Neurotech’s NTI164 treatment in children with PANDAS/PANS has demonstrated success in two key areas. Pic: ulkas via Getty Images.
Health & Biotech
Paediatric patients with orphan disorder PANDAS/PANS could have a new treatment available to them after Neurotech’s groundbreaking phase I/II trial of NTI164 reached its primary endpoints with significant and clinically meaningful improvements.
PANDAS (Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections) and PANS (Paediatric Acute-Onset Neuropsychiatric Syndrome) are a collective disorder where children go to bed perfectly normal but wake up with uncontrollable tic movements in their hands and legs and become severely obsessive compulsive with heightened anxiety and depression.
This disorder manifests due to some dysregulation from streptococcus or some other unknown pathogen that initiate’s an autoimmune response which sees inflammation in their brain.
Due to its rarity – and accompanying lack of approved drug therapies, PANDAS/PANS are classified as rare orphan disorders, which could lead to approved drugs securing development incentives such as market exclusivity, exemption from certain application fees and a fast track to approval.
Enter clinical-stage biopharmaceutical development company Neurotech International (ASX:NTI) and its proprietary NTI164 drug formulation derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN.
NTI164 is exclusively licenced for neurological applications globally with pre-clinical studies demonstrating a potent anti-proliferative, anti-oxidative, anti-inflammatory, and neuro-protective effects in human neuronal and microglial cells.
It is being developed as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.
The drug is currently undergoing a randomised, double-blind, placebo-controlled Phase 2/3 clinical trial in Australia for autism spectrum disorder following earlier successes and has secured HERC approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) for investigations in treating female Rett Syndrome patients. The Company recently completed recruitment of 14 girls into this trial.
NTI164 has now proven itself to be a promising treatment for PANDAS/PANS after reporting statistically significant and clinically meaningful improvements shown across a range of gold standard, clinically validated assessments over 12 weeks of treatment for the 15 patient open-label Phase I/II clinical trial.
Primary endpoint of anxiety and depression met with a 30% improvement in overall symptoms from high severity at baseline to low severity from week 4 onwards.
Additionally, the primary endpoint of severity of illness saw children re-classified from markedly ill at baseline to moderately ill at 12 weeks – an 18% improvement.
No serious adverse events have been recorded to date.
“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,” professor Russell Dale.
Professor Dale – a professor of paediatric neurology at the University of Sydney and Children’s Hospital at Westmead as well as the co-principal investigator of the NTI1 NTIPANS1 trial – said he observed quite profound improvements in a number of patients.
This makes NTI164 the first trial of its kind with a broad-spectrum cannabinoid therapy showing initial clinical utility like this with excellent safety.
“In addition, 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,” he said.
“This would be a major step-forward for PANDAS/PANS patients and assist in identifying relevant biomarkers of the disease.”
Neurotech executive director Dr Thomas Duthy said the company had commenced this clinical trial based on a small number of scientific publications that highlighted recurring, neuroinflammatory processes in these difficult to treat patients.
“With our established evidence in autism and supportive pre-clinical data we took the decision to run this world-first trial of NTI164 with Professor Dale and Professor Fahey, which has shown very strong benefits for these children over 12 weeks of daily treatment,” he said.
“Given the lack of safe and effective treatments for PANDAS/PANS with associated distressing symptoms and significant caregiver burden we remain very hopeful of an accelerated development plan for NTI164 to bring this therapy to market.”
Neurotech is currently awaiting the multi-omic exploratory endpoint, which for the first time could elucidate potential biomarkers of disease.
Its hypothesis is that NTI 164 will be anti- inflammatory and affect gene regulation, thereby improving clinical symptoms.
The company also believes that the transcriptomic signature can be modified with NTI164 treatment, corresponding with clinical improvements in neuropsychiatric (emotional) symptoms associated with PANDAS/PANS.
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.
