Neuren Pharmaceuticals (ASX: NEU) today shared highlights from partner Acadia Pharmaceuticals’ presentation at the J.P. Morgan Healthcare Conference. Neuren is focused on developing therapies for neurological disorders that emerge in early childhood with limited treatment options. Their leading drug, DAYBUE, is approved in the US for treating Rett syndrome. Acadia Pharmaceuticals projects global net sales for DAYBUE to reach approximately US$700 million by 2028.
Acadia’s projection is driven by several factors. These include the rollout of DAYBUE STIX, a new powder formulation approved by the FDA in December 2025, which could attract families who previously declined or discontinued the liquid formulation. Continued benefits are also anticipated from the expansion of US customer-facing teams in Q2 2025. International expansion, contingent on approval in Europe with a CHMP opinion expected in Q1 2026, is also expected to contribute to sales growth.
Since its US launch in 2023, DAYBUE has treated over 2,000 Rett syndrome patients, with 12-month persistency increasing to 55%. Furthermore, over 300 patients are participating in Acadia’s real-world LOTUS study, contributing to ongoing data development. The number of diagnosed Rett syndrome patients in the US has increased to approximately 6,000, up roughly 30% since launch. DAYBUE STIX is currently rolling out with full availability expected by the beginning of Q2 2026.
Outside the US, DAYBUE oral solution has been approved by the Ministry of Health in Israel. A Phase 3 clinical trial of trofinetide in Japan is underway, with top-line results expected between Q4 2026 and Q1 2027. Acadia’s presentation is accessible on their website.
