Neuren Pharmaceuticals (ASX:NEU) has entered 2026 with growing confidence in the global trajectory of DAYBUE, its first-in-class treatment for Rett syndrome, as new data and commercial milestones continue to accumulate.
Speaking at the J.P. Morgan Healthcare Conference, Neuren’s commercial partner, Acadia Pharmaceuticals, projected that global net sales of DAYBUE could reach approximately US$700 million by 2028, underpinned by strong patient uptake in the United States and expanding international opportunities.
Since its US launch in 2023, more than 2,000 patients with Rett syndrome have been treated with DAYBUE, a significant milestone in a rare disease where therapeutic options have historically been limited. Importantly, treatment persistency continues to improve, with 12-month persistency now reaching 55 per cent, reflecting both clinical benefit and growing confidence among patients, families and clinicians.
A key driver of the next phase of growth will be the rollout of DAYBUE STIX, a powder formulation approved by the US Food and Drug Administration in December 2025. The new formulation is expected to address a critical barrier for some families who either declined or discontinued the original liquid formulation. A limited US launch has already commenced, with full availability anticipated by early in the second quarter of 2026.
Acadia also highlighted the impact of its expanded US commercial infrastructure, following the scale-up of customer-facing teams in mid-2025. This investment, combined with rising disease awareness, has contributed to an increase in the estimated number of diagnosed Rett syndrome patients in the US, now approximately 6,000, around 30 per cent higher than earlier estimates.
Internationally, momentum is also building. DAYBUE’s oral solution has been approved by the Ministry of Health in Israel, marking an important step in the product’s global expansion. In Europe, a regulatory decision is anticipated in the first quarter of 2026, while in Japan, a Phase 3 clinical trial of trofinetide is ongoing, with top-line results expected between late 2026 and early 2027.
For Neuren, DAYBUE represents the first validation of a broader strategy focused on serious neurological disorders that emerge in early childhood and have few or no approved treatments. Under its global licensing agreement, Acadia leads the development and commercialisation of trofinetide, while Neuren continues to advance its second drug candidate, NNZ-2591, across multiple neurodevelopmental disorders, each supported by orphan drug designation in the United States and Europe.