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.
