Neurotech’s NTI164 shows excellent tolerability in key preclinical toxicology studies

Neurotech clears key safety hurdle for NTI164, paving the way for longer studies and global filings.

NTI164 shows strong safety in tox tests. Image: Getty.

Special Report: Neurotech has cleared a key safety hurdle for its lead drug NTI164, with new toxicology results paving the way for longer-term studies, and global regulatory submissions.

Neurotech International (ASX: NTI)has delivered a major win in the development of its proprietary lead drug formulation, NTI164.

NTI164 is a unique cannabis-based drug being developed to treat brain inflammation in kids with neurological disorders. Early studies show it may help protect brain cells and reduce inflammation.

The company today announced positive results from its recently completed 28-day Good Laboratory Practice (GLP) oral toxicology studies.

The results show that NTI164 demonstrated excellent tolerability and no safety concerns in both rats and dogs, a strong step forward in its regulatory and clinical development pathway.

The 28-day GLP-compliant repeat-dose toxicity studies were conducted in Sprague-Dawley rats (total n=116) and Beagle dogs (total n=42), following international ICH guidelines that are widely accepted by regulators like the FDA, TGA, and EMA.

The animals were given twice-daily oral doses across low, mid and high levels, followed by a 14-day recovery period.

NTI164 was well tolerated in both species, with no systemic or organ-specific toxicities attributed to the test article.

Neurotech confirmed that “no test article-related adverse effects were observed across clinical observations, ophthalmic exams, neurological assessments, or clinical pathology.”

Critical milestone as NTI64 advances

The absence of adverse findings across clinical pathology, histopathology and neurobehavioral assessments underscores the formulation’s favourable safety profile.

Importantly, all incidental observations resolved during the recovery phase, confirming it has a strong therapeutic margin.

This strong therapeutic margin means NTI164 was well tolerated even at high doses, with a clear buffer between the effective dose and any level that might cause harm – a key safety marker, especially for treatments aimed at children.

Dr Anthony Filippis, CEO and Managing Director of Neurotech, said the outcome was a critical milestone.

“We are extremely encouraged by these robust safety findings, which mark a crucial step in our regulatory and clinical development pathway,” he said.

“The positive outcomes of this toxicology study provide a solid foundation for advancing NTI164 into longer-term toxicity studies, as required by the FDA and TGA.”

Global jurisdictions

The findings lay the groundwork for what comes next.

The results confirm that NTI164 is safe enough to progress into longer-term toxicology studies and support upcoming regulatory submissions to the FDA, TGA and EMA.

According to the company, the study’s GLP design and use of two regulatory-relevant species ensure the data will be broadly accepted across global jurisdictions.

These results form the nonclinical safety foundation needed for Investigational New Drug (IND) applications and align with both FDA and TGA guidance for progressing chronic-use treatments.

Crucially, these findings significantly de-risk NTI164 for repeat-dose use in human clinical trials, and strengthen its position for future commercialisation; especially in paediatric and neurological settings where long-term safety is paramount.

“We look forward to progressing NTI164 rapidly into the next stages of clinical development and regulatory submissions, while continuing our focus on commercial opportunities for the business,“ said Dr Filippis.

Neurotech also reiterated that it remains committed to harmonised global regulatory submissions, and will use these foundational safety results to help support the development and commercialisation of NTI164 in international markets.

This article was developed in collaboration with Neurotech, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.

Originally published as Neurotech’s NTI164 shows excellent tolerability in key preclinical toxicology studies