Neurotech International Limited announced strong clinical results from twelve (12) paediatric Autism Spectrum Disorder (ASD) patients, following daily treatment of NTI164 over a 20 week period. In July 2022, Neurotech presented the results of the 28 day safety and efficacy data on a total of 14 patients. On the strength of the 28 day data, caregivers and clinicians recommended all patients continue to receive NTI164 treatment for an additional 54 weeks, with additional safety and efficacy results collected over that period.

The 20 week results were part of the Human Research Ethics Committee (HREC) approval to extend the trial, with approximately 6,300 assessment points collected to date. The Company will continue to collect safety and efficacy data over the additional period of treatment. Twelve (n=12) paediatric patients remained on daily treatment of NTI164 for the full duration of the 20 week period and were therefore evaluable for the analysis.

Their data at this time point (20 weeks) was compared to these same patients' data at baseline and 28 days. The two patients who discontinued treatment were censored (excluded) from the analysis undertaken (not related to drug effects of NTI164). All 12 patients will be followed-up for additional safety and efficacy analysis up-to 54 weeks, as per protocol revisions, and per HREC approval received in July 2022.

Overall, the significant results achieved across a large number of well-validated clinical assessment tools in children with ASD was very encouraging. For clinicians treating children with ASD, this repetitive and sustained effect is an important and meaningful clinical finding, when coupled with NTI164's strong safety profile. This supports the observation that the results are unlikely to be the result of durable placebo effects.

However, this will be confirmed via Neurotech's planned Phase II/III trial. At 20 weeks of treatment (n=12), the mean severity of illness rating of the CGI-S was 3.2, representing an improvement of 26% from baseline (CGI-S: 4.2). The mean difference between 20 weeks of treatment and baseline was -1.1, 95% Confidence Interval (CI) = -1.772, -0.3948, p value=0.005.

The mean difference of the severity of illness between 28 days of treatment and baseline was -0.714, 95% CI = -1.332, -0.097, p value=0.027. The results demonstrate that of the 40% of subjects markedly or severely ill at baseline ­ 0% of patients from week 4 onwards were classified as markedly to severely ill. In addition, these results show a significant improvement in average severity of illness scores over time.

It is therefore plausible that patients may see further improvements in the severity of illness over the extended 54 weeks of treatment under the HREC approval to continue daily treatments with NTI164 in these patients. At 20 weeks, the patients' adaptive behaviour as measured by the VinelandTM-3 adaptive behaviour scores, was significantly improved overall (mean difference of 3.8; 95% CI = 2.06, 5.61, p value=0.0005), and individual domains of communication (mean difference of 3.9; 95% CI = 1.76, 6.08, p value=0.002), daily living skills (mean difference of 4.7; 95% CI = 0.93, 8.40, p value=0.019), and socialisation (mean difference of 4.6; 95% CI = 1.12, 8.05, p value=0.014). Adaptive behaviour is an important factor in predicting long-term outcomes for people with ASD and improving this behaviour is a goal of any treatment intervention in ASD.

The Social Responsive Scale, 2nd Edition (SRS-2) is an internationally recognised tool used to identify social impairment associated with ASD and quantifies its severity using a Total score plus six sub-scales (Social Awareness, Social Cognition, Social Communication, Social Motivation, Restricted Interest and Repetitive Behaviour and Social Communication and Interaction). Of the 12 active patients, 11 completed the SRS-2. The mean total T-score for the 11 patients after 20 weeks of daily NTI164 treatment was 75.3 which is a significant improvement from baseline where it was 80.7 (mean difference of -5.45, 95% CI = -9.42, -1.49, p value=0.012). Within the SRS-2 treatment subscales, the greatest improvements were observed in Restricted Interest and Repetitive Behaviour (mean difference of -9, 95% CI = -15.15, -2.85, p value=0.009), Social Cognition (mean difference of -4.3, 95% CI = -7.99, -0.56, p value=0.03) and Social Communication and Interaction (mean difference of -4.1, 95% CI = -7.66, -0.52, p value=0.03).

At 20 weeks of daily therapy of NTI164 at the maximum tolerated dose for each patient, up to 20 mg/kg/day the side effects reported were not serious or severe and did not significantly interfere with patients' functioning. 58% of evaluable patients achieved approx. 20 mg/kg/day or higher dosing.

No changes were observed in any patients' full blood examination, liver function or kidney function tests. There were no changes observed in the patients' vital signs or weight. Excessive weight gain is a hallmark of Risperidone treatment in ASD, with Risperidone a regulatory approved treatment to manage irritability in children with ASD.

NTI164 has shown to be safe and well tolerated up to doses of 20/mg/kg/day. NTI164 has shown statistically significant efficacy in improving the symptoms associated with ASD after 20 weeks of daily therapy. 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. These results, combined with the extension of this study to accommodate parents/caregivers request to continue therapy, warrant for further clinical studies on NTI164 to assess long-term efficacy and safety.