An Open-Label Study of Oral NNZ-2591 in Phelan-McDermid Syndrome (PMS-001)

Phase 2 Completed 18 enrolled Neuren Pharmaceuticals Limited

NaSingle-groupOpen-labelTreatment

Summary

A study of the safety, tolerability and pharmacokinetics of NNZ-2591 and measures of efficacy in children and adolescents with Phelan-McDermid Syndrome.

Timeline

Start: 2022-08-08
Primary completion: 2023-11-01
Completion: 2023-11-17

Outcome

Met primary endpoint

release “In December 2023, Neuren announced positive top-line results from the Phase 2 clinical trial of NNZ-2591 in children with Phelan -McDermid syndrome (PMS).” neurenpharma.com ↗

Publications

Neumeyer AM, Srivastava S, Holder JL, Milad MA, Squires L, Jones NE, Glass L, Berry-Kravis E. NNZ-2591 in Children and Adolescents With Phelan-McDermid Syndrome: Single-Group, Open-Label, Phase 2 Trial Results. Neurol Genet. 2025 Dec 23;12(1):e200338. doi: 10.1212/NXG.0000000000200338. eCollection 2026 Feb.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	NNZ-2591	Small molecule	12 mg/kg	Oral