Neuren Pharmaceuticals (ASX:NEU) highlighted key updates from its partner Acadia Pharmaceuticals' (NASXDAQ:ACAD) presentation at the 44th annual J.P. Morgan Healthcare Conference.
Acadia projected that global net sales of DAYBUE (trofinetide) could reach approximately US$700 million by 2028, driven by the upcoming rollout of DAYBUE STIX, expansion of the US commercial team, and international market growth.
Since its US launch in 2023, DAYBUE has been administered to more than 2,000 patients with Rett syndrome, with 12-month treatment persistency rising to 55%.
Over 300 patients are currently enrolled in Acadia's real-world LOTUS study.
The US rollout of the new DAYBUE STIX powder formulation began in Q1 2026, with full availability expected by Q2 2026.
Internationally, DAYBUE oral solution has received approval from Israel’s Ministry of Health.
In Japan, the Phase 3 trial of trofinetide is ongoing, with top-line results expected between Q4 2026 and Q1 2027.
Neuren, focused on developing therapies for serious neurodevelopmental disorders, has granted Acadia an exclusive worldwide license to develop and commercialize trofinetide.
Its second candidate, NNZ-2591, has shown positive Phase 2 results in Phelan-McDermid, Pitt Hopkins, and Angelman syndromes and has been designated an orphan drug in the US and EU.