Neurotech gets green light to start phase I/II cerebral palsy trial

Neurotech receives key approvals and clearances to start Phase I/II trial of NTI164 into spastic diplegia cerebral palsy
Trial aims to determine efficacy and safety of NTI164 from baseline to 12 weeks of treatment
Neurotech sees NTI164 as a potential breakthrough treatment for children with spastic CP

Special Report: Neurotech International has been given the green light to kick off its Phase I/II clinical trial to explore the use of its lead compound NTI164 in paediatric patients with spastic diplegia cerebral palsy (spastic CP).

Neurotech International (ASX:NTI) has announced its received approval from the Human Research Ethics Committee (HREC) and clearance from the Therapeutic Goods Administration (TGA) under the Clinical Trial Notification (CTN) scheme for the Phase I/II clinical trial.

NTI says the single-arm, open-label clinical trial will recruit up to 14 paediatric patients with a clinical diagnosis of gross motor function classification system (GMFCS) severity of 2-3, non-ambulant spastic CP patients.

The trial aims to determine the efficacy and safety of NTI164 in these patients from baseline to 12 weeks of treatment with NTI164.

The primary endpoint of the trial is the Caregiver Priorities and Child Health Index of Life with Disabilities (CPCHILD) Questionnaire, evaluating caregivers’ perceptions of health-related quality of life (HRQOL) and the impact on caregivers of children with CP.

Secondary endpoints include safety, the impact of NTI164 on pain, sleep, seizure frequency, dystonia (involuntary muscle contraction) and spasticity.

The trial will be led by Professor Michael Fahey, Head of the Paediatric Neurology Unit and Director of Neurogenetics at Monash Medical Centre in Victoria.

Professor Fahey brings significant experience with NTI164, having led both NTI’s Phase I/II and Phase II/III clinical trials in autism.

Focus on paediatric neurological disorders

NTI is focused predominately on paediatric neurological disorders, where there is persistent neuroinflammation.

NTI’s proprietary lead drug formulation, NTI164, is derived from a unique cannabis strain with low THC and a novel combination of cannabinoids, including CBDA, CBC, CBDP, CBDB and CBN.

The drug is exclusively licenced for neurological applications globally with pre-clinical studies demonstrating its potent anti-proliferative, anti-oxidative, anti-inflammatory and neuro-protective effects in human neuronal and microglial cells.

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

The company announced in December all patients had been recruited for its Phase II/III NTIASD2 clinical trial for children with autism spectrum disorder (ASD).

A total of 56 patients have been enrolled in the trial, all with level 2 (requiring substantial support) or level 3 (requiring very substantial support) autism. Results of the trial are expected during the current quarter.

Breakthrough treatment for spastic CP

Spastic CP is the most prevalent form of CP, accounting for up to 80% of cases and representing a leading cause of childhood disability.

NTI says with ~750,000 children and adults affected in the US and around 34,000 individuals living with CP in Australia, the market is projected to reach an annual value of US$4.3 billion by 2030.

NTI executive director Dr Thomas Duthy says the company sees NTI164 as a potential breakthrough treatment for children with spastic CP.

“Although there are a variety of drug therapies used in the treatment of spastic CP, they are often associated with sedation, confusion, memory loss and attention deficits,” he says.

“For first-line treatment with oral Baclofen the actual evidence of efficacy remains somewhat subjective and not necessarily supportive of widespread use in spastic CP.

“Accordingly, we see NTI164 as potentially a new breakthrough treatment as part of the clinical armamentarium to treat CP spasticity more effectively noting improvements in gross motor function, increased participation at a social level and comfort are considered important treatment goals to improve the overall quality of life of the individual.”

Duthy says the Phase I/II clinical trial will be registered on the Australian New Zealand Clinical Trials Registry (ANZCTR) and is forecast to kick off in H1 CY24.

This article was developed in collaboration with Neurotech International, a Stockhead advertiser at the time of publishing.

This article does not constitute financial product advice. You should consider obtaining independent advice before making any financial decisions.