Neurotech International Limited announced the launch of a new clinical program in Rett Syndrome, comprising an upcoming filing for Human Research Ethics Committee (HREC) approval and thereafter, the commencement of a Phase II clinical trial investigating the use of NTI164 in female patients. The trial will be conducted across two centres in Australia with Co-Principal Investigators Dr Giuliana Antolovich, Department of Neurodevelopment & Disability, Royal Children's Hospital and Professor Michael Fahey, Head of the Paediatric Neurology Unit at Monash Medical Centre, Director of Neurogenetics. Rett Syndrome is a rare genetic neurological and developmental disorder and is almost exclusively the result of a mutation(s) in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.

Rett Syndrome occurs almost exclusively in girls, with incidence of one in 10,000 female live births. The prevalence is approximately 15,000 girls and women in the US and 350,000 globally. The proposed Phase II clinical trial (which may be subject to change by HREC) will examine the effects of daily oral treatment of NTI164 and is targeting the recruitment of approximately 15 Rett Syndrome patients initially.

The proposed primary endpoints at 12 weeks of treatment are the Rett Syndrome Behaviour Questionnaire (RSBQ), Clinical Global Impression Scale-Improvement (CGI-I) and CGI-severity of illness (CGI-S). Key secondary endpoints include safety, adverse events and measures associated with hand function, motor skills, communication and quality of life. In addition, the trial will analyse the effects of NTI164 on a range of biomarkers associated with neuronal function and neuroinflammation in Rett Syndrome.

If successful, the Company will follow (under the same HREC approval), with a 14-week double-blind, randomised, placebo-controlled Phase II in 34 participants to determine further efficacy and safety. Neurotech anticipates HREC approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) to commence the Phase II trial during second quarter current year 2023, with patient recruitment to commence in second half current year 2023. The preliminary results of the trial (n=15) are anticipated in first half current year 2024.