NTI164 Gains FDA Rare Pediatric Disease Status as Neurotech Expands Access

Expanding Access Through Authorised Prescribers

Neurotech International Ltd (ASX – NTI) has taken a significant step forward in the commercialisation of its lead investigational therapy, NTI164, by launching an Authorised Prescriber (AP) program in Australia. Overseen by Professor Michael Fahey of Monash Medical Centre, this program enables specialist-led, controlled access to NTI164 for eligible children with a range of neurodevelopmental conditions. This initiative responds directly to growing demand from families seeking treatment options beyond clinical trials, offering a pathway to broader real-world use while maintaining clinical oversight and safety.

The AP program is designed to be financially self-sustaining rather than profit-driven, with pricing set to cover supply costs plus a modest margin. This approach not only facilitates wider patient access but also allows Neurotech to gather valuable real-world data, complementing ongoing clinical trials and strengthening regulatory submissions. The timing aligns strategically with upcoming reforms to the National Disability Insurance Scheme (NDIS), which will shift support pathways for children with mild to moderate autism spectrum disorder by 2027. By establishing NTI164 within this evolving framework, Neurotech aims to demonstrate both clinical and social value to healthcare policymakers.

Regulatory Momentum with FDA Rare Disease Designation

Following the quarter’s end, Neurotech secured Rare Pediatric Disease Designation (RPDD) from the US Food and Drug Administration for NTI164 in the treatment of Rett Syndrome. This designation is a critical regulatory milestone that offers several advantages, including enhanced FDA guidance, eligibility for priority review, tax credits for clinical testing, fee exemptions, and seven years of market exclusivity upon approval. It complements previously granted Orphan Drug Designations and reinforces Neurotech’s strategic positioning in developing treatments for rare paediatric neurological disorders.

NTI164 is a unique full-spectrum medicinal cannabis extract with 0.08% THC, which has shown promising results in clinical trials. A peer-reviewed publication in the Journal of Paediatrics and Child Health detailed Phase I/II trial outcomes demonstrating that NTI164 was well tolerated and produced clinically meaningful improvements in neurological, behavioural, and functional symptoms of Rett Syndrome. These findings bolster confidence in NTI164’s therapeutic potential and its proposed mechanism targeting neuroinflammation and synaptic function.

Advancing Clinical and Regulatory Programs

Neurotech is progressing its chronic toxicology studies to establish the long-term safety profile of NTI164, a prerequisite for regulatory approvals in the US and other markets. Concurrently, the company is preparing an Investigational New Drug (IND) application for submission to the FDA, a pivotal step toward initiating US clinical trials. Plans are underway for further studies, including a Phase III registration trial in autism spectrum disorder, underscoring Neurotech’s commitment to advancing NTI164 through the clinical pipeline.

Financial Position and Corporate Engagement

During the September quarter, Neurotech reduced its cash operating expenses to approximately $1.2 million, down from $2.6 million in the prior quarter, reflecting focused investment in R&D and regulatory activities. The company ended the quarter with cash and equivalents of around $1.7 million. Importantly, Neurotech anticipates receiving a $4.7 million R&D tax incentive refund in the coming quarter, bolstered by a positive Advanced Overseas Finding outcome that increased the refundable tax offset.

On the corporate front, Neurotech has maintained active engagement with industry stakeholders and potential partners, participating in major biotech conventions in the US and Australia. Several discussions have advanced to confidential stages, signaling growing interest in the company’s clinical progress and pipeline potential.