OPERAT I NG REV I EW CON T I NU E D 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 “ultra-rare”, and in each disorder there are tens of thousands of potential patients. Combined with Neuren’s strategy to develop treatments for multiple disorders in parallel, this results in a substantial commercial opportunity. 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, which extend as far as 2032 for trofinetide and 2034 for NNZ-2591. Further international patent applications have been filed for both drugs which, if granted, will extend to 2040. 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 FDA approval of DAYBUE™ (trofinetide) In March 2023, Neuren’s partner for trofinetide in North America, 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. Acadia expects DAYBUE to be available by the end of April 2023. DAYBUE is the first and only approved treatment for Rett syndrome. The FDA approval for DAYBUE was supported by pivotal efficacy from the positive LavenderTM Phase 3 trial, supportive safety and efficacy data from LilacTM openlabel extension trial, Neuren’s positive Phase 2 trial and the DaffodilTM safety and pharmacokinetic trial in children aged 2-5 years. Neuren Pharmaceuticals Limited Annual Repor t 2022 4
RkJQdWJsaXNoZXIy MjE2NDg3