ASX:NEU - Jon Pilcher on Ausbiz: Neuren powers ahead with new Rett Syndrome treatment in US

Key Points:

DAYBUE STIX powder for Rett syndrome launches fully in the US via Acadia Pharmaceuticals
Early user feedback cited at ~80% satisfaction, targeting families dissatisfied with the liquid form
Neuren (ASX:NEU) guides to rising royalties and a US$50m milestone when sales reach US$500m annually
NNZ-2591 enters phase 3 in Phelan-McDermid syndrome, viewed as a larger fully owned pipeline asset

Neuren’s Rett syndrome powder, DAYBUE STIX, aims to accelerate US growth. Jon Pilcher, chief executive of Neuren Pharmaceuticals, outlines strong momentum for the company’s Rett syndrome treatment and new formulation, DAYBUE STIX, now broadly available in the United States through Acadia Pharmaceuticals. Pilcher states that the existing oral solution has been on the market for about three years, generating US$391 million of sales in 2025, with 2026 sales forecast at US$460–490 million, implying a 19–24% uplift.

DAYBUE STIX is presented as a key growth driver. Pilcher says early limited launch feedback shows around 80% satisfaction. The powder formulation is viewed as addressing major concerns from families: no refrigeration, no preservatives or colour, much lower sugar and flexible dosing in liquids children prefer. He says Acadia identified roughly 400 families who avoided or discontinued the liquid, which Pilcher suggests could materially expand the current base of just over 1,000 patients on treatment.

Neuren’s revenue stream currently stems largely from royalties and milestones from Acadia, with US$65 million royalties reported in 2025 and guidance for US$70–77 million in 2026. Pilcher highlights over US$500 million in cumulative income since 2023 and points to the next US$50 million milestone once annual North American sales reach US$500 million, anticipated in 2027. He also flags NNZ-2591, now in phase 3 for Phelan-McDermid syndrome, as a potentially larger opportunity than Rett and fully owned by Neuren (ASX:NEU).