Shots:

• The P-II study assessed NNZ-2591's (oral, BID, dose escalation to 12mg/kg for first 6wks.) safety, efficacy, tolerability & PK for 13wks. in Pitt Hopkins syndrome children (n=16, 3-17yrs.). Study included a 4wk. screening period before treatment with a 2wk. follow-up
• Of 11 children completing the study, 9 had a mean CGI-I score of 2.6 (5 having 1 or 2), 8 had a mean CIC score of 3.0 (4 having 2), 6 had improved CGI-S scores from 6 to 5 in 3 of them & from 5 to 4 in another 3 while 8 had improved caregiver top 3 concerns overall score with language/communication as the common concern
• NNZ-2591 was well-tolerated without any serious TEAEs & with TEAEs unrelated to drug. 1 individual discontinued due to inability to complete the safety monitoring procedures & 4 due to TEAEs

Ref: PR Newswire | Image: Neuren

Related News:- Neuren Reports Results for NNZ-2591 in P-II Trial for the Treatment of Children with Phelan-McDermid Syndrome (PMS)

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