Neurotech International’s NTI164 continues to impress with safe long-term efficacy in paediatric ASD patients

Clinical-stage biopharmaceutical development company Neurotech International (ASX: NTI) has updated the market on the progress of its world-first Phase I/II clinical trial examining the daily use of proprietary broad-spectrum cannabinoid drug therapy NTI164.

11 paediatric patients with autism spectrum disorder (ASD) have now crossed 90 weeks of daily oral therapy with NTI164, which is being administered at the Paediatric Neurology Unit (PNU) within Melbourne’s Monash Medical Centre.

The drug has been reported to exhibit an “exceptional” safety and tolerability profile, with all patients showing stable blood chemistries and normal liver and kidney function.

Neurotech said although no quantitative efficacy analysis had been collected since week 52, caregiver and clinician reports remained positive with symptomatic improvements maintained at 90 weeks.

Longest-ever study

Neurotech executive director Dr Thomas Duthy said the Phase I/II trial was the longest study to date examining the safety of a broad-spectrum cannabinoid drug treatment on ASD.

“This landmark clinical trial continues to demonstrate the excellent durability of NTI164 and importantly, no significant safety concerns have arisen beyond the original 52-week analysis undertaken and reported by Neurotech,” he said.

“In our view, this updated safety analysis underscores the potential for NTI164 as a long-term, chronically-administered therapeutic agent in the treatment of ASD without the safety- and side-effects observed in approved therapies that restrict certain behaviours, particularly aggression and irritability in paediatric patients.”

He added the drug had demonstrated clinically-significant improvements in standardised ASD scales relating to global improvement, severity of illness, socialisation and adaptive behaviour during the 52-week period.

Trial progress

PNU head Professor Michael Fahey said he was pleased with how the trial was progressing.

“I am delighted with the progress of my patients under this long-term extension to our original Phase I/II clinical trial,” he said.

“To have 11 patients still on treatment past 90 weeks is testament to the durable responses we have seen in our patients, coupled with a remarkable safety profile of this intervention.”

“We eagerly await the results of the double-blind, placebo-controlled Phase II/III trial [which commenced late last year] to confirm these earlier clinical findings.”