PANDAS and PANS are debilitating conditions characterised by distressing symptoms, ranging from rage to signs of obsessive compulsive disorder. It upends the lives of both kids and families, owing to significant caregiver burden and physiological stress. Until now, safe and effective treatment options for these disorders have been limited, leaving many families searching for hope and relief.
One company working on a solution is clinical-stage biopharma company Neurotech International (ASX: NTI). It has been studying the potential of cannabis-based drugs on treating patients with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS).
The Company has announced compelling clinical results from its Phase I/II clinical trial of NTI164—its proprietary drug formulation, derived from a unique low-THC cannabis strain. The study found that the drug has potential in alleviating anxiety and depression symptoms in children diagnosed with PANDAS/PANS.
Professor of Paediatric Neurology at the University of Sydney and Children’s Hospital at Westmead and Co-Principal investigator of the NTIPANS1 trial, Professor Russell Dale, said, “I am very pleased with the clinical results reported to date and wish to thank all patients and their families for participating in this novel clinical trial. I have observed quite profound improvements in a number of my patients with NTI164, making it the first trial of its kind with a broad-spectrum cannabinoid therapy showing initial clinical utility like this with excellent safety.”
Pointing to another challenge associated with PANDAS/PANS, i.e. catching it early, he added, “We await further evidence of genomic molecular changes from baseline measures and after 12 weeks of treatment to correlate this meaningful clinical response we have seen with biological evidence of effect. This would be a major step-forward for PANDAS/PANS patients and assist in identifying relevant biomarkers of the disease.”
The open-label Phase I/II clinical trial involved 15 pediatric patients who received daily treatment with NTI164 over a 12-week period. The trial’s primary endpoint was the change in the Revised Child Anxiety and Depression Scale – Parent Version (RCADS-P), a questionnaire assessing youth anxiety and depression. The results were remarkable, with the mean total T-score for the 15 patients improving significantly by 30%, from a baseline score of 83.7 to 58.2 after 12 weeks of NTI164 treatment.
Another efficacy endpoint, the Clinical Global Impression-Severity of Illness (CGI-S) scale, administered by clinicians, showed an 18% improvement in the patients’ mean severity of illness rating at the 12-week mark. Finally, the patients’ quality of life exhibited considerable improvement.
The trial reported a total of nine adverse events, with six possibly related to the study medication, including vomiting and nausea. But none of these events were considered serious or interfered with patient functioning.
All patients will receive treatment under the extended phase of the trial protocol, which has been granted an HREC extension to allow treatment until age 20.
President of the non-profit organisation PANS Australia and New Zealand Advocacy and Support Inc., James Fletcher, added, “As our researchers continue to expand their understanding of PANS, they also help us to raise awareness of PANS within the general medical community. This in turn leads to earlier intervention and better long-term outcomes for children and adults with PANS. A targeted treatment for PANS in the form of NTI164 is a very exciting development indeed. Many thanks to the researchers for their work on this therapy and to the children and families who participated in the study.”
In FY23, Neurotech’s revenue stood at $1.2 million against a loss of $7.7 million. Still, the Company ended the year with over $5 million in cash, up from FY22’s $1.8 million. Neurotech’s hope is that NTI 164 will eventually become to go-to treatment for PANDAS/PANS, but for now, its focus is on seeing through the trials effectively.
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