Neurotech has released promising results from a world first study of a medicinal cannabis product in children which could prove to be a game-changer in autism treatment.

Neurotech International (ASX:NTI) has released promising results from a Phase I/II clinical study of its proprietary NTI164 cannabis strain in paediatric Autism Spectrum Disorder (ASD).

Results included 93% of patients showing symptom improvement after 28 days of daily treatment with NTI164.

The study was conducted by Head of Paediatric Neurology at Monash Children’s Hospital in Melbourne Professor Michael Fahey.

NTI164 is one of NTI’s proprietary cannabis strains, exclusively licensed from Dolce Cann Global, in respect of neurological applications.

It is the world’s first full-spectrum medicinal cannabis product (less than 0.3% THC) to be successfully studied in children with ASD.

Crucial foundational study

The Phase I/II clinical study was designed to rigorously assess safety of NTI164 in a dose-escalation regime and to detect a signal for efficacy on the behaviour, focus and related cognitive parameters using a range of validated neuro-psychological tools.

It was also designed to form the foundation for follow-up studies in therapies relating to the treatment of a wide range of neurological disorders such as attention deficit hyperactivity disorder (ADHD), multiple sclerosis, motor neuron disease, rett syndrome and cerebral palsy.

The open-label study was conducted on 14 children aged from eight to 17 that have a medical diagnosis of Level II and III ASD, as confirmed by the Autism Diagnostic Observational Schedule (ADOS-2) criteria.

Safety and tolerability were assessed across dose regime including 5mg/kg, 10mg/kg, 15mg/kg and 20mg/kg.

Safety was monitored and measured by clinical examination including full blood examinations, liver, and renal function tests.

Efficacy was measured through parent/carer and physician questionnaires to assess parameters including, but not limited to:

  • Anxiety
  • Participation
  • Irritability
  • Hyperactivity
  • Mood, and
  • Self-stimulation

In total, over 2,250 assessment points were created through the landmark study.

Key study results

The safety data concluded that NTI164 at 5, 10, 15 and 20mg/kg administered in two doses daily, is safe and well-tolerated in the study population.

No changes were observed in patients’ full blood examination, liver function or kidney function tests. There were also no changes observed in the patients’ vital signs or weight.

Statistical analysis of key assessments, including Clinical Global Impression of Severity of Illness (CGI-S) demonstrated statistical significance at 28 days of treatment.

Other key results included:

  • 64% of patients had a global improvement of “much improved'”
  • 29% of patients had a global improvement of “minimally improved”
  • 7% of patients had “no change”
  • Two patients recorded a Marked Therapeutic Index Score of 2 = vast improvement and meaning complete or near remission of all symptoms.
  • 10 patients recorded a Moderate Therapeutic Index Score of 5 & 6 = representing decided improvement, meaning partial remission of symptoms.
  • The average rating for the severity of illness at baseline was 4.4. This was reduced to 3.6 after 28 days of NTI164 treatment.

Importantly, parental/carer observations also indicated consistent improvement in the trial participant’s ‘overall functioning’ when compared to baseline at the trial start.

Specific instances of markedly improved behaviours such as reduction in fear, agitation and anxiety which were observed will be the key focus of upcoming Phase II/III registration trials due to commence in calendar Q3 2022.

Encouraging results for game-changing therapy

NTI is in discussions with the US Food and Drug Administration (FDA) regarding pre-IND (investigational new drug) scheduled for later this year.

The only drug currently approved by the FDA for children with ASD is Risperidone and is  prescribed for children to assist with irritability.

Common side effects include headaches, drowsiness, anxiety, and uncontrollable muscle movements.

NTI has also initiated discussions with the Therapeutics Goods Administration (TGA) to assess product scheduling and classification for the Australian market.

Given the NTI trial results show no serious adverse side effects and high patient compliance, the company is well placed to make significant inroads into the ASD market expected to be worth more than US $5 billion by 2028.

Professor Fahey says he is very encouraged by these results with the study designed to be rigorous on all fronts to assess the potential application of NTI164 for the treatment of ASD.

“We are encouraged by the efficacy shown by NTI164 in this trial and we are looking forward to the extension of this trial, in addition to the planned initiation of a Phase II/III trial, to further assess the long-term safety and efficacy of NTI164 with the potential to lead to drug registration,” he says.

Neurotech International Chairman, Brian Leedman says results from the trial should not be underestimated.

“NTI is now a significant step closer in the drug development timeline to introducing to the market a treatment option for paediatric ASD which is natural, safe, and based on the results to date, offers substantial behavioural improvements in ASD,” he says.

Neurotech International CEO, Dr Alexandra Andrews, said she was extremely pleased with these breakthrough results for NTI164.

The fact that 93% of participants have shown notable improvements without any serious side effects is an outstanding outcome.

“It is heart-warming to think we are in a position to effectively support children with autism and their caregivers by providing a new therapeutic option that may improve their quality of life.”

The study has HREC approval to continue for a further 54 weeks with safety and efficacy assessments to be ongoing.

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.

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