Special Report: Clinical-stage biopharmaceutical development company Neurotech International is on track to deliver results for its Phase 2/3 NTIASD2 clinical trial for children with autism spectrum disorder (ASD) in Q1 CY24, with all patients now recruited.

Neurotech International (ASX:NTI) has completed recruitment for its Phase 2/3 NTIASD2 clinical trial with a total of 56 patients enrolled, all with level 2 (requiring substantial support) or level 3 (requiring very substantial support) autism.

 NTI says all patients were enrolled at the Paediatric Neurology Unit at Monash Medical Centre through the trial’s principal investigator Professor Michael Fahey, who is head of the unit and director of neurogenetics.

The trial covers treatment of NTI’s proprietary lead drug formulation NTI164, derived

from a unique cannabis strain with low THC and a novel combination of cannabinoids, including CBDA, CBC, CBDP, CBDB and CBN.

The drug is exclusively licenced for neurological applications globally with pre-clinical studies demonstrating its potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.

NTI164 is being developed as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.

The company says trial results are forecast for Q1 CY24.


Study to determine efficacy and safety

The Phase 2/3 randomised, double-blind, placebo-controlled study will determine the efficacy and safety of NTI164 versus placebo.

The study comprises an eight-week treatment period followed by an eighth-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.

The primary endpoint of the trial is 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.

Key secondary endpoints include changes in adaptive behaviours, social responsiveness, anxiety, depression and mood scale along with safety as measured by full blood, liver and kidney analyses at defined time points.

Results of its earlier trial show strong safety and efficacy, with significant improvement across many assessments versus baseline (Day 0).

Earlier this month, NTI announced Human Research Ethics Committee (HREC) approval to extend its Phase 2/3 clinical trial in ASD to allow patients who turn 18 to remain on treatment with its proprietary NTI164 during the extension phase for up to 54 weeks of total treatment.


Need for effective ASD treatment

NTI executive director Dr Thomas Duthy says completion of recruitment in the “world-first clinical trial” is exciting and seeks to build upon previous positive results, but with a much larger patient population.

“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,” Duthy says.

“It has been shown to significantly improve adaptive behaviours and socialisation and improve these children’s quality of life while reducing caregiver burden.

“The results of this trial will inform our discussions with the TGA to understand our pathway to market approval in Australia as our first market opportunity, where the prevalence of autism is estimated at 1 in 50 across the population, representing a 40-fold increase in the last 20 years.”


This article was developed in collaboration with Neurotech International, 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.