Neuren Pharmaceuticals (NEU) Investor Presentation summary

Strategic focus and pipeline

• Focus on neurodevelopmental disorders with severe unmet needs, including Rett, Fragile X, Phelan-McDermid, Pitt Hopkins, Angelman, Prader-Willi, SYNGAP1-related disorders, and Hypoxic-Ischemic Encephalopathy (HIE).

• NNZ-2591 is being developed as a multi-indication platform, with Phase 3 trials underway for Phelan-McDermid and advancing in Pitt Hopkins and HIE.

• Trofinetide (Daybue®) is commercialized for Rett syndrome, with global expansion ongoing.

• Orphan Drug and Rare Pediatric Disease designations secured for key programs.

• Pipeline includes multiple late-stage and registration-phase assets.

Financial performance and commercial outlook

• $490 million income from Daybue® in 2023 to date, with $310 million cash as of September 2025.

• Royalty income from Daybue® projected at A$63-66 million in 2025, based on US net sales guidance of $385-400 million.

• Tiered royalties from Acadia partnership range from mid-teens to low-20s percent of net sales, with significant milestone payments for US, Europe, and Japan.

• Record number of active Daybue® patients in Q3 2025, with 74% of new prescriptions from outside centers of excellence.

• Share buyback of A$50 million completed in 2025.

Market expansion and growth drivers

• US Rett patient base expanded to 5,500-5,800 diagnosed, with theoretical prevalence up to 9,000.

• Global expansion includes Canada (approved), Europe (MAA filed, opinion expected Q1 2026), Japan (Orphan Drug status, clinical trial underway), and named patient programs in other regions.

• Key growth drivers: increasing diagnosis, therapy initiation, and patient persistency rates.

• Acadia field force expanded by 30% in 2025 to support Daybue® uptake.

• Substantial market opportunities in PMS, PTHS, and HIE, with tens of thousands of potential patients globally.