Neurotech International Limited announced the initiation of a new Phase I/II Clinical trial of oral NTI164 in children diagnosed with Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections (PANDAS) and Paediatric Acute-Onset Neuropsychiatric Syndrome (PANS). PANDAS/PANS is a clinical diagnosis given to children who have a dramatic (typically within one day) onset of neuropsychiatric symptoms including intense anxiety, Obsessive-Compulsive Disorder (OCD) and/or severely restrictive eating. Children may exhibit repetitive tic movements, become moody, irritable/aggressive, and anxious.

The cause of PANS is unknown in most cases; however, the disorder is hypothesised to be triggered by infections, metabolic disturbances, and other inflammatory reactions. The PANDAS subgroup is defined by an association with Group A Streptococcus infection, and specific neuropsychiatric symptoms similar to PANS. Both PANDAS and PANS are considered rare paediatric (orphan) neurological disorders.

There are currently no US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved therapies for PANDAS/PANS and very limited clinical trials of new therapeutic interventions. With the development of PANDAS/PANS Diagnostic Criteria and Treatment Guidelines in 2015 and 2017, respectively,2 there is a unique opportunity for Neurotech's proprietary full spectrum cannabinoid drug formulation, NTI164. NTI164 has proven potent anti-inflammatory and neuro-protective effects in vitro, along with an excellent safety and tolerability profile in paediatric patients with Autism Spectrum Disorder.

NTI164 has the potential to reduce the neuroinflammation associated with PANDAS/PANS, which is postulated to play a role in the pathogenesis in the majority of cases. Neuroinflammation is one of the main triggers for neurodegeneration. NTIPAN1 is proposed to be a single-arm, open-label, Phase I/II clinical trial that will recruit 10 paediatric patients with a clinical diagnosis of moderate to severe PANDAS/PANS to determine the efficacy and safety of orally administered NTI164 in these patients.

The primary endpoint of the trial is the change from baseline for Clinical Global Impression Scales (CGI: severity, global improvement and therapeutic response) and the Children's Yale-Brown Obsessive Compulsive Scale (CY-BOCS)(OCD severity) - both of which are considered gold-standard, validated clinical assessment tools that are part of standard care in neurology. Key secondary endpoints will examine the Safety and Tolerability of orally administered NTI164 (at 5,10,15 and 20 mg/kg/day). The trial intends to enrol children at two centres within Australia; the Children's Hospital at Westmead and the Paediatric Neurology Unit at Monash Medical Centre.

Neurotech anticipates Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) to commence the Phase I/II prior to the end of CY2022, with patient recruitment to commence in 1H CY2023. The results of the trial are anticipated in 2H CY2023. The Company has filed additional patent applications covering this novel use of NTI164 in PANDAS/PANS and additional rare paediatric neurological disorders where neuro-inflammatory processes are well established in the scientific and medical literature.