Neuren Pharmaceuticals (NEU) Investor Presentation summary

Strategic focus and pipeline

• Focus on neurodevelopmental disorders with severe unmet needs, targeting Rett, Fragile X, Phelan-McDermid, Pitt Hopkins, Angelman, Prader-Willi, SYNGAP1-related disorders, and HIE.

• NNZ-2591 positioned as a multi-indication platform, with Phase 3 underway for Phelan-McDermid and accelerated development in Pitt Hopkins and HIE.

• Trofinetide (Daybue) commercialized in North America for Rett syndrome, with global expansion plans in Europe, Japan, and other regions.

• Multiple late-stage and preclinical programs, with exclusive and wholly owned rights for key compounds.

• Robust financial position with A$300 million cash and A$473 million income from Daybue to date.

Commercial performance and financials

• Daybue net sales in the US grew 97% year-over-year, reaching US$348 million in 2024, with 2025 guidance of US$380–405 million.

• Royalties to Neuren from Daybue are projected at A$62–67 million for 2025, based on tiered royalty rates of 10–12% of net sales.

• Partnership with Acadia yields significant milestone payments and royalties, with up to US$250 million in North America and US$100 million upfront outside North America.

• Additional sales milestones and royalties possible from Europe, Japan, and rest of world, with tiered rates up to 15%.

• Completed A$50 million on-market share buyback in 2025.

Market expansion and growth drivers

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

• Only about one third of diagnosed patients currently start therapy; persistency rates exceed 50% after 12 months.

• Two-thirds of US patients have yet to try Daybue, with field force expansion and community outreach underway.

• Global expansion includes approval in Canada, MAA filed in Europe (potential approval Q1 2026), and clinical trial in Japan starting Q3 2025.

• Managed access programs active in Europe, Israel, and select other countries.