Neurotech granted dosage extension of NTI164 for patients in autism spectrum disorder trial

The Human Research Ethics Committee (HREC) has granted Neurotech International (ASX: NTI) a two-year dosage extension for the use of lead drug NTI164 on autism spectrum disorder (ASD) patients who previously took part in the company’s Phase I/II clinical trial.

The extension will allow 11 patients who have continued daily oral treatment of the cannabinoid-based drug beyond the initial 18 month trial period to remain on treatment for an additional two years.

They will be transitioned to a compassionate use program through Monash Medical Centre in Melbourne later this year, with Neurotech continuing to collect periodic safety information as per HREC requirements.

The transition will be conducted under the Special Access Scheme Category B pathway which allows registered health practitioners to prescribe unapproved drugs to their patients.

It means Neurotech will receive reductions on certain charges associated with pharmacy, clinician and pathology costs from Monash, which will result in a considerable reduction to the overall cost of monthly drug supply.

Dosage extension

Neurotech executive director Dr Thomas Duthy said the dosage extension was initiated by clinical trial patients.

“Our ASD study showed outstanding clinical efficacy and safety for our oral formulation of NTI164,” he said.

“All 11 patients requested to continue treatment for the foreseeable future given the significant improvements in their clinical symptoms and the excellent safety profile of our treatment over time.”

Positive results

In March, Neurotech reported positive results from the first-in-human clinical trial of NTI164 on children with ASD.

The results revealed a “significant benefit” across a large number of clinically validated assessments at the 52-week mark.

Lead investigator Professor Michael Fahey said patients showed a “global improvement” in the severity of illness, socialisation and adaptive behaviour.

The benefits were coupled with a lack of serious adverse events and “clean” pathology results.

Earlier extension

Today’s news follows an earlier HREC extension of NTI164 for the treatment of paediatric autoimmune neuropsychiatric disorders associated with streptococcal infections (PANDAS) and paediatric acute-onset neuropsychiatric syndrome (PANS).

That approval will allow participants in a current Phase I/II clinical trial to continue receiving treatment up to 20 years of age.

Neurotech completed the recruitment of 15 paediatric patients earlier this month, with clinical results expected later this year.