NTI164 Shows No Toxicities at 10x Human Dose in GLP Study

Positive Safety Profile Confirmed

Neurotech International Limited (ASX: NTI) has announced encouraging results from a rigorous 90-day Good Laboratory Practice (GLP) toxicology study of its lead drug candidate, NTI164. Conducted in non-rodent species, specifically Beagle dogs, the study demonstrated that NTI164 was well tolerated across all dose levels, including the highest dose of 216 mg/kg/day, approximately ten times the highest dose previously tested in humans.

This milestone is significant for Neurotech, a clinical-stage biopharmaceutical company focused on paediatric neurological disorders, as it supports the drug’s potential for long-term dosing. The absence of mortality or dose-limiting toxicities throughout the study period provides a strong foundation for advancing NTI164 through the clinical development pipeline.

Comprehensive Safety Assessments

Importantly, the study reported no clinically meaningful adverse effects on body weight, food consumption, blood markers, or general health observations. Any mild treatment-related effects observed at higher doses were reversible following a 14-day recovery period, with no progressive or life-threatening toxicities detected.

Following consultations with the US Food and Drug Administration (FDA), Neurotech incorporated enhanced central nervous system (CNS) safety assessments into the study. These included detailed brain sectioning and analysis, which revealed no neurological safety concerns, further reinforcing confidence in NTI164’s safety profile.

Regulatory Implications and Next Steps

These findings bolster Neurotech’s Investigational New Drug (IND) and Therapeutic Goods Administration (TGA) safety package, critical for upcoming clinical and registration activities in both the US and Australia. Dr Anthony Filippis, Managing Director and CEO of Neurotech, emphasised the importance of demonstrating long-term safety, describing the results as a key milestone that supports NTI164’s development as a potential chronic therapy for neurological and neurodevelopmental disorders.

With a Phase III clinical study in Autism Spectrum Disorder (ASD) already cleared by human ethics committees, these toxicology results provide a timely boost to Neurotech’s clinical ambitions. The company’s broad-spectrum oral cannabinoid therapy continues to show promise across multiple paediatric neurological indications, including ASD, PANDAS/PANS, and Rett Syndrome.

As Neurotech prepares for further regulatory interactions and clinical trials, the market will be watching closely to see how these safety data translate into clinical efficacy and eventual commercialisation.