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