Neurotech ready to test cannabinoid drug treatment for rare Rett Syndrome
Specialist biopharmaceutical development company Neurotech International (ASX: NTI) has obtained critical approval to undergo testing of a potential treatment for Rett Syndrome, a disease that mainly affects female sufferers.
Rett Syndrome is a rare genetic neurological and developmental disorder that affects the way the brain develops. The disorder can lead to a progressive loss of motor skills and language.
The 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.
Official go-ahead for further trials
The company has now received approvals to test its proprietary drug formulation NTI164 on selected patients.
Derived from a unique cannabis strain, NTI164 has been exclusively licenced for neurological applications globally. Pre-clinical studies have demonstrated a potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.
The written notice of Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) scheme clearance by the Therapeutic Goods Administration (TGA) now allows the company to begin Phase I/II clinical trial investigations into the use of NTI164 in female Rett Syndrome patients.
Australian sites chosen
Multiple Australian centres have been selected to conduct the trials under the guidance of principal investigator associate professor Carolyn Ellaway, a senior staff specialist with the NSW Genetic Metabolic Disorders Service, the Sydney Children’s Hospital Network and Metabolic Genetics at The Children’s Hospital at Westmead and associate principal investigators professor Michael Fahey, head of the Paediatric Neurology Unit at Monash Medical Centre, director of Neurogenetics and Dr Giuliana Antolovich, from the department of Neurodevelopment & Disability, Royal Children’s Hospital Melbourne.
Neurotech International executive director, Dr Thomas Duthy, said the company was pleased to have received a go-ahead after making its submission for HREC approval in early April.
“Rett Syndrome is the second leading cause of intellectual disability in girls, with an urgent medical need to develop safe and effective therapies to treat this progressive neurological disease. With its positive effects on neuroinflammation, we believe NTI164 could represent an effective intervention in Rett Syndrome, where mutations in the MECP2 gene are known to exacerbate neuroinflammation.”
According to associate professor Ellaway, girls who suffer from Rett Syndrome require continued supportive care, with the disease negatively impacting their quality of life and activities of daily living.
“Despite recent advances in the field, as a clinician who has witnessed first hand the impact of this progressive disorder on my patients, I am very excited to be involved with this clinical trial which will assess safety, tolerability and effectiveness of daily oral NTI164 in these girls. We certainly look forward to the results of the study following 12 weeks of initial treatment.”
Phase II trial
The Phase II clinical trial will study the effects of a daily oral treatment of NTI164 and is targeting the recruitment of 14 Rett Syndrome patients initially.
The company is looking to commence patient recruitment early in the third quarter of CY2023, and aiming to receive preliminary results in the first quarter of 2024.
Trial planning has identified a number of key secondary endpoints including safety, observance of any adverse events and measures associated with hand function, motor skills, communication, and quality of life.
The company’s plan is to follow-up a successful first stage with a 14-week double-blind, randomised, placebo-controlled Phase II in 34 participants to determine further efficacy and safety, which will require a separate HREC submission and approval.