Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has reached a critical milestone with the dosing of the initial patient using its unique treatment for Rett Syndrome.
The first patient has now been enrolled and treated in the company’s Phase I/II clinical trial of the NTI164 treatment for female Rett Syndrome patients.
Rett Syndrome affects brain development
Occurring almost exclusively in girls compared to boys, Rett Syndrome is a rare genetic neurological and developmental disorder which can impact normal brain development and function.
Approximately one in 10,000 female live births across all racial and ethnic groups worldwide can be affected by the disease with data from the Rett Syndrome Research Trust estimating that approximately 15,000 girls and women in the US and 350,000 globally will be affected each year.
Dr Thomas Duthy, executive director of Neurotech International, said dosing of the first patient is an important step in NTI164’s path to development.
“We warmly congratulate our principal investigator associate professor Carolyn Ellaway, on commencing this important trial in Rett Syndrome which seeks to provide initial evidence on the safety and efficacy of NTI164 in this patient population,” Dr Duthy stated.
Unique cannabinoid treatment
Derived from a unique cannabis strain, NTI164 is a proprietary drug formulation with low THC levels which has been exclusively licenced for neurological applications globally.
Pre-clinical studies have confirmed a potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.
Apart from Rett Syndrome, the treatment is being developed as a therapeutic drug product for a range of neurological disorders in children.
Phase II of the NTIRTT1 clinical trial is studying the effects of daily oral treatment of NTI164 and is targeting the recruitment of 14 Rett Syndrome patients initially. The trial will include more than 16 weeks of treatment with NTI164 at the maximum tolerated dose or 20mg/kg/day.
Important time for treatment development
Dr Duthy said the latest stage of the NTI164 trial comes at a time when there is a need for safer and more effective therapies that target the persistent neuroinflammation associated with the rare neurological disorder.
“Recent corporate activity in the Rett Syndrome therapeutic space highlights to us the significant opportunity ahead for the company in developing NTI164 for this patient population.”
If successful, up to 34 patients will be enrolled in plans to follow-up the current trial with a 14-week double-blind, random and placebo-controlled Phase II.
Recent TGA approval
In early July Neurotech received written Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) scheme clearance by the US Therapeutic Goods Administration (TGA) to commence the Phase I/II clinical trial of NTI164 in female Rett Syndrome patients.
The aim is for the trial to be conducted across three centres in Australia with principal investigator associate professor Ellaway, senior staff specialist NSW Genetic Metabolic Disorders Service, the Sydney Children’s Hospital Network and Metabolic Genetics at The Children’s Hospital at Westmead and associate principal investigator, professor Michael Fahey, head of the Paediatric Neurology Unit at Monash Medical Centre.
