• Achieved record financial performance in 2024 with A$445 million in DAYBUE royalties
• Positive Phase 2 trial results for NNZ-2591 in Phelan-McDermid, Pitt Hopkins, and Angelman syndromes
• Alignment with FDA on Phase 3 trial design for Phelan-McDermid syndrome

Neuren Pharmaceuticals Ltd had another highly successful year in 2024, with record financial performance, record sales of DAYBUE, and positive Phase 2 trial results for NNZ-2591 in Phelan-McDermid, Pitt Hopkins, and Angelman syndromes. The company received A$445 million in royalties from Acadia for DAYBUE sales over 2023 and 2024, at a 100% pre-tax margin, leading to A$341 million in cash as of March 2025. Neuren was awarded the Australian Growth Company of the Year Award for Health and Life Sciences for the second consecutive year. The DAYBUE launch has been very successful, with net sales reaching US$348 million in the first full year. Acadia has committed additional resources to expand DAYBUE in the USA and forecasts continued growth in 2025. For NNZ-2591, Neuren was excited by the positive Phase 2 trial results in Phelan-McDermid, Pitt Hopkins, and Angelman syndromes, which validated the company's thesis that NNZ-2591 can potentially have a broad impact on neurodevelopmental conditions. Neuren has aligned with the FDA on the efficacy endpoints for the Phase 3 trial in Phelan-McDermid syndrome, and is eager to embark on the first ever Phase 3 trial in this indication. The company is also targeting hypoxic-ischemic encephalopathy (HIE) as a new indication for NNZ-2591. Neuren is in a strong financial position, with the resources to confidently execute its plans, and remains bullish about the shareholder value creation potential.

Acadia has provided CY2025 DAYBUE US net sales guidance of US$380 - 405 million, implying A$62 - 67 million in royalties to Neuren.

Neuren is excited to commence the Phase 3 trial for Phelan-McDermid syndrome later this year and seek alignment with the FDA on the registration path for Pitt Hopkins syndrome, Angelman syndrome, and hypoxic-ischemic encephalopathy. The company is also advancing Prader-Willi syndrome and/or undisclosed indications for NNZ-2591.