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Neuren Pharmaceuticals
Neuren Pharmaceuticals (NEU) investor relations material
Neuren Pharmaceuticals Investor Presentation summary
Complete event summary combining all related documents: earnings call transcript, report, and slide presentation.Investor Presentation summary9 Nov, 2025
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.
Impact of Acadia's field force expansion on Daybue sales
NNZ-2591 HIE treatment paradigm details
NNZ-2591's competitive landscape in HIE
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Net income reached TRY 29.5 billion, with strong dividends and a robust net cash position.
Next Neuren Pharmaceuticals earnings date
H2 202527 Feb, 2026
Neuren Pharmaceuticals
Next Neuren Pharmaceuticals earnings date
H2 202527 Feb, 2026
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Frequently asked questions
Neuren Pharmaceuticals Limited is an Australia-based biopharmaceutical company focused on developing new therapies for neurodevelopmental disorders, including conditions that manifest in early childhood and are characterized by impaired brain cell connections and signaling. The company has a deep expertise in the field, accumulated through extensive clinical development efforts. Neuren Pharmaceuticals has gained recognition for developing the FDA-approved treatment, DAYBUE™ (trofinetide), specifically for Rett syndrome, marking a significant advancement in the treatment of this condition. Furthermore, the company is advancing its research and development pipeline with therapies aimed at addressing a range of neurodevelopmental disorders. The company is headquartered in Melbourne, Victoria, and its shares are listed on the ASX.
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