Neurotech International (ASX: NTI) has reported favourable NTI164 toxicology study results, with its lead drug candidate demonstrating strong long-term safety across a 90-day preclinical study. The highest dose tested (216 mg/kg/day) was approximately 10 times the highest human dose evaluated to date, with zero mortality and no dose-limiting toxicities observed.
NTI164 clears critical 90-day safety hurdle with no dose-limiting toxicities
The GLP (Good Laboratory Practice) repeat-dose oral toxicity study was conducted in Beagle dogs in accordance with US FDA requirements. NTI164 was administered twice daily over the full 90-day period, with all animals completing the planned dosing schedule.
The study’s results strengthen Neurotech’s IND/TGA-enabling safety package, required to support further clinical and regulatory activities under US FDA and Australian TGA registration guidelines. For a drug targeting paediatric neurological disorders, demonstrating safety at multiples of the intended human dose is a critical regulatory milestone.
Key findings from the study:
- No mortality recorded at any stage across all dose levels
- Well tolerated at the highest administered dose of 216 mg/kg/day
- Regulatory support strengthened for IND/TGA submissions
- No clinically meaningful adverse effects on body weight, food consumption, haematology, or coagulation parameters
- No CNS safety concerns identified following FDA-guided assessments
Long-term safety data acts as a critical gatekeeper for regulatory advancement. Clearing this milestone de-risks the clinical pathway and supports the company’s ability to pursue chronic therapy indications in neurological and neurodevelopmental disorders.
What is a GLP toxicology study and why does it matter?
GLP toxicology studies are regulatory requirements mandated by authorities like the FDA and TGA before drugs can advance to certain clinical stages or registration activities. Non-rodent studies (in this case, Beagle dogs) are standard regulatory practice to assess safety in a second species, providing additional confidence in a drug’s safety profile.
A 90-day repeat-dose study assesses chronic exposure safety, which is particularly relevant for drugs intended as long-term therapies rather than acute treatments.
Why this matters to investors:
- GLP toxicology data is non-negotiable for IND (Investigational New Drug) applications in the US and TGA submissions in Australia
- Successful completion removes a significant regulatory hurdle and supports the pathway toward Phase III and registration activities
- The absence of dose-limiting toxicities at 10 times human dose levels provides a substantial safety margin for future clinical development
No CNS safety concerns following FDA-guided assessments
Following direct consultation with the US FDA through Type C and Type D meeting interactions, additional central nervous system-focused safety assessments were incorporated into the study design. Enhanced brain sectioning and detailed neurological analysis were conducted to address this critical area of potential regulatory scrutiny.
The expanded evaluations did not identify any safety concerns related to CNS function. For a drug targeting paediatric neurological disorders including Autism Spectrum Disorder (ASD), PANDAS/PANS, and Rett Syndrome, demonstrating CNS safety is fundamental to regulatory progression.
This proactive FDA engagement demonstrates regulatory sophistication and addresses a potential objection point before it can arise in formal regulatory submissions. The consultation and subsequent assessments reflect the company’s strategic approach to de-risking its regulatory pathway.
Reversible findings and full recovery reinforce tolerability
Any treatment-related findings identified at higher dose levels were mild in nature and fully reversible. A 14-day recovery period (non-dosing phase) demonstrated complete resolution of these effects, with all parameters returning to baseline.
Importantly, there were no clinically meaningful adverse effects on body weight, food consumption, haematology, coagulation parameters, or general clinical observations throughout the dosing phase. All animals completed the study with no treatment-related early terminations, and no progressive, irreversible, or life-threatening toxicities were identified.
| Parameter | Outcome |
|---|---|
| Mortality | None |
| Dose-limiting toxicity | None |
| Body weight impact | No clinically meaningful effect |
| Haematology/coagulation | No clinically meaningful effect |
| Recovery (14 days) | Full resolution |
The combination of tolerability at high doses and complete reversibility of mild effects supports NTI164’s profile as a potential chronic therapy suitable for long-term administration.
Building the regulatory pathway toward Phase III
This toxicology milestone arrives at a strategic inflection point in Neurotech’s development programme. The company has completed a Phase II/III randomised, double-blind, placebo-controlled clinical trial in ASD with statistically significant results reported across clinically-validated measures alongside excellent safety.
In addition to the completed ASD trial, Neurotech has completed and reported statistically significant and clinically meaningful Phase I/II trials in PANDAS/PANS and Rett Syndrome. Ethics committee clearance has been received for a Phase III clinical study in ASD.
The addition of robust long-term safety data to this clinical foundation positions NTI164 for its next development stage. With Phase II/III efficacy data in hand, Phase III ethics clearance secured, and now comprehensive preclinical safety data supporting chronic dosing, the regulatory package is strengthening across multiple fronts.
Management perspective on the milestone
Dr Anthony Filippis, Managing Director and CEO
“Demonstrating long-term safety is one of the most important milestones in drug development. These results provide additional non-clinical safety data for NTI164 and support its development as a potential chronic therapy for neurological and neurodevelopmental disorders. This study also strengthens our safety package for future regulatory interactions.”
The emphasis on long-term safety reflects the regulatory and commercial reality for drugs targeting chronic paediatric conditions, where safety profiles must support years of continuous use.
Company context
Neurotech International (ASX: NTI) is a clinical-stage biopharmaceutical company focused on paediatric neurological disorders. Its lead candidate NTI164 is a broad-spectrum oral cannabinoid therapy targeting conditions including ASD, PANDAS/PANS, and Rett Syndrome.
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