Neuren Pharmaceuticals flagged results from its Phase 2 clinical trial of NNZ-2591 in children with Phelan-McDermid syndrome, a severe neurological disorder.
Both clinicians and caregivers observed improvements across multiple measures, including communication, behaviour, cognition/learning, and socialisation.
Based on Clinical Global Impression of Improvement and Caregiver Overall Impression of Change assessments, improvements were noted in 16 out of 18 and 15 out of 18 children, respectively.
NNZ-2591 was also safe and well-tolerated, with no clinically significant changes in laboratory values or other safety parameters.