Neurotech International (ASX: NTI), a clinical-stage biopharmaceutical development company, has achieved a significant milestone by initiating the dosing of the first patient in its ground breaking clinical trial for the treatment of Rett Syndrome. This marks a crucial step forward in the company’s mission to provide an innovative therapy for individuals affected by this rare and complex genetic neurological disorder.
Addressing Rett Syndrome: A Rare Neurological Disorder
Rett Syndrome is a rare neurological disorder primarily affecting females, characterized by its impact on brain development and function. The disorder’s prevalence is estimated at approximately one in 10,000 female live births across different racial and ethnic groups worldwide. Rett Syndrome Research Trust data suggests that around 15,000 girls and women in the United States and 350,000 globally are affected each year by this condition, emphasizing the need for effective therapeutic interventions.
Enrolment and Treatment Initiation
Neurotech International has successfully enrolled and treated the first patient in its Phase I/II clinical trial of the NTI164 treatment for female Rett Syndrome patients. This milestone underscores the company’s commitment to advancing research and innovation in the field of neurology.
Harnessing Unique Cannabinoid Treatment
At the heart of this ground breaking initiative is NTI164, a proprietary drug formulation derived from a distinctive cannabis strain. This formulation boasts low levels of THC and has been exclusively licensed for neurological applications on a global scale. Pioneering pre-clinical studies have demonstrated NTI164’s potent anti-proliferative, anti-oxidative, anti-inflammatory, and neuro-protective effects on human neuronal and microglial cells, suggesting its potential as a transformative treatment option.
Pathways to Therapeutic Development
The significance of this clinical trial extends beyond Rett Syndrome, as NTI164 holds promise for a range of neurological disorders in children. The Phase II of the NTIRTT1 clinical trial focuses on assessing the effects of daily oral treatment with NTI164. With plans to recruit 14 Rett Syndrome patients initially, the trial aims to provide comprehensive insights into the drug’s safety and efficacy. A pivotal aspect of this phase involves administering NTI164 at the maximum tolerated dose or 20mg/kg/day over a span of more than 16 weeks.
Addressing Neuroinflammation: An Urgent Need
Dr. Thomas Duthy, Executive Director of Neurotech International, emphasises the critical timing of this trial. The persistent neuroinflammation associated with Rett Syndrome necessitates the development of safer and more effective therapies. Dr. Duthy highlights the emerging opportunities in the Rett Syndrome therapeutic landscape, underscoring the company’s dedication to addressing the unmet needs of this patient population.
Future Prospects and Regulatory Approvals
Neurotech International’s forward momentum received a boost with the receipt of Human Research Ethics Committee (HREC) approval and Clinical Trial Notification (CTN) scheme clearance from the US Therapeutic Goods Administration (TGA) in early July. This regulatory clearance paves the way for the Phase I/II clinical trial to be conducted across three esteemed centres in Australia, guided by a team of accomplished principal and associate investigators.
Collaborative Expertise for a Transformative Future
The trial’s principal investigator, Associate Professor Carolyn Ellaway, leads a multidisciplinary team encompassing the NSW Genetic Metabolic Disorders Service at the Sydney Children’s Hospital Network, the Metabolic Genetics unit at The Children’s Hospital at Westmead, and other esteemed experts in the field. This collaborative effort signifies a collective commitment to advancing medical knowledge and providing hope for individuals and families affected by Rett Syndrome.
