Neurotech Advances Phase 3 Autism Trial and Safety Data for NTI164

Phase 3 Trial Launch Marks Major Step for NTI164 in Autism

Neurotech International (ASX:NTI) has initiated the first clinical site for its pivotal Phase 3 "Beyond Harmony" trial evaluating NTI164 in children with Autism Spectrum Disorder (ASD). The Monash Children’s Hospital site in Melbourne, a leading paediatric centre, is now open for recruitment following regulatory approvals, with plans to enrol 150 patients under an adaptive trial design that allows for data-driven modifications without compromising scientific rigor. This milestone follows the Human Research Ethics Committee (HREC) approval secured earlier in the year, reinforcing the company’s momentum toward potential regulatory submissions and commercialisation of NTI164. Additional sites are expected to come online in coming months to support global trial expansion. This development builds on earlier progress, including the first clinical site activated announcement in March.

Robust Safety Profile Reinforced by 90-Day Toxicology Study

During the quarter, Neurotech reported positive outcomes from a 90-day GLP repeat-dose oral toxicology study in Beagle dogs, designed with input from the US FDA. NTI164 was well tolerated at doses up to 216 mg/kg/day, approximately ten times higher than the highest human dose tested, without any mortality or dose-limiting toxicities. Comprehensive assessments, including enhanced brain analyses, revealed no adverse neurological effects, supporting the drug’s safety for long-term use. Mild, reversible treatment-related findings were observed but resolved after a 14-day recovery period. These results bolster the company’s safety package for regulatory submissions to both the US FDA and Australian TGA, echoing earlier positive findings from the 90-day GLP toxicology study reported in February.

Scientific Validation Through PANS Data Publication

Neurotech’s lead drug NTI164 also gained independent scientific validation with the publication of clinical and mechanistic data in the peer-reviewed journal Neurotherapeutics. The study examined a sub-cohort of 14 children with Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS) treated with NTI164 over 12 weeks, showing statistically significant improvements across multiple symptom domains including anxiety, obsessive-compulsive symptoms, and ADHD. Mechanistic analyses revealed that NTI164 normalized dysregulated epigenetic, ribosomal, and immune pathways implicated in PANS, providing molecular evidence for the clinical benefits observed. This publication strengthens the scientific foundation for NTI164’s broader development strategy across neuroinflammatory paediatric disorders.

New Collaboration Targets Rett Syndrome Mechanisms

Expanding its pipeline, Neurotech announced a research collaboration with The University of Sydney to investigate NTI164’s effects on Rett syndrome, a rare neurodevelopmental disorder. Led by Professor Wendy Gold, the collaboration will utilize human-derived neuronal models to explore how NTI164 modulates disease mechanisms related to MECP2 deficiency, mitochondrial dysfunction, and synaptic abnormalities. This initiative follows Neurotech’s Phase 1 clinical study in Rett syndrome and aims to deepen understanding of NTI164’s multi-modal activity in this challenging condition.

Investor Relations and Corporate Developments

Neurotech maintained a strong investor engagement program during the quarter, including media coverage in the Herald Sun highlighting the Phase 3 trial launch and potential for NTI164 to address core autism symptoms. CEO Dr Anthony Filippis participated in the Ignite Investment Summit in Hong Kong and presented at the NWR Virtual Healthcare Conference, underscoring the company’s commitment to raising its profile. Although discussions with pharmaceutical and biotech partners have advanced, no binding agreements were signed during the quarter. Notably, Neurotech’s US Medical Advisor Dr Bonni Goldstein was appointed to the National Compassionate Care Council, enhancing the company’s influence on cannabinoid therapy policy and clinical practice in the US.

Financial Position Reflects Focused R&D Investment

Neurotech reported cash operating expenses of approximately AUD 2.2 million for the quarter, down from AUD 3.4 million in the previous period, driven mainly by research and development activities supporting clinical trials and regulatory submissions. The company closed the quarter with AUD 4.3 million in cash and equivalents, following a net cash outflow from operations. It also raised AUD 300,000 through equity issuance and continues to monitor its funding position closely. With an expected R&D tax incentive refund of approximately AUD 3.5 million in the next quarter, Neurotech anticipates a reduction in operating cash outflows, aiming to sustain its clinical programs and strategic initiatives. The company’s cash runway is estimated at just under two quarters, underscoring the importance of upcoming funding events and operational discipline.