Neurotech International extends NTI164 Rett Syndrome trial to 52 weeks

Biopharmaceutical development company Neurotech International (ASX: NTI) has extended a Phase I/II clinical trial investigating the use of NTI164 in female Rett Syndrome patients to 52 weeks.

The extension replaces the previous 16-week duration and was approved by the human research ethics committee at Sydney’s Children’s Hospital at Westmead under clinical trial protocol.

A total of 14 patients and their families have elected to continue daily oral treatment with NTI164 for a period of one year.

The open-label trial will seek to provide initial evidence on the safety and efficacy of NTI164 in light of a market need for safer and more effective therapies that target persistent neuro-inflammations associated with the rare disorder.

Unique cannabis strain

NTI164 is a proprietary drug formulation derived from a unique cannabis strain with low tetrahydrocannabinol and a novel combination of cannabinoids.

The drug has been exclusively licenced for neurological applications worldwide.

Pre-clinical studies have demonstrated a potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effect in human neuronal and microglial cells.

Neurotech is developing NTI164 as a therapeutic drug product for a range of neurological disorders in children where neuroinflammation is involved.

Rare disorder

Rett Syndrome is a rare genetic neurological and developmental disorder caused by mutations in the methyl CpG binding protein 2 gene on the X chromosome, which is required for normal brain development and function.

It 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 disorder affects approximately 15,000 girls and women in the US and 350,000 globally.

It is typically characterised by normal development up to 18 months of age, followed by a slowing of development, loss of functional use of the hands and distinctive hand movements along with difficulty walking, communicating, irritability and seizures.

There is currently no cure for Rett Syndrome and no approved therapies.

Current treatments only address symptoms and provide support which may improve movement, communication and social participation into adulthood.