Phase 2 trial shows significant improvements in Phelan-McDermid syndromeHighlights:• Significant improvement was assessed by both clinicians and caregivers across multiple efficacymeasures• Improvements were consistently seen across clinically important aspects of Phelan-McDermidsyndrome, including communication, behaviour, cognition/learning and socialisation• Clinician and caregiver global efficacy measures showed a level of improvement typicallyconsidered clinically meaningful Clinical Global Impression of Improvement (CGI-I) - mean score of 2.4, with 16 out of 18children showing improvement assessed by clinicianso Caregiver Overall Impression of Change (CIC) – mean score of 2.7, with 15 out of 18children showing improvement assessed by caregivers• For 10 out of 14 efficacy endpoints, improvement from baseline on overall/total scores wasstatistically significant (Wilcoxon signed rank test p<0.05)• NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values orother safety parameters during treatment
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Clinical Global Impression of Improvement (CGI-I) - mean score of 2.4, with 16 out of 18children showing improvement assessed by clinicianso Caregiver Overall Impression of Change (CIC) – mean score of 2.7, with 15 out of 18children showing improvement assessed by caregivers• For 10 out of 14 efficacy endpoints, improvement from baseline on overall/total scores wasstatistically significant (Wilcoxon signed rank test p<0.05)• NNZ-2591 was safe and well tolerated, with no clinically significant changes in laboratory values orother safety parameters during treatment
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