Neurizon gets OK to test liquid form of ALS medication in Australia

Neurizon Therapeutics has received ethics approval for an Australia-based Phase 1 clinical trial that will test an oral liquid formulation of NUZ-001, which the company is developing for amyotrophic lateral sclerosis (ALS), in healthy volunteers.

A tablet version of NUZ-001 is being tested in a Phase 2/3 clinical trial in the U.S. The company is developing the liquid formulation “in direct response to the practical challenges faced by people living with ALS, particularly as swallowing difficulties become more prominent during disease progression,” Sergio Duchini, Neurizon’s interim executive chairman, said in a company press release. “In addition to supporting flexibility and continuity of treatment, the formulation is intended to improve the overall practicality of administration for patients, caregivers and clinical teams.”

The medication may be administered through feeding tubes, allowing for treatment continuity as ALS progresses, the company says. Neurizon aims to “support long-term treatment accessibility and expand the potential utility of NUZ-001 across different stages of disease progression,” Duchini said.

The Bellberry Human Research Ethics Committee, an independent Australian ethics review board that oversees research involving human participants, approved the company’s plan for the trial, which will compare the liquid formulation with the tablet version in 32 healthy volunteers.

The four-arm trial will assess the safety and pharmacological properties of the two formulations when taken in a fasting state or after a meal. It also aims to evaluate changes in ALS biomarkers and the palatability of the liquid formulation to support its continued development. The study will start in the third quarter of 2026 and conclude by the end of the year, “subject to the completion of remaining operational and site initiation activities,” the company said.

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A novel helped me process heavy questions I face as an ALS caregiver

Restoring autophagy

Impaired autophagy, the process cells use to break down and recycle damaged or unnecessary components, is thought to contribute to the accumulation of misfolded proteins that drive nerve cell damage in ALS.

Preclinical studies have suggested NUZ-001, originally developed as a veterinary deworming agent, may help restore autophagy and reduce toxic protein buildup. It does that by inhibiting the mTOR signaling pathway, which helps regulate this recycling system.

In a Phase 1 clinical trial (NCT04894240) and its open-label extension (NCT06177431), daily treatment with NUZ-001 was generally safe and well tolerated in people with ALS. Trial data also suggested that the therapy may slow disease progression and extend survival.

NUZ-001 is being evaluated in the HEALEY ALS platform trial (NCT04297683), which is testing multiple experimental ALS therapies simultaneously using a shared study design and placebo group.

NUZ-001 was the ninth therapy to be included in the trial. Its Phase 2/3 arm (NCT07410806), which recently dosed its first participant, expects to enroll about 160 adults at ALS centers across the U.S.

The NUZ-001 Phase 2/3 arm (NCT07410806), which recently dosed the first patient, will enroll about 160 patients who will be randomly assigned to receive a 10 mg/kg dose of NUZ-001 or a placebo for 36 weeks (about 8.3 months). For each patient given a placebo, three will be given the experimental therapy. The main goal is to assess changes in disease progression and survival, but researchers will also assess changes in lung function and other clinical outcomes, and continue to track safety.