Neurotech Phase I/II clinical trial extension results show further effectiveness of NTI164 in PANDAS/PANS
Patients continue to improve significantly between 12 and 24 weeks with no adverse or serious events recorded
Neurotech to seek early dialogue with regulatory bodies about clinical and regulatory development of NTI164
Special Report: Neurotech reports paediatric patients in the PANDAS/PANS Phase I/II clinical trial extension phase continue to show significant improvements between 12 and 24 weeks of treatment.
Clinical-stage biopharmaceutical development company Neurotech International (ASX:NTI) today announced positive clinical data in the extension phase of its PANDAS/PANS Phase I/II trial (NTIPANS1) evaluating its broad spectrum cannabinoid therapy NTI164.
The original Phase I/II trial reported 12-week data in October 2023, showing statistically significant and clinically meaningful improvements across a range of gold-standard, clinically validated assessments.
As a result, all 15 paediatric patients entered the 54-week extension phase at The Children’s Hospital at Westmead in Sydney with the clinical assessments at 24 weeks.
The assessments found that patients continue to improve significantly between 12 and 24 weeks with no adverse or serious events recorded for the same period.
Improvements were recorded at 24 weeks versus baseline relating to the gold-standard validated measure of anxiety/depression RCADS-P with 39% improvement, p=0.0008 versus 30%, p=0.016 at week 12.
Severity of illness (CGI-S) continued to improve with patients down-staged from markedly ill at baseline to mildly ill at 24 weeks (32% improvement, p=0.00007), which was a further improvement from 12 weeks versus baseline (18%, p=0.0005).
PANDAS/PANS treatment need
PANDAS (Pediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections) and PANS (Pediatric Acute-Onset Neuropsychiatric Syndrome) represent a collective condition.
Children go to sleep in a normal state but awaken with uncontrollable tic movements in their hands and legs, coupled with severe obsessive-compulsive behaviours, heightened anxiety, and depression.
This disorder is triggered by dysregulation caused by streptococcus or other unidentified pathogens, initiating an autoimmune response leading to inflammation in the brain.
PANDAS/PANS are categorised as rare orphan disorders, meaning incentives for drug development, including market exclusivity, exemption from specific application fees, and an expedited pathway to approval.
There are currently no approved treatments for PANDAS/PANS with an addressable market of US$1.4 billion annually in the US.
NTI says NTIPANS1 was the first-ever clinical trial to show highly significant clinical improvements in PANDAS/PANS patients with a broad-spectrum cannabinoid drug therapy (NTI164) with excellent safety at 12 weeks.
Delving deeper into safety and clinical outcomes
Russell Dale – Professor of Paediatric Neurology, University of Sydney and The Children’s Hospital at Westmead and co-principal investigator of the NTIPANS1 trial – is pleased with the extension trial results so far.
“Following a minimum of 12 weeks of daily oral administration of NTI164 patients diagnosed with PANDAS/PANS experienced notable enhancements in their clinical functionality, coupled with an absence of safety concerns,” he says.
“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.”
Dale says noteworthy improvements have also been observed in measures of tic severity and obsessive-compulsive behaviours, all of which exhibit statistical significance when juxtaposed with 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,” he says.
“We eagerly anticipate disseminating these pivotal scientific insights through publication in a leading medical journal.”
NTI executive director Dr Thomas Duthy says the company is very pleased with the progress of its PANDAS/PANS patients under the extension phase of the NTIPANS1 clinical trial.
“The original 12-week trial demonstrated a statistically significant and clinically meaningful beneficial impact on these children’s symptoms,” he says.
“The results of today’s data release at 24 weeks highlights the durability of treatment with NTI164 with patients recording further clinical improvements and importantly, such benefits did not come at the cost of safety.
“NTI164 continues to demonstrate an attractive safety profile, devoid of any serious
events, which has traditionally hampered the acceptability of paediatric neurological drugs by the medical community.”
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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