• Neurotech International says ASD patients in its Phase 1/2 trial of NTI164 have now been on treatment for more than two years
  • Initial positive results of the Phase 1/2 ASD trial have been a catalyst for the Phase 2/3 ASD study
  • NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low THC


Special Report: Autism patients participating in Neurotech’s world-first broad-spectrum cannabinoid drug therapy have crossed two years of daily oral treatment.

Clinical-stage biotech Neurotech International (ASX:NTI) has achieved a key milestone, marking more than two years of treatment on its lead cannabinoid drug NTI164 for patients involved in its Phase 1/2 ASD trial.

The trial was an open-label study to evaluate the safety and efficacy of orally NTI164 in Children with ASD, recruiting 11 ASD patients.

Held at the Paediatric Neurology Unit (PNU) at Monash Medical Centre in Melbourne, it was led by principal investigator Professor Michael Fahey, head of the PNU and director of neurogenetics.

NTI says there were no reportable serious adverse events or adverse events recorded from 90 weeks to the two-year milestone.

Importantly, NTI says no patient has dropped-out due to safety or reversal in their clinical improvements that would warrant withdrawal from treatment.


Catalyst for Phase 2/3 ASD trial

The initial positive results of the Phase 1/2 ASD trial have been a catalyst for the Phase 2/3 ASD study, also being held at PNU with Fahey as principal investigator.

NTI recently announced the Phase 2/3 trial had successfully met its primary endpoint and key secondary endpoints with comprehensive data analysis and interpretation  underway.

The Phase 2/3 trial had recruited 54 patients aged 8-17 (inclusive) with Level 2 autism (requiring substantial support), and Level 3 autism (requiring very substantial support).

The results of the Phase 2/3 trial will inform NTI discussions with the Therapeutic Goods Administration (TGA) to understand its pathway to market approval in Australia as the first market opportunity.


NTI164 aimed at neurological disorders in children

NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN.

The drug has been exclusively licensed for neurological applications globally with pre-clinical studies demonstrating a 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 recently reported further primary and secondary endpoint analysis of its Phase 1/2 trial NTIRTT1 showing significant additional benefits in Rett Syndrome girls after 12 weeks of daily oral treatment NTI164.

Earlier this month NTI reported its Phase 1/2 trial of NTI164 for PANDAS/PANS demonstrates continued symptom improvement in patients after a year of treatment, prompting caregivers to express appreciation for the positive impact on their children’s overall well-being and behaviour.

NTI has also received human ethics committee clearance for a Phase 1/2 clinical trial in spastic cerebral palsy.


Reinforce NTI164 as treatment for ASD

NTI says NTI164 is proving to be an attractive, long-term therapy for ASD, which is rising in prevalence in Australia.

ASD is estimated at 1 in 50 across the population in Australia, 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.

“Today marks an important milestone for Neurotech with our 11 level II or level III ASD patients who originally participated in the Company’s first clinical trial of NTI164 now having been on daily oral treatment with NTI164 for two years,” NTI executive director Dr Thomas Duthy says.

“We once again thank Professor Fahey and his team at Monash Medical Centre for their continued support of this ground-breaking clinical trial.

“The initial positive results of this trial have been the catalyst for further R&D investment by Neurotech which has now delivered strong clinical benefits in a further Phase 2/3 ASD study and additional clinical benefits in Phase 1/2 trials in PANDAS/PANS and Rett Syndrome without compromising patient safety,”

Duthy says all three neurological disorders lack safe and effective therapies and accordingly represent a significant commercial opportunity for NTI as it develops its core NTI164 asset.


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.