Shots:

• The P-II trial evaluates NNZ-2591 (oral liquid dose, BID) in 20 children (aged 3-12yrs.) with PMS for 13wks. followed by an assessment post 2wks. of treatment. The 1EPs of the study include safety, tolerability & PK & 2EPs include 14 efficacy measures assessed by clinicians (CGI-I) & caregivers (CIC)
• As per the safety results, NNZ-2591 was well tolerated & efficacy data showed a significant improvement from baseline for 10/14 endpoints. Of 18 evaluable patients, 16 & 15 depicted improvements measured by CGI-I & CIC with mean scores 2.4 & 2.7 respectively
• The company is also conducting a P-II trial for NNZ-2591 in children with Pitt-Hopkins syndrome (results expected by Q2'24), Angelman syndrome & Prader-Willi syndrome

Ref: PR Newswire | Image: Neuren

Related News:- Acadia Expands its License Agreement with Neuren to Acquire Ex-North American Rights to Trofinetide and Global Rights to NNZ-2591

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