Neuren Pharmaceuticals Annual Report 2025

76 85 91 97 85 96 101 110 Q1 2024 Q2 2024 Q3 2024 Q4 2024 Q1 2025 Q2 2025 Q3 2025 Q4 2025 920 954 987 Unique patients received DAYBUE in the Quarter 923 917 1,006 1,070 OPERATING REVIEW CONTINUED The number of unique patients receiving a DAYBUE shipment grew to 1,070 in Q4 2025, exceeding 1,000 for the first time. The persistency on therapy rate after 12 months of treatment increased to approximately 55%. There is substantial potential for further growth in the US. The number of diagnosed Rett patients in the US has grown from approximately 4,500 at the launch of DAYBUE to 6,000, with two-thirds of the diagnosed patients yet to try DAYBUE. Prevalence studies suggest the total number of patients may be 6,000 to 9,000. During 2025, Acadia completed an Net sales of DAYBUE in 2025 were US$391 million, with sequential growth in each quarter and record net sales of US$110 million in Q4 2025. Quarterly sales expansion of its DAYBUE field force in the US by ~30% to accelerate future growth in the community outside the Rett syndrome centers of excellence. In Q4 2025 momentum continued to build with 76% of new prescriptions originating from community physicians outside centers of excellence. Acadia has provided guidance for growth in net sales in 2026 to between US$460 million and US$490 million. The guidance comprises sales only from the US and international named patient programs. ABOUT RETT SYNDROME Rett syndrome is a seriously debilitating and life-threatening neurological disorder. It is first recognized in infancy and seen predominantly in girls, but can occur very rarely in boys. At diagnosis, Rett syndrome has often been misdiagnosed as autism, cerebral palsy, or non-specific developmental delay. Most cases of Rett syndrome are caused by mutations on the X chromosome on a gene called MECP2. Rett syndrome strikes all racial and ethnic groups and has been estimated to occur worldwide in 1 of every 10,000 to 15,000 female births, causing problems in brain function that are responsible for cognitive, sensory, emotional, motor and autonomic function. These problems can include learning, speech, sensory sensations, mood, movement, breathing, cardiac function, and even chewing, swallowing, and digestion. Rett syndrome symptoms appear after an early period of apparently normal or near normal development until six to eighteen months of life, when there is a slowing down or stagnation of skills. A period of regression then follows, with loss of communication skills and purposeful hand use, loss or impairment of walking, and the onset of stereotypic hand movements. Other problems frequently include seizures and erratic breathing patterns, an abnormal side-to-side curvature of the spine (scoliosis), and sleep disturbances. i Neuren Pharmaceuticals Limited Annual Report 2025 7

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