On track: Analysts hail FDA lift as turning point for Neurizon’s ALS therapy ambitions

• Analysts say lifting of clinical hold by US FDA on Neurizon’s lead drug candidate NUZ-001 removes “major regulatory overhang” 
• Decision means NUZ-001 can progress to phase II/III trial stage through the Healey ALS Platform with first patient enrolment targeted for end of CY25
• Petra Capital and Morgans expect renewed investor interest in Neurizon at it progresses toward a trial start and data readouts 

Analysts have reacted positively to news that the US Food and Drug Administration (FDA) has lifted the clinical hold on Neurizon Therapeutics’ (ASX:NUZ) lead drug candidate NUZ-001 (monepantel) for treatment of amyotrophic lateral sclerosis (ALS) – the most common form of motor neurone disease (MND).

The FDA’s decision to lift the IND clinical hold on Neurizon’s repurposed animal drug NUZ-001 represents a major regulatory milestone, with analysts saying it removes a “major regulatory overhang” that has been on Neurizon since the hold was first placed in January.

“The IND acceptance follows a comprehensive package of preclinical safety data and manufacturing information, bolstered by NUZ’s strategic licensing agreement with Elanco,” Morgans’ healthcare analyst Iain Wilkie wrote in a note to clients.

The FDA decision means NUZ-001 can progress to a phase II/III trial stage, which will be undertaken through the Healey ALS Platform. The significant ongoing program in the US aims to accelerate ALS treatment development and test several potential drug candidates at the one time.

“This lift marks a major inflection point for the company, clearing the way for NUZ-001 to enter the Healey ALS Platform Trial at Mass General Hospital (MGH),” Wilkie wrote.

“With the IND now active, Neurizon is set to move quickly, with MGH expected to make an IND protocol amendment to include NUZ-001 into its trial, and first patient enrolment targeted by the end of the year.”

In a note to clients Petra Capital senior healthcare analyst Tanushree Jain wrote the FDA’s acceptance of the IND was a key milestone and “de-risking catalyst” for Neurizon, establishing the foundation for advancing NUZ-001 towards a potential disease modifying therapy for ALS.

“The IND approval essentially confirms that all the pre-clinical, clinical and manufacturing data generated by NUZ and by Elanco on the active ingredient monepantel has now passed FDA’s scrutiny, underscoring the robustness of the data,” she wrote.

“This has positive read through for future expansion of the drug to other neurodegenerative conditions and is also likely to enhance NUZ’s credibility with potential partners.”

Interim analyses may be mid CY26

Wilkie wrote the Healey Platform continued to expand, with Eli Lilly’s LY3873862 recently added as the newest treatment regimen and once started data was expected in a condensed timeframe.

Including NUZ-001 and LY3873862 Healey will have two active regimens in its latest cohort and Wilkie wrote it may look to add a couple more in time.

“If NUZ-001 enrolment begins by the end of the year, the first patients will complete the 36-week treatment period by Q3 CY26,” he wrote.

He noted the protocol for Healey had been optimised to improve statistical power, lengthen treatment periods and refine patient selection criteria, increasing the potential for meaningful efficacy signals.

Interim analyses may be conducted before full cohort completion, providing early insights into the drug’s safety and effectiveness.

“Assuming these trial commencement and recruitment rate timeframes hold, interim analyses could be conducted as early as mid-2026, depending on recruitment velocity and the protocol’s statistical plan,” he wrote.

“Topline data for the full cohort could be available by late 2026 or early 2027 and interim efficacy and safety signals may be released earlier if the trial reaches pre-specified milestones, along with the potential for accelerated approval.”

In August Neurizon reported positive topline results from an open-label extension (OLE) study, which reached its primary endpoint, showing long-term treatment with NUZ-001 at the phase II dose was safe and well tolerated.

Preliminary efficacy findings showed NUZ-001 treatment, compared with matched historical controls from the PRO-ACT ALS database, demonstrated a significant survival advantage, slowed global functional decline, reduced respiratory deterioration, and stable or downward biomarker trends.

The PRO-ACT database is the largest publicly available ALS clinical trial dataset and serves as a critical external control.

Catalyst for new investor interest

As Neurizon advances toward a trial start and data readouts, Wilkie expects renewed investor interest in the stock.

“With a validated IND and a clear path into Healey, we expect renewed investor interest and momentum in the stock as the company advances towards trial and data,” he wrote.

“We view NUZ as a strong proposition in the rare disease space with significant near-term catalysts in a condensed timeframe and precedent for an accelerated approval pathway.

“While considerable clinical risk remains, we view NUZ-001 as a drug with a sound scientific basis in ALS, strong safety profile and promising hint of potential efficacy above existing treatments.”

Jain is also confident in renewed investor interest in Neurizon as it prepares to enter the Healey trial.

“We now expect investor focus to shift to the attractive commercial opportunity in ALS (the most common form of motor neurone disease) and the relatively short timelines to market for NUZ-001,” she wrote.

Petra Capital has lifted its target price to 56 cents per share, retaining a buy rating, and now assumes a 35% probability of success for NUZ-001 in ALS, up from 30% previously.

The firm increased FY28 NPAT forecasts by 30% based on higher risk-adjusted revenue assumptions, while FY26 and FY27 forecasts remain unchanged.

Well funded to move NUZ-001 forward

Jain said Neurizon was also financially well-positioned to advance its development program for NUZ-001.

“We estimate NUZ has proforma cash of ~$10.9m (incl. net proceeds of ~$4.9m from recent placement and an expected $4.1m net R&D rebate in Q4 CY25),” she said.

“In our view, these funds support NUZ’s speedy progression towards dosing commencement in the HEALEY ALS trial, which would be the next key inflexion point.”

Both analysts emphasise that the next 12–24 months could be transformative for Neurizon, given the short timelines to trial initiation, condensed follow-up periods, and potential for early data readouts.

Petra Capital, as already noted further above, maintains a buy rating, while Morgans has maintained its speculative buy rating, with a risk-adjusted 12-month target price of 39 cents per share, slightly down from 42 cents reflecting recent placement dilution.

Neurizon officially lists on US OTCQB

In further good news for Neurizon this week, the biotech has officially listed on the US OTCQB Venture Market under the code NUZTF.  The listing allows US investors to trade Neurizon’s ordinary shares in US dollars during US market hours — a move expected to boost liquidity across its primary ASX trading and enhance visibility among international investors.

To further strengthen its North American presence, Neurizon has appointed leading US investor relations firm Integrous Communications, recognised for its deep expertise in the life sciences sector.

Neurizon said the expansion into the US market, ahead of its planned entry into the Healey ALS Platform Trial, followed growing industry and investor engagement.