Neuren Releases Results from Phase 2 Clinical Trial of NNZ-2591

Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS).

Highlights:

• • Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline after 13 weeks that was statistically significant (Wilcoxon signed rank test p<0.05) and considered clinically meaningful:

• • Clinical Global Impression of Improvement (CGI-I) – mean score of 3.0, with 11 out of 13 children showing improvement assessed by clinicians (p=0.0010)

• • Caregiver Overall Impression of Change (CIC) – mean score of 3.2, with 8 out of 12 children showing improvement assessed by caregivers (p=0.0273)

• • Every child in the younger age segment of 3-12 years showed improvement measured by both the CGI-I (mean score 2.8 p=0.0078) and the CIC (mean score 2.6 p=0.0078)

• • Improvements were seen in clinically important aspects of Angelman syndrome, including communication, behavior, cognition and motor abilities

• • NNZ-2591 was safe and well tolerated as an oral liquid dose, with no serious adverse events and no meaningful trends in laboratory values or other safety parameters during treatment

• • Results further strengthen confidence in potential of NNZ-2591 for multiple neurodevelopmental disorders