AI June 2026 updated to 30-45% odds range. Success is defined by the Healey coordinator as a 30% reduction in the rate of decline of patients by a key measure. The trial is 80% powered.

I'm pretty sure it's just telling me what I want to hear, but there are some keen beans on HC that have dug into the science and think we're closer to 90%. March estimate was a 15-20% chance of a positive readout at the start of the current Healey phase 2/3 trial. Healey is the acid test.

The overall ALS field success rate is 1% but that includes everything ever tried. The analysis then improved for Neurizon based on the safety and current level of evidence.

I got into Neurizon way too early. I think now is a much better time and I topped up a smidge today. There are a bunch of reasons why confidence will be turning today:

• There was a question mark over what happens if NUZ-001 has to go it alone at Healey, centered on both funding and how to maintain statistical power. That was answered by Healey. They've maintained 80% power for their main endpoint by expanding enrolments, and they provided the gap funding.
• There was also a question of what was here-say by Neurizon execs verses the reality on the ground at Healey. It is clearly real.
• There's a supply deal coming in a month with the manufacturer, that message was repeated yesterday. The manufacturer is a shareholder and has a seat on the board and is an $11b US veterinary product manufacturer.
• There's a new CEO coming soon. That will be the final 'uncertainty' risk gone besides the trial itself.
• Topline is expected in July 2027, and there's a small but decent probability of immediate deals. If you want to be one of the last Australians' to benefit from the CGT discount on a windfall, there's an incentive to buy soon. If only the labor party understood what incentivises the funding of medical research in Australia. But I digress.

I consider this a punt with hard-to-define odds.

Neurizon’s phase 2 regime at the Healey trial is to be expanded from 160 to 240 patients. This is to be largely funded by the philanthropic funds of the Healey ALS centre, and will actually accelerate the timeline for Neurizon. 

Neurizon Webinar this Thursday 4pm and Healey webinar this Friday

Something like this was expected, because the other drug candidates have not shown up yet at Healey - leaving Neurizon alone and underpowered on the placebo side.

It allows Neurizon to have a de-facto stand-alone trial, with the costs of expansion largely covered. They will have 180 on NUZ-001 and 60 on placebo - which maintains the 3:1 sorting ratio that incentivises ALS patients to enrol.

There’s more than one positive signal:

1. After 60 patients were dosed, Healey decided to expand enrolments. This suggests there have been no worrying safety or tolerability issues, a tick because Neurizon’s phase 1 had only 12 patients.
2. Timing has improved:
3. The enrolment has been Healey’s most rapid to date and 113 patients are assigned with 74 dosed.
4. We’re basically at halfway of the expanded enrolment, 3 months in. The 2nd half should fill faster. The company is confident of last patient dosing (which requires 36 weeks on trial) in Q2 2027 and topline results in early Q3 2027. 
5. The dataset for NUZ-001 will now be more internally consistent, and ‘expected to enhance future regulatory, partnering and commercial opportunities for NUZ-001, while also strengthening our ability to generate valuable biomarker and translational insights relevant to the broader neurodegenerative disease landscape.’
6. By an unplanned turn of events, NUZ-001 has become the focus of resources at Healey, the most prestigious ALS institution in the US.

Milestones:

• July 2027: Topline results
• Q2 2027: Last patient dosing
• For safety and tolerability, every month that goes by is evidence in an expanding group of patients.
• For efficacy, there will be no formal 'halfway' futility announcement, so no news will be good news. But I'm guessing that the ALS community will value statistically relevant data about the mechanism of action here, involving TPD-43 which is a focus of current neurodegenerative research. Phase 1 (on a not-statistically-relevant sample) had all signals pointing in a positive direction. So I assume only safety issues or a totally flat line would stop phase 2.

For anyone curious about Neurizon's phase 2 ALS candidate, I recommend these 2 videos to get up to speed:

Healey ALS Platform trial 2026 (55m) presented by 2 lead investigators of Healey with representatives of Neurizon

• History and rationale of the Healey platform in the USA
• What they want to confirm in the current drug's mechanism of action with bio-markers
• Expecting a hockey stick fulfilment of enrolments over months as sites open and word spreads
• Representatives of Neurizon run through phase 1 and OLE data
• Signals of life extension and disease modification

Phase 1 trial science and results (16m) from late 2024 presented by lead scientist, an Associate Prof. at Monash Uni.

• Small number of people (12 on drug) but showed some positive life-extension signals
• 'probably the easiest drug trial I've ever done'
• I don't think this doctor has any financial interest in Neurizon
• Note that further data came after this from Open Label Extension - see Healey video

Footnote: It's a punt - For the sake of anyone new to drug trials, you should understand that the phase 1 trial was on a very small number of patients (12), and compared against matched controls from a historical patient database, not a placebo/standard control group. So, the current Healey ALS Platform trial is the acid test - it will be what statistically confirms the signals of effectiveness or falls short.

The first patients are expected to begin enrolling for Neurizon’s regimen of the Healey ALS Platform Trial in the next few weeks.

This is not an ordinary clinical trial. It is an ongoing, multi-candidate effort by the ALS medical community to identify potential treatments via a shared stage 2 placebo-controlled system. It is being run out of Mass General Hospital, the largest teaching hospital of Harvard Medical School, with 70 trial sites across the US.

Last week, industry publication Fierce Biotech, held a webinar with the trial coordinator, the Neurizon CEO, and the 2 lead investigators. I recommend watching it for:

• an overview of the platform trial
• Neurizon’s scientific rationale and stage 1 trial results
• the feeling the lead investigators have for Neurizon’s candidate.

Most people would know that ALS is a fast, terminal neuro-degenerative disease with no known cure. So patients need a treatment, now. With this platform trial, they have not only the latest available stage 2 candidates, but also a 3:1 drug to placebo sorting ratio.

Since 2020, 7 other candidates have been trialed. Despite not reaching their endpoints, 2 candidates showed enough life extension signal to be ear-marked for stage 3 trials (to be run independently of Healey). The last trial ended in mid-2025.

Following very interesting stage 1 results, Neurizon will be the 8th candidate, and benefits from the well-established system and shared data from 1500 patients, with:

• a clinic network of 70 US trial sites
• an improved bio-marker screening protocol for best patient outcomes
• a longer trial time of 36 weeks (previously 24) for improved signal

Hopefully it’s clear this is maximum exposure for Neurizon, and a state-of-the-art, tried and tested trial system that will minimise the risk of any further FDA hiccups.

Healey regimen I:

• n=160, 3:1 randomised treatment:placebo
• First dosing in the coming weeks (Q1)
• 36 week double blind, followed by 36 week active extension (similar to an OLE).
• Therefore, primary results readout should be mid-2027.

Neurizon will be joined at some stage by an Eli Lily candidate and one other (with correspondingly increased patient pool, I believe), however until that happens all active treatment enrolled patients will be assigned to Neurizon.

Neurizon has been on a bit of a journey this year, and as a consequence are in an interesting spot if you like a good pharmaceutical speculation. They are finally about to commence in the Healey ALS Platform trial, and are in the middle of scraping together equity funds to see them through it, as required.

The Healey ALS Platform is not your average trial, it is a head-to-head multi-round trial set up for the ALS community to efficiently and ethically identify their best candidates by running multiple candidates all sharing a placebo group. Should Neurizon emerge showing the same improved survival and biomarker signal as it has already shown on a small number of patients, then things should get interesting at the other end.

Webinar will be worth a listen.