Special Report: Neurotech has treated the last patient in its Phase II/III NTIASD2 clinical trial for children with Autism Spectrum Disorder. The trial, aiming to confirm the therapeutic effects of NTI164 observed in previous trials, seeks to address the urgent need for new treatments in Australia, where autism prevalence is rising.
Neurotech International (ASX:NTI) has reached a milestone in its Phase II/III NTIASD2 clinical trial for children with Autism Spectrum Disorder (ASD), after the 54th and last patient in the trial completed their last visit (LPLV).
The trial had recruited patients aged 8-17 (inclusive) with Level 2 autism (requiring substantial support), and Level 3 autism (requiring very substantial support).
All of these patients have now completed 8 weeks of daily NTI164 treatment (the randomisation period of the trial) at the Paediatric Neurology Unit at Monash Medical Centre.
The trial was led by principal investigator Professor Michael Fahey, head of the PNU and director of neurogenetics.
Dr Thomas Duthy, executive director of Neurotech, said the eight-week randomisation phase of this world-first clinical trial seeks to confirm the therapeutic effects of Neurotech’s broad-spectrum cannabinoid therapy NTI164, which had already shown positive results in a previous trial.
“With the explosion in autism-associated costs under the Australian National Disability Insurance Scheme, there is an urgent need for new enabling treatments like NTI164, which has been shown to significantly improve adaptive behaviours and socialisation and improve these children’s quality of life in an earlier clinical trial,” said Duthy.
The results of the NTIASD2 clinical trial are expected in early Q2 this year.
More about the Phase II/III NTIASD2 trial
NTIASD2 is a randomised, double-blind, placebo-controlled, Phase II/III clinical trial that has recruited 54 patients with ASD to determine the efficacy and safety of NTI164 versus placebo.
The study comprises an eight-week treatment period, followed by an eight-week open-label maintenance period, followed by a two-week wash-out period.
Participants who choose to continue receiving NTI164 beyond the duration of the study may do so for an additional 38 weeks. They will undergo the two-week down-titration phase at the end of their extension phase.
The primary endpoint of the trial is the Clinical Global Impression-Severity (CGI-S), which reflects a clinician’s impression of severity of illness on a 7-point scale ranging from 1 = “not at all” to 7 = “among the most extremely ill”.
Secondary endpoints, meanwhile, include:
- Change in Vineland Adaptive Behaviour Scales
- Change in Social Responsiveness Scale
- Change in Clinical Global Impression Scale -Improvement (CGI-I)
- Change in Anxiety and/or Depression
- Mood Scale (ADAMS); and,
- Safety as measured by full blood, liver and kidney analyses at defined time points.
“The results of this trial will inform our discussions with the Therapeutic Goods Administration to understand our pathway to market approval in Australia as our first market opportunity,” said Duthy.
The prevalence of autism in Australia is estimated at 1 in 50 across the population, which represents a 40-fold increase in the last 20 years.
More recent 2023 data from the NDIS agency suggests a prevalence rate of 1 in 25 for 7-14-year-olds with a primary diagnosis of autism in Australia, which is among the highest rates in the world.
NTI164 is also being trialled for other indications
In earlier clinical trials, daily treatment of NTI164 over a 52-week period had shown strong safety and efficacy effects on ASD patients.
After 52 weeks of treatment, results demonstrated that of the ~40% of subjects markedly or severely ill at baseline, none of these patients from week 4 onwards were classified as markedly to severely ill.
NTI164 is a compound derived from a unique cannabis strain with low THC. The cannabinoids include CBDA, CBC, CBDP, CBDB and CBN.
Apart from autism, NTI164 is also being studied in various clinical trials under Neurotech’s broad paediatric program.
Rett Syndrome is the latest indication to be targeted for NTI164’s application. The company has recruited 14 Rett Syndrome female paediatric patients for the Phase I/II clinical trial, all of whom will receive NTI164 treatment for 52 weeks.
In addition, NTI64 is being trialled in the PANDAS/PANS Phase I/II trial, which has already shown encouraging results after 24 weeks of treatment.
PANDAS (Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections) and PANS (Pediatric Acute-Onset Neuropsychiatric Syndrome) represent a collective condition.
Children with PANDAS/PANS go to sleep in a normal state but awaken with uncontrollable tic movements in their hands and legs, coupled with severe obsessive-compulsive behaviours, heightened anxiety, and depression.
This article was developed in collaboration with Neurotech International, a Stockhead advertiser at the time of publishing.
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