Neurotech to extend Phase 2/3 autism spectrum disorder trial to adults

Neurotech receives HREC approval to extend Phase II/III ASD trial to adults
Company committed to developing NTI164 as a first-line treatment for patients with autism
Trial on track to report results during the first quarter of calendar year 2024

Special Report: Neurotech’s Phase 2/3 clinical trial in autism spectrum disorder (ASD) has hit another milestone after receiving approval to allow patients who turn 18 to remain on treatment during the extension trial.

Neurotech International (ASX:NTI) has announced Human Research Ethics Committee (HREC) approval to extend its Phase 2/3 clinical trial in ASD to allow patients who turn 18 to remain on treatment with its proprietary NTI164 during the extension phase for up to 54 weeks of total treatment.

The clinical-stage biopharmaceutical development company is focused predominately on paediatric neurological disorders.

The current HREC approval for NTIASD2 – a Phase 2/3 trial – covers treatment with NTI164 in a paediatric population of ASD.

NTI164 drug formulation derived from a unique cannabis strain with low THC and a novel combination of cannabinoids including CBDA, CBC, CBDP, CBDB and CBN and is exclusively licenced for neurological applications globally.

Pre-clinical studies have demonstrated the drug’s 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.

Earlier study in ASD shows success

The double-blind, placebo-controlled Phase 2/3 clinical trial in Australia has been assessing the efficacy of NTI164 up to 20mg/kg/day on the severity of ASD in up to 54 patients aged 2-17 years, now extended to beyond 18.

The primary endpoint of the trial is Clinical Global Impression-Severity (CGI-S), which reflects a clinician’s impression of severity of illness on a 7-point scale ranging from 1=not at all, to 7=among the most extremely ill.

Results of its earlier trial show strong safety and efficacy, with significant improvement across many assessments versus baseline (Day 0).

The side effects reported were not serious or severe and did not significantly interfere with patients’ functioning.

No clinically significant abnormal laboratory values were reported. At 52 weeks of treatment, the mean severity of illness rating of the CGI-S was 3.0, representing an improvement of 30% from baseline.

Endpoints reached in PANDAS/PANS trial

In October NTI reported paediatric patients with orphan disorder PANDAS/PANS could have a new treatment available to them after NTI ground-breaking phase I/II trial of NTI164 reached its primary endpoints with significant and clinically meaningful improvements.

PANDA (Paediatric Autoimmune Neuropsychiatric Disorders Associated with Streptococcal Infections) and PANS (Paediatric Acute-Onset Neuropsychiatric Syndrome) are a collective disorder.

Children go to bed perfectly normal but wake up with uncontrollable tic movements in their hands and legs and become severely obsessive compulsive with heightened anxiety and depression.

This disorder is believed to manifest due to some dysregulation from streptococcus or some other unknown pathogen that initiates an autoimmune response which sees inflammation in their brain.

Need for ASD therapies remain significant

Executive director Dr Thomas Duthy says NTI is pleased to secure the additional HREC approval for NTI164 in the patient population for the Phase 2/3 trial into ASD.

“I am delighted with the progress of this large double-blind, placebo controlled clinical trial,” he says.

“We remain steadfastly committed to developing NTI164 as a first-line treatment for Level 2-3 patients with autism where the market need for new therapies remains significant.

“Importantly, with recruitment into the trial to complete during the current quarter, we are on track to report results during the first quarter of calendar year 2024 (Q3 FY24).”

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.