Neurotech International announces strong results from NTI164 PANDAS/PANS trial extension

Neurotech International (ASX: NTI) has announced “highly significant” improvements during the extension phase of a clinical trial into the impact of its lead candidate NTI164 on children with paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).

NTIPANS1 was the first-ever clinical trial to show improvements in PANDAS/PANS patients using the broad-spectrum cannabinoid drug therapy with excellent safety results recorded at 12 weeks.

Neurotech has now provided further evidence of the clinical utility of NTI164 in PANDAS/PANS, with a 39% improvement in patients at 24 weeks versus baseline according to the gold-standard validated measure of anxiety/depression known as the revised child anxiety and depression scale.

The severity of illness continued the improvement on the 12 weeks versus baseline results, with 32% of patients down-staged from markedly ill at baseline to mildly ill at 24 weeks.

Data analysis conducted at the six-month mark showed continued improvements in patients with no adverse or serious adverse events recorded between 12 and 24 weeks.

Strong results

Co-principal trial investigator and professor of paediatric neurology at the University of Sydney Russell Dale said the extension phase had produced some strong results.

“Following a minimum 12 weeks of daily oral administration of NTI164, our patients diagnosed with PANDAS/PANS experienced notable enhancements in their clinical functionality, coupled with an absence of safety concerns,” he said.

“Consequently, these patients opted to prolong their treatment into the extension phase of the trial for an additional year.”

“This extension has afforded us the opportunity to delve deeper into both safety profiles and clinical outcomes beyond the initial study duration, revealing continued substantial clinical advantages compared to baseline, along with marked enhancements in anxiety, depression and overall illness severity.”

Noteworthy improvements had also been observed in measures of tic severity and obsessive-compulsive behaviours, which all exhibited statistical significance alongside baseline data.

“These findings consistently bolster the case for the prolonged usage of NTI164 within this challenging-to-treat patient cohort, showcasing benefits across various critical and validated clinical metrics,” professor Dale said.

Pleasing progress

Neurotech executive director Dr Thomas Duthy said the company was pleased with the progress of the extension phase.

“The original 12-week trial demonstrated a statistically significant and clinically meaningful beneficial impact on [patient] symptoms,” he said.

“The results of today’s data release at 24 weeks highlight the durability of treatment, with patients recording further clinical improvements and […] NTI164 continuing to demonstrate an attractive safety profile devoid of any serious events that have traditionally hampered the acceptability of paediatric neurological drugs by the medical community.”

Clinical diagnosis

PANDAS/PANS is a clinical diagnosis given to children who have a dramatic onset of neuropsychiatric symptoms including obsessive-compulsive disorder, restrictive eating and repetitive tic movements.

Sufferers may also become moody, irritable, aggressive and anxious and display some level of difficulty with their schoolwork.

The NTIPANS1 trial involves paediatric patients at the Children’s Hospital at Westmead in Sydney.