Neurizon Therapeutics Limited

ASX:NUZ

Neurizon Therapeutics Limited

ASX:NUZ
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$0.064 (-4.5%)
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Forum Topics NUZ NUZ Expanded enrolments

Pinned straw:
lastever
Added 2 months ago

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.
lastever
Added 2 months ago

I watched both webinars - Neurizon's last night (for investors) and Healey's this morning (for the ALS patient community). There was clear enthusiasm at both, including from the Healey team and statisticians.

I want to develop a balanced view on what they are enthusiastic about - was it the fact they had any trial at all after several quiet years, or something about NUZ-001? It appears to be both.

Regarding enrolments, the coordinator said at the patient webinar that this is the fastest enrolment she has ever seen, not just at Healey but in fact at any trial (I assume she meant ALS trial, but it is very fast anyway).

Regarding potential efficacy of NUZ-001, the statistician noted at the patient webinar that we don't want to overpromise, but certainly there are still a couple of patients on the drug after 4 years and that gives hope, and the signals from the small phase 1 are something everyone wants to test further. And it appears safe and well tolerated.

Part of their job is to advance the science, for the benefit of future patients. The TPD-43 target of action by NUZ-001 is one of the main priorities for neurodegenerative research. So they have that incentive. However, they are also patient advocates on a direct and personal level, and they are willing to say they think they have a safe drug that might just extend life and provide clinically meaningful benefit.

I hadn't been 100% sure that the alleged enthusiasm of the Healey people was somewhat 'magnified' by Neurizon for investor consumption. It's clear now that the enthusiasm is real.

So, in my opinion, there is a 'de-risking' signal here: They're unlikely to stop for futility - because it will be fully enrolled in a few months and it's all funded. There is no way you are taking a safe and potentially beneficial drug away from these patients early when a few further months on drug might make the difference. At the least, a full trial provides data which is shared with the ALS research community.

In trials, an ambiguous safety event is an ever-present risk. They now have 60+ people on drug, and there are one or two patients who have been on it for 4 years. The independent Melbourne doctor from the original phase 1 said it was the easiest trial she's ever been involved in. I'd say the risk is very low.

One of the factors that prevents success is simply the trial being stopped early. Remove that risk and you marginally increase the odds of success.

5
lastever
Added a month ago

3 weeks ago there were 70 patients screened and assigned for Healey. Today there are over 167, with 129 already dosed - out of a required 240. At this pace recruitment will close in the next few weeks.

6
lastever
Added 3 weeks ago

2 weeks ago there were 167 assigned patients and 129 dosed out of a target 240.

Today there were at least 201 assigned and 174 dosed.

We could be under a month to all 240 patients dosed and the start of the 36 week clock, unless there's a strategy to look for specific cohorts rather than taking all-comers.

36 week trial from late July plus say 2 months for data analysis gives a readout around late May 2027.

3
lastever
Added 3 weeks ago

Enrolment is now closed for Healey at all sites, over 240 patients have been screened ('over' because a handful will have had contra-indications), and the last patients are expected to be dosed over the next few weeks. So data readout could easily come in May 2027.

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