Neurotech reports further positive Phase 2/3 autism trial results

Neurotech reports further benefits from Phase 2/3 trial of NTI164 in autism patients
Patients showed 56% improvement in CGI-S score from baseline at week 12
Patients unblinded at end of week eight showed 21% improvement in CGI-S score to week 12

Special Report: Neurotech has reported further benefits from its Phase 2/3 trial of autism patients participating in its world-first broad-spectrum cannabinoid drug therapy NTI164 with significant improvements in symptoms.

Neurotech (ASX:NTI) says it continues to see further significant improvements in ASD patients who received its lead cannabinoid drug NTI164 as measured by the clinician via the Clinical Global Impression – Severity of illness Scale (CGI-S).

A further analysis has been carried out on the effect of NTI164 in children who were receiving placebo for the first eight weeks of the Phase 2/3 NTIASD2 trial and then crossed-over to the drug from the start of week nine to week 12 per the trial protocol.

In addition, CGI-S information was analysed at week 12 for those patients who were initially enrolled in the NTI164 arm of the trial.

NTIASD2 is a randomised, double-blind, placebo-controlled, Phase 2/3 clinical trial that recruited 54 patients with ASD to determine the efficacy and safety of NTI164 versus placebo.

The study comprised an eight-week treatment period followed by an eight-week open-label maintenance period and then a two-week wash-out period.

NTI says participants who choose to continue receiving NTI164 beyond the duration of the study may do so for an additional 38 weeks.

56% improvement from baseline

NTI says at 12 weeks, patients who received NTI164\ following the primary endpoint analysis at eight weeks showed a mean CGI-S score of 2.42, representing a 56% improvement from baseline (CGI-S: 5.54) with children re-classified under this scale as borderline ill (CGI-S score of 2.42).

In general, for a CGI-S score of 2, ASD symptoms are present but only just noticeable and not significantly impairing for the child.

NTI says this is a very significant improvement at 12 weeks for those patients on NTI164 relative to their baseline score of 5.54 (markedly ill), and versus eight weeks (mildly ill, CGI-S 3.77).

NTI says markedly ill patients show significant impairments, needing substantial, consistent support to manage daily life.

Encouraging results in placebo cohort

For the placebo group, NTI says patients were unblinded at eight weeks and started NTI164 therapy at the low dose being 5mg/kg per day in week 9, up to 20mg/kg or the maximum tolerated dose per day in week 12.

NTI says despite just four weeks on therapy starting with a low dose of NTI164, these patients showed initial positive improvements in their clinical scores with CGI-S improving 21% to 4.11 (moderately ill) from 5.21 (markedly ill).

Furthermore, no serious adverse events or adverse events related to NTI164 were observed from week nine through to week 12 in all 54 children.

Five participants did record adverse events relating to headache, viral infections and a single urinary tract infection, which were not deemed NTI164 related.

‘Rapid clinical onset of improvement’

NTI executive director Dr Thomas Duthy said it has been good to understand the immediacy of benefits of NTI164 in patients previously receiving placebo who crossed-over to receive progressively higher amounts of the drug over four additional weeks before entering the 52-week extension part of the trial.

“The results once again confirm the benefits conferred are drug-related with a rapid clinical onset of improvement,” he says.

“Moreover, patients who have received NTI164 for 12 weeks in total continued to improved following the primary analysis at eight weeks, so much so their symptoms are barely noticeable,” he says.

“In general this means substantial lifestyle improvements for the patient and their caregivers, which makes us very proud to be supporting these clinical trials in autism.”

In another milestone NTI recently reported more than two years of treatment on its NTI164 for patients involved in its Phase 1/2 ASD trial.

The trial was an open-label study to evaluate the safety and efficacy of orally-administered NTI164 in children with ASD, recruiting 11 ASD patients.

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.