Posted: 14 August 2024
Neuren Pharmaceuticals (ASX: NEU) today announced top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). NNZ-2591 was safe and well tolerated as an oral liquid dose and improvements were seen in clinically important aspects of AS. Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline that was statistically significant and considered clinically meaningful. There are no approved treatments for AS despite its severely debilitating impact on the lives of patients, as well as their parents and siblings.
Neuren CEO Jon Pilcher commented “These results provide additional confirmation that NNZ-2591 as an oral liquid dose may address the core symptoms of diverse neurodevelopmental disorders, independent of the origin of the underlying genetics. We are very grateful to the people in the Angelman syndrome community and at the trial sites in Australia who enabled the successful completion of the trial.”
