare/). ## What does this mean for clinicians and patients? Our analysis: for clinicians, this is a trial to watch, not a treatment to discuss with patients yet. The release names no dose, route or schedule and no timeline, and does not say how it settled on 75 participants. NuNerve stresses that the announcement does not invite anyone to enrol; its site lists the registration as ACTRN12626001318370 on the Australian New Zealand Clinical Trials Registry and directs people to their treating clinician ([NuNerve](https://nunerve.com/clinical-program/)). EphA4 is one of several new approaches in the pipeline; our roundup of [trends in new drug and therapy development](https://healthcarenews.io/qa/5-breakthrough-trends-in-new-drug-and-therapy-development/) covers the wider field. ## Frequently asked questions ### Is NUN-004 an approved treatment for MND? No. NuNerve describes NUN-004 as an investigational product that has not been approved for commercial use ([NuNerve](https://nunerve.com/clinical-program/)). ### How many people will take part in the NUN-004 Phase 2 trial? The company says 75 people with MND, at six Australian clinical sites, with two receiving the drug for every one receiving placebo during the blinded part. ### Will every participant eventually receive NUN-004? Yes, according to the release: after the 24-week blinded phase, everyone moves to a 24-week open-label extension on the drug. ### Can people with MND join the trial through this announcement? No. NuNerve says the announcement is not recruitment material, and points people to their treating clinician and the official trial registry. ### Who is funding the research? NuNerve was set up by the Estate Peter Goodenough Unit Trust and UniQuest, and FightMND has given the program three consecutive grants. ## Sources - [Australian therapy aimed at halting MND progression and restoring function advances to national Phase 2 trial](https://www.globenewswire.com/news-release/2026/11/10/3399135/0/en/australian-therapy-aimed-at-halting-mnd-progression-and-restoring-function-advances-to-national-phase-2-trial.html): press release from NuNerve via GlobeNewswire, 10 November 2026. - [Evaluation of NUN-004, a Novel Engineered Ephrin Antagonist, in Healthy Volunteers and Patients with Amyotrophic Lateral Sclerosis: A Phase I/Ib, Open-Label, Escalating Dose and Extended Access Study](https://doi.org/10.1007/s40261-024-01410-x): the published first study, in Clinical Drug Investigation. - [NuNerve clinical program](https://nunerve.com/clinical-program/): the company's description of the Phase 1/1b and Phase 2 studies. - [Amyotrophic lateral sclerosis](https://medlineplus.gov/genetics/condition/amyotrophic-lateral-sclerosis/): MedlinePlus Genetics, US National Library of Medicine.