OPERATING REVIEW CONTINUED NNZ-2591 Placebo 160 80 80 Double-blind, 13 weeks NNZ-2591 Open label, 12 months Randomised 1:1 giving estimated 95% primary endpoint Screening Up to 4 weeks power for each coPHELAN-MCDERMID SYNDROME (PMS) Commencement of “Koala” - the first ever PMS Phase 3 program During 2025, Neuren agreed the primary endpoints for a single Phase 3 pivotal clinical trial of NNZ-2591 in PMS with the FDA. Alignment with FDA was previously reached on the other key features of the Phase 3 program at an End of Phase 2 Meeting. In the second half of 2025, Neuren initiated the first two sites in the US for the Koala Phase 3 trial and the first participants commenced dosing in February 2026. Koala is a Phase 3, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of NNZ-2591 in approximately 160 children aged 3 to 12 years with PMS. A screening period of up to 4 weeks is followed by treatment with NNZ-2591 or placebo for 13 weeks. All participants may be eligible to continue treatment with NNZ-2591 for 12 months in an open-label extension trial. Same age range (3-12) and same length of treatment (13 weeks) as Phase 2 ~20 trial sites, mostly in US Target dosing equivalent to dose tested in Phase 21 Program fully funded from existing cash 1 12.5 mg/kg per day in Phase 3 vs 12 mg/kg in Phase 2, and titration period two weeks in Phase 3 vs six weeks in Phase 2 Neuren Pharmaceuticals Limited Annual Report 2025 11
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