US 6,000 - 9,000 Rett patients 1 Launched inApr 2023 Canada 600 - 900 Rett patients1 Approved in Oct 2024 First sales in Q3 2025 Europe 9,000 - 12,000 Rett patients 1 MAA filed with potential approval Q12026 Initiation of Managed Access Program Q2 2025 Acadia building EU leadership and launch teams Japan 1,000 - 2,000 Rett patients 1 PMDA discussions ongoing; clinicalstudy start by Q3 2025 to support marketing application 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. OPERATING REVIEW CONTINUED i For Japan, Acadia has had productive discussions with the regulatory agency (PMDA) and plans to initiate a small clinical study by Q3 2025 to support a marketing application. There is urgent unmet need for a treatment for Rett syndrome around the world, evidenced by communications received from families, patient support groups and physicians. Acadia is assessing a strategic approach to make DAYBUE available in select markets through named patient programs in 2025. 1 Acadia estimates Neuren Pharmaceuticals Limited Annual Report 2024 8
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