Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has launched a new clinical program into the use of daily oral cannabinoid-based drug to potentially treat Rett Syndrome in female patients.
The program will comprise a filing for human research ethics committee (HREC) approval and the commencement of a Phase II clinical trial investigating the use of lead candidate NTI164 in up to 15 female patients aged 5 to 20 years.
It will be conducted across two centres in Australia with co-principal investigators from the Department of Neurodevelopment & Disability at the Royal Children’s Hospital and the Paediatric Neurology Unit at Monash Medical Centre.
The trial will examine the effects of daily oral treatment of NTI164 with proposed primary endpoints at 12 weeks of treatment being the Rett Syndrome behaviour questionnaire, clinical global impression scale improvement and CGI-severity of illness.
Key secondary endpoints include safety, adverse events and measures associated with hand function, motor skills, communication and quality of life.
The trial will also analyse the effects of NTI164 on a range of biomarkers associated with neuronal function and neuroinflammation in Rett Syndrome.
If successful, it will trigger the start of a 14-week double-blind, randomised, placebo-controlled Phase II trial in 34 participants to determine further efficacy and safety.
Neurotech anticipates HREC approval and Clinical Trial Notification (CTN) scheme clearance by Australia’s Therapeutic Goods Administration in the coming months, with patient recruitment to commence in the second half of this year.
Preliminary results of the trial are expected in early 2024.
Unique cannabis strain
NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low levels of tetrahydrocannabinol and a novel combination of other cannabinoids.
The drug has been exclusively licenced for neurological applications globally, with pre-clinical studies demonstrating potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.
NTI164 is being developed as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.
Nuerotech executive director Dr Thomas Duthy said the neuroprotection shown by NTI164 including strong anti-inflammatory effects in brain-derived neuronal and microglial cells could translate to improved clinical outcomes in Rett Syndrome patients.
Rett Syndrome is a rare genetic neurological and developmental disorder believed to result from a mutation in the human MECP2 gene, which is required for normal brain development and function.
The condition occurs almost exclusively in girls, with an incidence of approximately 1 in 10,000 female live births across all racial and ethnic groups worldwide.
According to the Rett Syndrome Research Trust, the prevalence is approximately 15,000 girls and women in the US and 350,000 globally.
Rett syndrome is typically characterised by normal development up to 18 months, followed by a slowing of development including loss of motor skills, along with difficulty walking, communicating, irritability and seizures.
There is currently no cure for Rett Syndrome and no approved therapies.
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