• First investigational site initiated in the US for Neuren's Phase 3 trial of NNZ-2591 in Phelan-McDermid syndrome
• Randomized, double-blind, placebo-controlled trial to assess treatment in approximately 160 children aged 3-12 with PMS
• Trial program fully funded by Neuren's existing cash reserves

Neuren Pharmaceuticals (ASX: NEU) today announced the initiation of the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome (PMS), after receiving Institutional Review Board (IRB) approval. Other trial sites in the US are at various stages of the initiation process. This is the first ever Phase 3 trial in PMS, a serious neurodevelopmental disorder with no approved treatments. The randomised, double-blind, placebo-controlled trial will assess treatment for 13 weeks in approximately 160 children aged 3-12 with PMS. All participants may be eligible to continue treatment with NNZ-2591 for 12 months in an open-label extension trial. The trial program is fully funded by Neuren's existing cash reserves. Neuren conducted an End of Phase 2 Type B meeting and a subsequent Type C meeting with the US Food and Drug Administration, at which alignment was reached on the design of the Phase 3 trial, including the primary efficacy endpoints.