Monepantel

Neurizon is a small Melbourne-based company listed on the Australian stock exchange; it was formerly called PharmAust. NUZ-001 is its lead drug, and it licenses monepantel from the animal-health company Elanco. It reports A$11.4 million cash at the end of September 2026 and says it is funded through the HEALEY results.

Monepantel is thought to dampen mTOR, a cell "growth switch", which can turn up autophagy, the cell's recycling system for clearing clumped proteins such as TDP-43 that build up in ALS. This idea comes from lab studies; whether it works this way in people with ALS is not proven.

NUZ-001 is "Regimen I" of the HEALEY ALS Platform Trial in the US, where 250 people were randomly given the drug or placebo (3 to 1) for 36 weeks, followed by 36 weeks where everyone gets the drug. The FDA paused the programme from January to October 2025 to ask for more animal data, and in January 2026 it declined a Fast Track request. In October 2026 the FDA said a positive HEALEY result could on its own support an approval application.

In the Phase 1 MEND study, 12 people took monepantel without a placebo group. Their ALSFRS-R score fell about 0.74 points a month, which the company says is 39% slower than matched people from an old trial database (58% slower in the 6 people on the higher dose). Comparisons with historical patients are often misleading, so this is only an early hint. The placebo-controlled HEALEY result will be the first real test.

This is the most favourable result reported. See all key studies for the full picture.

HEALEY enrols adults with sporadic or familial ALS at US sites, and Regimen I uses 10 mg per kg of body weight daily, the dose picked from the Phase 1 study. The drug is not limited to any genetic form of ALS.

Neurizon expects the 36-week placebo-controlled part to end in early Q2 2027 and to report the main results in late Q2 2027. Separately, an NIH-funded expanded access programme for up to 200 people who cannot join trials is due to start enrolling in Q1 2027.

Regimen I stopped enrolling in July 2026 after reaching 250 participants, so new patients cannot join it. An NIH-funded expanded access programme, run by the Healey & AMG Center at about 20 US sites plus a virtual site, is expected to start enrolling in Q1 2027 for people who do not qualify for trials.

• HEALEY ALS Platform Trial, Regimen I (NUZ-001) (NCT04297683) active not recruiting · USA (78 sites)