Neuren Pharmaceuticals (ASX:NEU) has reported record quarterly royalties from its commercial partner Acadia Pharmaceuticals, as sales of DAYBUE (trofinetide) continue to expand in the United States and abroad.
DAYBUE is approved by the U.S. Food and Drug Administration (FDA) and Health Canada for the treatment of Rett syndrome. Neuren has granted an exclusive worldwide licence to Acadia Pharmaceuticals Inc. for the development and commercialisation of trofinetide.
Acadia recorded US$101.1 million in DAYBUE net sales for the September quarter, an 11 per cent increase on the corresponding period in 2024 and up 5 per cent on the previous quarter. The result marks the first time quarterly sales of the Rett syndrome therapy have exceeded the US$100 million threshold.
For Neuren, this translated into A$16.4 million in third-quarter royalties, up 24 per cent year-over-year and 12 per cent from the second quarter of 2025. Acadia narrowed its full-year guidance for DAYBUE to between US$385 million and US$400 million, implying full-year royalties for Neuren of A$63 to 66 million, assuming an exchange rate of 0.65.
The number of patients receiving DAYBUE shipments rose above 1,000 for the first time, with 74 per cent of new prescriptions written by community-based physicians rather than specialists in designated Rett syndrome centres. The quarter also delivered the most significant increase in referrals since launch, supported by Acadia’s expanded field force.
Long-term treatment persistence remained steady above 50 per cent after 12 months, while overall U.S. market penetration reached about 40 per cent. Within community settings, penetration stood at roughly 27 per cent, highlighting continued room for growth.
Beyond the United States, Acadia expects a CHMP opinion on its European marketing application in the first quarter of 2026 and has initiated a Phase 3 trial in Japan. Named patient supply programs for DAYBUE are active in multiple regions, including Europe, Israel, the Middle East, and Latin America.
Neuren CEO Jon Pilcher said the result underscored the expanding global reach of DAYBUE and its impact for patients with Rett syndrome. “We’re delighted to see the continued growth in patient access and the strengthening of community-based prescribing,” Mr Pilcher said. “The increasing awareness and uptake of DAYBUE demonstrate both the clinical value of the therapy and the quality of Acadia’s commercial execution.”
Neuren is also progressing its second drug candidate, NNZ-2591, which has produced positive Phase 2 results in Phelan-McDermid, Pitt Hopkins, and Angelman syndromes. All three programs have received orphan drug designation in both the U.S. and the European Union.