Autism Spectrum Disorder (ASD) affects 1 in 150 Australians. Boys are 4 times more likely to have it than girls, and symptoms are noticeable at around 2-3 years of age.

According to the Australian Institute of Health and Welfare, the number of people with ASD in Australia is increasing, providing an impetus for biopharmaceutical company Neurotech International (ASX: NTI) to continue development of their drug NTI164 for the treatment of ASD.

The cannabinoid based drug, licensed from Dolve Cann Global is the world’s first full-spectrum medicinal cannabis product (with less than 0.3% THC).

The Company has successfully completed a Phase I/II study in children affected by ASD, led by Professor Michael Fahey at Monash Children’s Hospital in Melbourne.

The study evaluated NTI164’s safety profile in a dose-escalation model. Investigators measured the drug’s effect on behaviour, focus and cognition via a range of widely accepted neuro-psychological tools.

The open-label study saw children between the ages of eight and seventeen with a diagnosis of Level I or II ASD dosed with one of four amounts. Carers and physicians were also involved in the study by answering questionnaires looking at things like anxiety, participation, irritability, hyperactivity, mood and self stimulation in ASD affected children.

The drug was well tolerated at all dose levels, with no changes detected in patients’ blood, liver or kidney function. Additionally, the medication did not affect any vital signs or weight. Parents and caregivers also reported improvement in their child’s overall functioning compared to baseline. A total of 93% of patients showed some sort of symptom improvement relating to illness severity within 28 days of treatment with NTI164.

The current standard of care treatment for ASD patients is speech and language therapy, occupational therapy and cognitive behaviour therapy. Pharmacologically, the only drug approved for ASD is Risperidone, prescribed to assist with irritability. Risperidone is an antipsychotic that rebalances chemicals in the brain to improve the ability to think, mood and behaviour. Common side effects of the drug are headaches, anxiety, involuntary muscle movements and drowsiness.

NTI produced no side effects in trial participants, leading to higher patient compliance- a promising start for the new drug.

“We are extremely pleased with these breakthrough results for NTI164,” said Neurotech CEO, Dr Alexandra Andrews. “The fact that 93% of participants have shown notable improvements without any serious side effects is an outstanding outcome. We are incredibly grateful for the generous participation of patients and their families as well as the team of staff and clinicians at Monash Children’s hospital who have made this landmark trial possible. 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.”

Neurotech is now in discussions with the US FDA for pre-Investigational New Drug status. The application is scheduled to be lodged later this year.

Additionally, the Company has commenced discussions with Australian regulator, the Therapeutic Goods Administration (TGA) to map out a full regulatory development pathway for the commercialisation of the drug.