Neurotech International completes recruitment for NTI164 trial in Rett Syndrome
Clinical-stage biopharmaceutical company Neurotech International (ASX: NTI) has completed the recruitment of 14 patients into a Phase I/II clinical trial investigating the use of lead candidate NTI164 in females with Rett Syndrome.
The 16-week 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 which target persistent neuro-inflammations associated with the rare disorder.
Rett Syndrome is the result of a mutation in the methyl CpG binding protein 2 (MECP2) gene located on the X chromosome, which is required for normal brain development and function.
It occurs almost exclusively in girls and has an incidence of one in 10,000 female live births.
In the US, the disorder affects approximately 15,000 girls and women while global numbers sit around 350,000.
Rett Syndrome is characterised by a slowing down of normal development from around 18 months of age and includes loss of functional hand use and distinctive hand movements along with difficulty walking, communicating, irritability and seizures.
There is currently no cure and no approved therapies.
Standard of care treatments address symptoms and provide support which may improve movement, communication and social participation into adulthood.
Neurotech executive director Dr Thomas Duthy said the company expected early results from the trial.
“As a result of the rapid recruitment into this study, we anticipate results to be available in the new year, providing us with an accelerated clinical insight into this rare neurological disorder,” he said.
“Following on from our successful presentation at the 2023 International Rett Syndrome Scientific Meeting in Tennessee, the need for safer and more effective therapies is of paramount importance.”
Neurotech plans to follow the Phase I/II trial with a 14-week double-blind, randomised, placebo-controlled Phase II trial of NTI164 in 34 participants to determine further efficacy and safety.
It will be subject to a second approval from the Human Research Ethics Committee.