Neurotech International progresses plans for NTI164 trial in Rett syndrome

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By Lorna Nicholas - 
Neurotech International ASX NTI164 trial Rett syndrome autism spectrum disorder paediatric inflammatory neurological conditions

Neurotech International is also assessing NTI164 in treating autism spectrum disorder and other paediatric inflammatory neurological conditions.


Clinical stage biopharmaceutical company Neurotech International’s (ASX: NTI) plans for a phase II trial evaluating its NTI164 candidate in children with Rett syndrome are progressing, with final regulatory approvals expected in the current quarter.

The company launched the clinical program in late March and its plans to investigate NTI164, which is a cannabinoid-based drug in up to 15 female Rett patients aged five to 20 years.

This trial will involve a daily oral dose of NTI164 and will be conducted at the Royal Children’s Hospital and Monash Children’s Hospital, which are both Melbourne.

Earlier this month, the company added another proposed site for the trial, which Associate Prof Carolyn Ellaway of Sydney Children’s Hospitals Network will lead.

Associate Prof Ellaway is a clinical geneticist and is a senior staff specialist at the NSW Genetic Metabolic Disorders Service, and works in Metabolic Genetics at The Children’s Hospital of Westmead.

Neurotech expects to receive Human Research Ethics Committee approval and clinical trial notification scheme clearances for the trial in the current quarter.

Patient recruitment is then scheduled to begin in the second half of this year.

Neurotech is focused on developing treatments for paediatric neurological disorders that have persistent neuroinflammation.

Rett syndrome trial

Rett syndrome is a rare genetic neurological and developmental disorder with no cure.

In the phase II study, 12-week primary end points will be assessed via the Rett syndrome behaviour questionnaire, clinical global impression scale improvement and severity of illness.

Secondary end-points to be assessed include safety, adverse events, and measures associated with hand function, motor skills, communication, and quality of life.

If the study is successful, Neurotech will undertake a 14-week phase II trial in 34 patients to further determine efficacy and safety.

Autism spectrum disorder

Neurotech is evaluating NTI164 in other conditions including Autism Spectrum Disorder (ASD).

The company revealed the full results in March of a phase I/II clinical trial evaluating NTI164 in paediatric patients with the disorder.

Patients underwent 52 weeks of daily treatment in the trial and results shows “strong safety and efficacy”.

Additionally, Neurotech noted there were “significant improvements” in severity of the illness, social responsiveness and adaptive behaviour.

Results from this research will underpin United States Food and Drug investigational new drug (IND) enabling clinical studies in the United States.

A virtual pre-IND was held with the FDA to discuss development plans for NTI164 in ASD, which gave Neurotech valuable information for progressing the treatment.

Neurotech also has permission to extend the phase I/II trial for six months on a patient-specific basis.

Under the arrangement, individual patients can continue to receive NTI164 daily for up to 80 weeks.

This will generate more safety data for Neurotech and enhance regulatory submissions for further trials in ASD or other paediatric neurological disorders.

Neurotech pointed out that it was encouraged by the attention ASD was attracting in the media and in government including costs to Australia’s National Disability Insurance Scheme.

“A new study out of Western Australia has highlighted the potential cost savings to be made from early intervention in ASD, together with improved outcomes for the individual,” the company stated.


Neurotech has also locked-in ethics approval and clearance to begin a phase I/II clinical trial of NTI164 in paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).

This trial is being carried out at The Children’s Hospital in Westmead and the Paediatric Neurology Unit at Monash Medical Centre.

It is planned 15 paediatric patients will be recruited with a moderate-to-severe PANDAS/PANS diagnosis.

The trial aims to determine the efficacy and safety of NTI164 administered orally on a daily basis.

Results are expected in the second half of the year.

Neurotech noted it remains fully-funded to complete its planned clinical studies.