Neurotech International battles PANDAS with cannabinoid-based lead drug
PANDAS refers to difficult-to-treat paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections.
Emerging drug developer Neurotech International (ASX: NTI) is on a war against PANDAS – nothing to do with attacking China’s loveable piebald national symbol, but difficult-to-treat paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS).
PANDAS are a subset of the paediatric acute-onset neuropsychiatric syndrome (PANS).
As a clinical stage drug developer focused on paediatric neurological disorders, Neurotech is furthering clinical trials of its cannabinoid-based lead formulation NTI-164.
In February, Neurotech said the first of 15 planned patients had been treated in its phase I/II PANDAS/PANS trial, undertaken at the Children’s Hospital at Westmead in Sydney and the Monash Medical Centre’s paediatric neurology unit in Melbourne.
In the meantime, the company is expanding a more advanced phase II/III autism spectrum disorder (ASD) trial.
What is PANDAS?
PANDAS/PANS is marked by a dramatic onset of symptoms, including obsessive compulsive disorder and/or restrictive eating.
Sufferers may also have repetitive tic movements and become moody, aggressive or anxious and have difficulty with schoolwork.
While the cause of the disorders is unknown, suspected triggers are infections, metabolic disturbances and other inflammatory reactions.
PANDAS/PANS is classified as a rare (orphan) disease and there are currently no approved therapies to treat this debilitating neurological disorder.
Lead drug NTI-164
Exclusively licenced by the company globally for neurological applications, NTI-164 is a proprietary cannabinoid drug formulation, with very low levels of the psychoactive ingredient THC, making it suitable for paediatric use.
Neurotech executive director, Dr Thomas Duthy says NTI-164 has the potential to reduce neuroinflammation associated with neurodegeneration.
Preclinical studies have already showed NTI-164 has a “potent antiproliferative, anti-oxidative, anti-inflammatory and neuroprotective effect”.
The primary endpoint of the single-arm, open-label PANDAS/PANS trial is the change in baseline after 12 weeks. This is measured by a globally-accepted parent-rated score, as well as a clinician’s impression of the level of patient improvement.
Secondary endpoints include measures such as tic severity and obsessive-compulsive activity.
The company expects to complete trial recruitment in the current (March) quarter, with results expected in the second half of 2023.
Advanced autism spectrum disorder trial
Neurotech is also investigating NTI-164 in ASD.
With regards to ASD, Neurotech has completed a phase I/II clinical trial, based at the Monash Medical Centre.
The initial results showed “excellent” safety and efficacy, after four and 20 weeks of daily oral treatment under the 54-week treatment regimen.
Neurotech has now received ethics committee approval to expand the treatment by six months to 80 weeks, on a patient-specific basis.
Dr Duthy says patients had shown an “unwavering level of support” well beyond the original 28-day trial period, “due to the exceptional clinical and safety results observed to date.”
The 54-week results are expected by the end of March.
Representing 1.5 years of daily treatment data, the extended trial will support planned future regulatory submissions, for either PANDAS/PANS, ASD or cerebral palsy (another indication of interest).
Meanwhile, in the December quarter the company began recruiting the first of up to 54 patients for a separate, phase II/III ASD trial. Carried out at the Monash Medical Centre, the double-blinded study will compares the efficacy of NTI-164 relative to sham (placebo) treatment over 16 weeks.
The primary endpoint is improvement as measured by the Clinical Global Impression Severity scale, which measure illness on a seven-point scale.
Neurotech’s progress and development
Neurotech listed on the ASX in November 2016, having raised $7 million in an IPO. Initially the company focused on its flagship Mente Autism device, an at-home management tool for kids on the autism spectrum.
The company continues to commercialise Mente, which aims to increase engagement levels of children with Autism and improve their relaxation.
On a financial note, Neurotech is fully funded for the clinical programs, having reported a cash balance of just over $8.094 million as of 31 December 2022.
The December quarter statement shows expenditure of $2.67 million and net cash outflows of $1.35 million.
In late October 2022, the company completed a $9 million share placement, to institutional and sophisticated investors.
Neurotech also raised $1.1 million from the exercise of share options and received a $1.2 million federal government research and development tax incentive rebate.