Neurotech Wins Ethics Approval for Pivotal Phase 3 ASD Trial Targeting TGA/FDA

Ethics Approval Clears Path for NTI164’s Pivotal ASD Registration Study

Neurotech International (ASX: NTI) has secured Human Research Ethics Committee (HREC) approval to commence the Neurotech NTI164 Phase 3 Trial, titled Beyond Harmony, in individuals with autism spectrum disorder (ASD) Levels 2 and 3. The registrational study is designed to support regulatory submissions to both the Therapeutic Goods Administration (TGA) in Australia and the US Food and Drug Administration (FDA). Professor Michael Fahey will lead the trial as Principal Investigator, supported by Co-Principal Investigators and a network of satellite clinical sites across Australia, with progressive expansion into the United States.

The Neurotech NTI164 Phase 3 Trial represents the final clinical hurdle before the company can submit NTI164 for market authorisation. HREC approval enables immediate progression to site activation and participant recruitment, directly advancing the therapy towards potential commercialisation in two major healthcare markets.

Key trial parameters include:

• Phase: Phase 3 (registrational)
• Target Population: ASD Levels 2 and 3
• Regulatory Pathways: TGA (Australia), FDA (United States)
• Lead Investigator: Professor Michael Fahey
• Compliance: Good Clinical Practice (GCP) guidelines

What is a Phase 3 Clinical Trial and Why Does It Matter?

A Phase 3 clinical trial is the final and largest stage of clinical testing before a drug can seek regulatory approval from authorities such as the TGA or FDA. These studies generate the pivotal data regulators require to assess safety and efficacy at scale, typically involving hundreds or thousands of participants across multiple sites.

In Neurotech’s case, the Beyond Harmony study builds on earlier-stage trials that provided the clinical foundation for this registrational design. The company has completed both Phase II/III and Phase I/II studies in ASD, which demonstrated statistically significant and clinically meaningful improvements across multiple core symptom domains.

For investors, reaching Phase 3 signals a company has successfully de-risked earlier clinical stages and is now pursuing the data package needed for market authorisation. This represents a significant value inflection point, as successful Phase 3 results can directly enable commercial launch.

Clinical trial phases explained:

1. Phase 1: Tests safety and dosing in small groups (typically 20-80 healthy volunteers or patients)
2. Phase 2: Evaluates efficacy and side effects in larger groups (100-300 patients with the condition)
3. Phase 3: Confirms effectiveness, monitors adverse reactions, and compares to existing treatments in large patient populations (300-3,000+ participants)

Building on Clinically Meaningful Prior Results

Neurotech’s Phase 3 design is informed by robust data from its completed Phase II/III randomised, double-blind, placebo-controlled trial in ASD. That study demonstrated statistically significant and clinically meaningful improvements across multiple core ASD symptom domains, alongside consistent improvements in caregiver-reported quality-of-life measures. The results were published in a peer-reviewed journal in March 2025.

The earlier-stage trials provide critical validation for the Beyond Harmony study design, reducing execution risk by confirming both safety and preliminary efficacy signals. By advancing a therapy with established clinical benefits into Phase 3, Neurotech increases the probability of meeting the endpoints required for regulatory approval.

CEO Commentary

“This HREC approval is a major regulatory milestone for Neurotech and the penultimate step in advancing NTI164 into a pivotal, Phase 3 clinical program for ASD Levels 2 and 3. Building on the strong and clinically meaningful outcomes demonstrated in our earlier studies, it is most noteworthy that Beyond Harmony has been designed to support future regulatory submissions in both Australia and the United States,” said Dr Anthony Filippis, Managing Director and CEO of Neurotech.

Trial Design and Regulatory Strategy

The Beyond Harmony study has been rigorously designed to meet the regulatory requirements of both the TGA and FDA, enabling Neurotech to pursue market authorisation in Australia and the United States through a single pivotal trial. This dual-regulatory strategy is cost-efficient and accelerates potential market entry by avoiding the need for separate studies in each jurisdiction.

The trial will be conducted in accordance with Good Clinical Practice (GCP) guidelines, ensuring methodological rigour and regulatory alignment. Professor Michael Fahey, a leading expert in paediatric neurology and neurodevelopmental disorders, will serve as Principal Investigator, supported by a group of Co-Principal Investigators. Satellite clinical sites will be activated across Australia first, with progressive expansion into the USA as recruitment advances.

Designing a single study to satisfy multiple major regulators (TGA and FDA) maximises capital efficiency whilst expanding the addressable market opportunity. Successful trial execution positions NTI164 for concurrent regulatory submissions, potentially enabling simultaneous market launches in two large healthcare markets.

What Comes Next for Neurotech?

With HREC approval secured, Neurotech will now commence study initiation activities, including site activation and participant recruitment. The company expects to provide further updates as the clinical network expands across Australia and progressively into the United States.

This marks the penultimate step before regulatory submission, with successful recruitment and trial execution now the key operational milestones to monitor. Each update on recruitment progress, site activation, and eventual data readouts will signal advancement toward potential commercialisation.

Upcoming catalysts include:

• Site activation across Australian clinical network
• Commencement of participant recruitment
• Progressive expansion into USA sites
• Interim data readouts (timing to be confirmed)
• Final data package for regulatory submission

Investment Thesis: Why This Matters for Shareholders

HREC approval for the Neurotech NTI164 Phase 3 Trial positions the company’s lead therapy on a clear registrational pathway in ASD, a large and underserved paediatric neurological market. The dual-regulatory strategy targeting both Australia (TGA) and the United States (FDA) significantly expands the addressable commercial opportunity, whilst the single-study design maximises capital efficiency.

The clinical foundation underpinning this Phase 3 trial reduces execution risk materially. Neurotech’s prior Phase II/III study delivered statistically significant and clinically meaningful improvements across multiple core ASD symptom domains, with peer-reviewed publication validating the results. This de-risks the probability of meeting Phase 3 endpoints, as the study design has been informed by robust earlier-stage data.

Strategically, the Beyond Harmony study aligns with Neurotech’s long-term commercialisation objectives and global market entry strategy. Successful trial execution would generate the high-quality data required to support regulatory submissions in two major markets, positioning NTI164 for potential market authorisation and commercial launch.

Ethics approval for a registrational Phase 3 study is a major de-risking event for biotechnology investors. It provides visibility on a defined pathway toward potential market authorisation, with near-term catalysts including site activation, recruitment progress, and eventual data readouts. For Neurotech (ASX: NTI), this milestone transforms NTI164 from an early-stage clinical asset into a late-stage therapy pursuing regulatory approval, fundamentally altering the company’s risk-reward profile.

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