2023 net sales of US$177m 2024E net sales of US$370 – 420m Historical Acadia guidance # * Ba US$ ^ Ne reac 370 23 67 87 177 420 Q2 2023 Q3 2023 Q4 2023 CY2023 CY2024 Acadia Guidance DAYBUE Net Sales (US$m) 21-23 45-55 80-87.5 OPERATING REVIEW CONTINUED As well as the exclusivity periods, Orphan Drugs have many other commercial advantages compared with existing markets that have apparently attractive large sales in which established products and companies have to be displaced. The serious and urgent unmet need results in a more supportive regulatory and pricing environment and strong engagement from the patient community and leading physicians. Historical data indicates a higher probability of achieving regulatory approval and the potential for immediate access to known patients means that a large sales organisation is less important. In short, the Orphan Drug business model targets a leadership position in markets with urgent need, at an attractive price and with a higher probability of getting to market. The neurodevelopmental disorders that Neuren is aiming to treat are “rare diseases”, however they are not “ultrarare”, and in each disorder there are tens of thousands of potential patients. COMMERCIAL EXCLUSIVITY In addition to the primary protection of the important exclusivity periods from Orphan Drug designation explained above, Neuren has additional commercial protection from issued patents and pending patent applications, which extend as far as 2041. Since trofinetide and NNZ-2591 are new chemical entities, following the first marketing authorisation for each drug, the term of one patent may potentially be extended by up to 5 years in many countries, including the United States, Europe and Japan. TROFINETIDE FOR RETT SYNDROME Successful launch of DAYBUE™ (trofinetide) in the United States In March 2023, Neuren’s partner for trofinetide, Acadia Pharmaceuticals (NASDAQ: ACAD), received US Food and Drug Administration (FDA) approval of DAYBUETM (trofinetide) for the treatment of Rett syndrome in adult and pediatric patients two years of age and older. On 17 April 2023, Acadia Pharmaceuticals launched DAYBUE™ (trofinetide) in the United States as the first approved treatment for Rett syndrome. Adoption of DAYBUE in the diagnosed Rett syndrome population has been faster than expected, with approximately 860 patients on DAYBUE at the end of February 2024. Caregivers and physicians have continued to report meaningful improvements in patients and the high demand has been well supported by access from Medicaid and private health insurance payors. In the United States there are approximately 5,000 diagnosed Rett syndrome patients and prevalence studies suggest the total number of patients may be 6,000 to 9,000. Acadia reported net sales of US$177 million for 2023 and has provided guidance for net sales in 2024 of between US$370 million and US$420 million. Further information about DAYBUE, including prescribing information can be accessed at www.DAYBUE.com Neuren Pharmaceuticals Limited Annual Report 2023 5
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