Neurotech eyes further clinical trial for lead drug candidate after more promising autism spectrum disorder results
Daily treatment of ASD patients with NTI-164 has shown improvement in severity of illness, socialisation and adaptive behaviour.
Emerging drug developer Neurotech International (ASX: NTI) has reported further positive results from first-in-human clinical trialling of its lead drug candidate NTI-164 to treat children with Autism Spectrum Disorder (ASD).
With 11 evaluable children in the phase I/II trial, the results showed a “significant benefit across a large number of clinically validated assessments” at the 52-week mark.
Exclusively licenced by the company globally for neurological applications, NTI-164 is a proprietary drug formulation derived from several cannabinoid combinations, with low levels of the psychoactive ingredient THC.
Carried out at Melbourne’s Monash Children’s Hospital, the trial was initially planned for 28 days, but following promising initial results it was extended to 52 weeks and then another six months.
Commencing in May 2021, the trial enrolled 14 patients aged between eight to 17 years, with level two or level three ASD.
The patients were measured on the criteria of severity of illness, social responsiveness and adaptive behaviour, relative to the base line of the start of the trial.
Supporting early results
Supporting the 20-week results that indicated “excellent” safety and efficacy, the 54-week results showed a 30% improvement, in terms of reclassification of patients from ‘moderately ill’ at baseline to ‘borderline’ or ‘mildly ill’.
Ten of the patients (90%) recorded a score of “much improved” or “very much improved”.
The read-out also showed no serious adverse events and no change in blood analysis or liver function tests.
The trial’s lead investigator Professor Michael Fahey said the results continued to show a “global improvement” in the severity of illness, socialisation and adaptive behaviour. These benefits were coupled with a lack of serious adverse events and “clean” pathology results.
“We certainly look forward to the next phase of this exciting development opportunity in ASD,” he said.
Larger study planned
Neurotech executive director Dr Thomas Duthy said the company hopes the results will be replicated in a large phase II/III trial. This double-blind, placebo-controlled clinical study will also be carried out at the Monash Children’s Hospital and is expected to commence in the 2023 December quarter.
The company also hopes to lodge an Investigational New Drug Application with the US Food and Drug Administration (FDA) – paving the way for an expanded trial to support an eventual marketing application.
Autism Spectrum Disorder
About one in 44 US children are affected by ASD, with the current market valued at US$1.85 billion (A$2.76 billion).
However, current treatments are known for a range of side effects including nausea, anxiety and stomach pains.
Other trials for NTI-164
While ASD is Neurotech’s most advanced program, the company also recently enrolled the first of 15 planned patients to test NTI-164 for two difficult-to-treat conditions called PANDAS and PANS.
PANDAS is paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections and a subset of PANS (paediatric acute-onset neuropsychiatric syndrome).
The company noted the success of peer Neuren (ASX: NEU), which this week saw its market valuation soar on the back of FDA assent for its treatment for Rett syndrome, a rare genetic neurological disorder that mainly affects girls.