OPERATING REVIEW CONTINUED Net sales of DAYBUE in 2024 were US$348.4 million, with sequential growth in each quarter and record net sales of US$96.7 million in Q4 2024. These sales generated royalty income for Neuren in 2024 of A$56 million. 920 patients received DAYBUE in Q4 2024 and 62% of currently active patients had been on therapy for 12 months or longer. Acadia continues to collect and report realworld experience in the LOTUS study, with the majority of caregivers reporting meaningful improvements in patients. A characteristic of all long-term medicines is that not all patients who commence treatment will persist with treatment. Furthermore, for patients and caregivers, adjusting to a novel treatment regimen can take time, especially when it is the first treatment ever to become available. The number of patients commencing treatment and the proportion that persist with treatment long-term are key factors in the sales outcome. That persistency rate in the real world has consistently tracked at more than 10 percentage points above the clinical trial experience and has been stable at approximately 50% after 12 months of treatment. In Q4 2024 discontinuations improved by approximately 15% compared with Q3. The LOTUS study indicates that initial dose titration improves tolerability, suggesting that the future persistence rate for new patients can be higher than the early experience after launch. The number of diagnosed Rett patients in the US has grown from approximately 4,500 at the launch of DAYBUE to between 5,500 and 5,800. Prevalence studies suggest the total number of patients may be 6,000 to 9,000. Acadia has provided guidance for growth in US net sales in 2025 to between US$380 million and US$405 million. Q1 2025 net sales will be lower than Q4 2024 due to seasonal impacts, which is consistent with the previous year. With 70% of the diagnosed patients yet to try DAYBUE, there is substantial potential for growth in the US. In January 2025, Acadia announced initiatives to accelerate adoption, in particular among the 65% of patients that are treated outside the Rett syndrome Centers of Excellence. Acadia is expanding its field force by approximately 30%, optimizing patient support, launching branded Direct-to-Consumer campaigns to showcase DAYBUE benefits and utilizing a range of communication channels to bring the DAYBUE clinical data to life for both physicians and families. Further information about DAYBUE, including prescribing information can be accessed at www.DAYBUE.com 23 67 87 76 85 91 97 Q2 2023 Q3 2023 Q4 2023 Q1 2024 Q2 2024 Q3 2024 Q4 2024 US Net Sales (US$m) DAYBUE US Comparable Drug Launch (US)^ ^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics 1. Latest stats based on Acadia 4Q and full year 2024 Earnings Presentation 26 February 2025 and 43rd Annual JP Morgan Healthcare Conference Presentation 14 January 2025 CY23 DAYBUE US$177m CY24 DAYBUE US$348m (up 97% y-o-y) ^ For illustrative purposes only. Comparable Orphan Drug has different patient/clinician experience, approval and distribution/logistical dynamics Neuren Pharmaceuticals Limited Annual Report 2024 6
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