Neuren's Phase 2 trial results in Angelman syndrome

Neuren Pharmaceuticals (ASX: $NEU) has released top-line results from its Phase 2 clinical trial of NNZ-2591 in children with Angelman syndrome (AS). The trial demonstrated statistically significant and clinically meaningful improvements in the core symptoms of Angelman syndrome, with no serious adverse events reported during the treatment period.

Executive commentary on Phase 2 trial results

Neuren CEO Jon Pilcher expressed gratitude to the Angelman syndrome community and trial sites in Australia for their contribution to the successful completion of the trial. He stated, '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.'

Summary of Phase 2 trial results and outlook

Neuren's Phase 2 trial of NNZ-2591 in children with Angelman syndrome demonstrated statistically significant and clinically meaningful improvements in communication, behavior, cognition, and motor abilities. The results further strengthen confidence in the potential of NNZ-2591 for multiple neurodevelopmental disorders. Neuren is actively developing NNZ-2591 for various seriously debilitating neurological disorders with different genetic origins, aiming to address the urgent unmet need for effective treatments. The company's positive results in Phase 2 trials for other syndromes and its upcoming meeting with the US Food and Drug Administration indicate a promising outlook for the development and potential approval of NNZ-2591 for multiple neurodevelopmental indications.