Neurotech updates market on NTI164 progress for paediatric disorders
Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has updated the market on progress of its lead candidate cannabinoid-based drug NTI164 to treat several paediatric neurological disorders where there is an unmet medical need for safe and effective therapies.
In May, the company announced it had gained clearance from the Human Research Ethics Committee (HREC) to extend a Phase I/II trial of NTI164 in children with autism spectrum disorder.
The extension allows for an additional two years of daily oral treatment with NTI164 for 11 patients who have already participated in the trial for a total of 18 months.
These patients will transition to a compassionate use program (Special Access Scheme Category B) through Melbourne’s Monash Medical Centre under the care of treating clinician Professor Michael Fahey.
Neurotech confirmed supply of the drug would entitle it to reduced charges associated with pharmacy, clinician and pathology costs.
Patient recruitment
Also in May, Neurotech announced it had completed patient recruitment for a Phase I/II clinical trial of NTI164 in children diagnosed with paediatric auto-immune neuropsychiatric disorders associated with streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).
A total of 15 patients have started treatment with NTI164 with the aim of gathering insights into the clinical efficacy and safety of the drug over a 12-week period.
Neurotech has been granted an HREC extension to allow children to continue to receive treatment after they turn 18 years of age, at which point they may elect to continue daily oral dosing beyond the study, into a 54-week period.
Rett Syndrome paper
In June, associate professor Carolyn Ellaway from Sydney Children’s Hospitals Network presented a paper titled NTI164: A Novel, Full-Spectrum Medicinal Cannabis-Derived Treatment for Rett Syndrome at a US meeting of the International Rett Syndrome Foundation (IRSF).
Ms Ellaway is a clinical geneticist and a senior staff specialist at the NSW Genetic Metabolic Disorders Service, and also works in Metabolic Genetics at The Children’s Hospital of Westmead.
The meeting served as a platform to share knowledge and engage with the Rett Syndrome community regarding the progress of NTI164.
Delegates included clinicians and researchers representing academia, industry and government from around the world.
Trial approval
Earlier this month, Neurotech announced it had received HREC approval and Therapeutic Goods Administration clearance to initiate a Phase I/II clinical trial of NTI164 in female patients with Rett Syndrome.
The study will be conducted across three centres in Australia, led by Ms Ellaway and associate principal investigators Professor Michael Fahey from the Paediatric Neurology Unit at Monash Medical Centre and Dr Giuliana Antolovich from the Department of Neurodevelopment & Disability at Melbourne’s Royal Children’s Hospital.
Rett Syndrome is a rare genetic neurological and developmental disorder which affects brain development and can lead to a progressive loss of motor skills and language.
It occurs almost exclusively in girls, with an incidence of one in 10,000 female live births.
The syndrome affects approximately 15,000 girls and women in the US and 350,000 females globally.