Neurizon (ASX:NUZ) Clears FDA Hurdle for ALS Trial- Speculative Buy at 75% Off Highs?

Neurizon Therapeutics (ASX: NUZ) just hit a major milestone. The FDA has cleared its lead drug NUZ-001 to enter the HEALEY ALS Platform Trial at Massachusetts General Hospital, with patients now enrolling for this Phase 2/3 study in Q4 2025. This follows the FDA lifting its clinical hold back in October 2025. For investors, the timing is interesting. The stock has fallen roughly 75% from its highs despite this regulatory progress. In our view, this disconnect between share price and clinical progress creates a potential opportunity, but only for those who understand the risks.

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NUZ-001’s Early Results Stand Out in the ALS Space

The early data from Neurizon’s Phase 1 trial are hard to ignore. Patients treated with NUZ-001 showed an 80% lower risk of death compared to similar patients who did not receive the drug. We believe this is significant because current ALS treatments only extend life by a few months at best. If these results hold up in the larger trial, NUZ-001 could represent a major step forward for patients facing this devastating disease.

The numbers get more impressive when you dig deeper. After more than 2.5 years of treatment, seven of the original twelve patients are still alive. The drug also slowed disease progression by 39-58% and reduced breathing decline by 48%. On the safety side, there have been no deaths linked to the treatment. This suggests the drug is both effective and well-tolerated, a combination that regulators look for.

NUZ-001 works by targeting a protein called TDP-43 that clumps together in brain cells. This same problem occurs in other brain diseases like Alzheimer’s and Parkinson’s. The implication here is that if the drug works for ALS, Neurizon could potentially expand into these much larger markets down the track, giving the company multiple shots on goal.

The HEALEY Trial Could Fast-Track Approval

The HEALEY ALS Platform Trial is run by Massachusetts General Hospital and tests multiple drugs at once. This setup saves time and money because all drugs share the same comparison group. For Neurizon, this means faster results and lower costs than running a trial alone. We believe this efficient structure improves the odds of success for a small company with limited cash.

There is also a possible shortcut to the market. The FDA offers accelerated approval for drugs that treat serious diseases with few treatment options. ALS fits this perfectly. The survival and breathing improvements NUZ-001 has already shown could support this faster pathway. If Phase 2/3 data confirms the early promise, this could shave years off the path to commercialisation.

The Investor’s Takeaway for Neurizon

At around A$0.13 per share, Neurizon trades well below the A$0.39 analyst target, implying a potential upside of about 200%. The company’s market value of roughly A$70 million appears cheap for a biotech entering a major Phase 2/3 trial with this kind of early data. In our view, the market’s scepticism has created a valuation gap that could close quickly if trial results are positive.

But make no mistake, this is speculative. Neurizon has no revenue and is burning cash. It will likely need to raise more money before any drug reaches patients. The trial outcome is binary. Good results could send the stock much higher. Bad results could wipe out most of its value.

For investors comfortable with high risk, the mix of strong early data, regulatory progress, and beaten-down price makes this worth watching. Keep position sizes small, given the all-or-nothing nature of biotech trials. More cautious investors should wait for Phase 2/3 data before jumping in.