Neurizon Presentation Highlights 36 Patients Dosed in HEALEY ALS Platform Trial

Neurizon advances NUZ-001 through HEALEY trial with 36 patients dosed

In its April 2026 investor presentation, Neurizon Therapeutics outlined progress for its lead drug candidate NUZ-001, which is advancing through the Phase 2/3 HEALEY ALS Platform Trial. As at 28 April 2026, the trial had reached 36 dosed participants from 50 regimen assignments, supported by 106 master screenings across 57 activated sites. The company confirmed that the first patient was dosed in February 2026, marking the formal start of clinical execution for the registrational study. The trial targets enrolment of 160 patients, with completion anticipated during Q3-Q4 2026.

Management highlighted the registrational nature of the HEALEY trial, which operates under an adaptive Phase 2/3 design. If Phase 2 data demonstrate sufficient efficacy and safety, NUZ-001 could be eligible for accelerated regulatory approval based on these interim results. This pathway potentially shortens the timeline to commercialisation compared to traditional sequential phase structures. The trial’s progress represents a critical de-risking milestone for Neurizon, as consistent patient recruitment and dosing demonstrate operational execution capability.

The HEALEY Platform Trial model allows multiple investigational treatments to be tested efficiently within a shared infrastructure, reducing costs and timelines. For NUZ-001, this structure provides access to a validated regulatory pathway whilst maintaining capital efficiency during clinical development.

Understanding ALS drug development and the HEALEY Platform Trial model

Amyotrophic lateral sclerosis (ALS), the most common form of motor neurone disease, is a rapidly progressive neurodegenerative condition affecting nerve cells in the brain and spinal cord. Patients typically experience muscle weakness, loss of motor control, and eventually respiratory failure. Median survival from diagnosis ranges from two to five years, and approximately 30,000+ people currently live with ALS in the United States alone.

The HEALEY ALS Platform Trial represents an innovative clinical trial design known as a “platform trial,” where multiple investigational therapies are tested simultaneously against shared placebo arms. This adaptive approach enables researchers to efficiently evaluate different treatments under standardised conditions, accelerating data generation whilst reducing costs. For NUZ-001, the trial’s adaptive Phase 2/3 structure means that interim efficacy signals from Phase 2 could support accelerated approval without requiring a separate Phase 3 programme.

NUZ-001 targets TDP-43 protein aggregation, a pathological hallmark present in approximately 97% of ALS cases. Under normal cellular conditions, TDP-43 regulates RNA processing, but in ALS it misfolds and accumulates, disrupting neuronal function and contributing to cell death. Preclinical data presented by Neurizon indicate that NUZ-001 increases activity across multiple protein clearance pathways, including autophagy and the proteasomal system, which may help restore cellular proteostasis and slow disease progression.

The trial’s primary endpoint is the change from baseline through Week 36 in the ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised) total score, combined with survival data. ALSFRS-R is a validated measure of disease severity covering motor function, speech, swallowing, and respiratory capacity. Secondary endpoints include respiratory function measured via slow vital capacity and survival through Week 36.

Trial timeline and upcoming catalysts

Management provided the following milestone timeline for Regimen “I” in the HEALEY trial:

1. Q3-Q4 2026: Enrolment complete (160 patients enrolled)
2. Q2-Q3 2027: Last patient dosed
3. Q3 2027: Database lock and topline data release

Neurizon confirmed it will provide ongoing enrolment updates at key thresholds, including 50% enrolled, 100% enrolment complete, and Last Patient In. These updates will serve as interim indicators of recruitment momentum and operational execution.

Elanco supply agreement expected in Q2 2026

Neurizon outlined plans to execute a commercial supply agreement with Elanco during Q2 2026, finalising a critical component of its global licensing relationship. Under the existing exclusive global licence agreement, Elanco provides Neurizon with worldwide rights to utilise Elanco’s intellectual property for the treatment, palliation, prevention, or cure of neurodegenerative diseases in humans. The supply agreement will secure long-term access to GMP-compliant monepantel, the active pharmaceutical ingredient in NUZ-001, supporting both ongoing clinical requirements and future commercial-scale manufacturing.

Elanco has deepened its strategic involvement in Neurizon through participation in the company’s December 2025 placement, becoming a top five shareholder. The veterinary pharmaceutical company has also appointed Justine Conway, its Global Head of Business Development, as a Board Observer, signalling active strategic engagement. Management confirmed that a recent monepantel shipment has secured supply requirements through to completion of the HEALEY trial.

The pending supply agreement represents a key non-clinical milestone for Neurizon, as it establishes manufacturing pathways critical for regulatory submissions and potential commercialisation. Access to Elanco’s established manufacturing infrastructure and intellectual property reduces development risk and capital requirements compared to building proprietary manufacturing capacity.

Capital position and funding strategy

As at 31 March 2026, Neurizon held cash of $16.7 million, with net cash of $11.1 million after accounting for $5.6 million in convertible note debt. The company’s market capitalisation stood at $63.1 million, with an enterprise value of $51.8 million.

The presentation detailed Neurizon’s funding strategy, which prioritises shareholder value protection through flexible instruments and dilution minimisation:

• Raised approximately $7.1 million via placement at $0.08 per share in December 2025
• Raised $8.5 million through a 2-for-5 entitlement offer at $0.08 per share, completed via the January 2026 offer and April 2026 shortfall placement
• Secured an R&D Tax Rebate under the Advance and Overseas Finding (AOF) programme, providing at least a 43.5% cash rebate on HEALEY trial expenditure
• Established a $20 million convertible note facility with Obsidian Global GP, LLC, with $5 million drawn in February 2026 and the remaining $15 million committed but optional

The R&D Tax Rebate structure provides a non-dilutive funding source tied directly to HEALEY trial expenditure. The AOF approval binds the Australian Taxation Office and AusIndustry to provide the 43.5% rebate on qualified foreign R&D spend, creating predictable cash inflows without equity dilution. Management noted this mechanism as a key component of runway visibility through clinical milestones.

ALS market validates commercial potential

The presentation highlighted recent high-value transactions in the ALS therapeutic space as evidence of strategic acquirer interest. In April 2026, Shionogi completed its acquisition of global rights to RADICAVA (IV edaravone) and RADICAVA ORS (oral edaravone) from Tanabe Pharma for US$2.5 billion upfront, with potential additional royalties on future sales. This transaction involved a commercial, revenue-generating franchise with both intravenous and oral formulations already in market, strengthening Shionogi’s rare disease commercial platform.

Additional recent deals referenced by management include collaborations between Eli Lilly and Alchemab (2025, reported up to US$415 million) and QurAlis (2024, US$45 million upfront plus potential milestones). Eli Lilly also entered an ALS discovery collaboration with Verge Genomics in 2021, with public reports describing potential transaction economics of approximately US$694 million. Prilenia and Ferrer announced a strategic collaboration and licensing agreement for pridopidine in 2025.

Management noted that the ALS market is projected to reach US$1.28 billion by 2029, driven by a 15% annual sales growth rate and approximately 0.8% annual patient population growth. NUZ-001’s competitive positioning centres on its oral formulation and disease-modifying potential targeting the underlying TDP-43 pathology implicated in the vast majority of ALS cases. The oral route of administration offers convenience and scalability advantages over intravenous therapies, potentially supporting broader patient access if approved.

The transaction landscape demonstrates that strategic pharmaceutical companies are willing to commit significant capital to differentiated ALS therapies, particularly those with oral delivery, disease-modifying mechanisms, or established clinical validation. This backdrop supports partnership and licensing optionality for Neurizon as the HEALEY trial progresses.

Near-term catalysts to watch

Management outlined the following milestones expected across Q2-Q4 2026:

• Commercial supply agreement with Elanco (Q2)
• HEALEY enrolment updates (ongoing)
• Ethics approval for liquid formulation pharmacokinetic study
• EMA Scientific Advice preparation
• PMDA regulatory consultation (Japan)
• CEO appointment
• HEALEY enrolment complete (Q3-Q4)
• Participation at CNS Partnering, Target ALS, and other major international conferences and partnering events

These milestones span clinical execution, regulatory strategy, corporate governance, and business development. The liquid formulation pharmacokinetic study represents a supplementary development programme aimed at providing an alternative dosing option to the current oral tablet. EMA Scientific Advice and PMDA consultations position Neurizon for potential regulatory pathways in Europe and Japan, expanding the addressable commercial opportunity beyond the United States and Australia.

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