Neuren Pharmaceuticals (ASX:NEU) has moved swiftly to reinforce its confidence in DAYBUE's (trofinetide) prospects in Europe, formally backing its partner Acadia Pharmaceuticals’ plan to seek a re-examination of a negative opinion from the European Medicines Agency’s Committee for Medicinal Products for Human Use.
The re-examination request follows confirmation that the CHMP adopted its earlier negative trend vote on Acadia’s Marketing Authorisation Application for DAYBUE for the treatment of Rett syndrome in patients aged two years and older. Acadia has confirmed it will now request a re-examination of that opinion, with Neuren stating it 'fully supports' the move.
The CHMP refusal was issued despite the pivotal LAVENDER trial meeting its co-primary and key secondary endpoints. The committee cited perceived deficits, including the magnitude of treatment effect at 12 weeks, the study’s capture of core Rett symptoms, and the influence of patient discontinuations on longer-term outcomes. Acadia has indicated that this feedback will inform its re-examination strategy, while reiterating its belief in trofinetide’s clinical value based on engagement from patients, caregivers and clinicians.
Neuren CEO Jon Pilcher underscored the company’s position, noting that DAYBUE has been making a difference for patients in approved markets for nearly three years and that the unmet need in Europe remains substantial and urgent. A re-examination by the CHMP is expected to conclude with an opinion likely by the end of the second quarter of 2026, following formal adoption of the initial decision.
The European regulatory outcome stands in contrast to continued commercial momentum elsewhere. DAYBUE generated net sales of US$391 million in 2025, up 12 per cent on the prior year, delivering $65 million in royalty income to Neuren. More than 1,000 unique patients received a shipment during the year, and the 12-month persistency rate improved to approximately 55 per cent.
Acadia has guided to 2026 net sales of US$460 million to US$490 million, implying potential royalties to Neuren of US$ 70 million to US$ 77 million under conservative assumptions. This guidance excludes any contribution from potential European commercialisation, meaning a successful re-examination outcome would represent upside beyond current forecasts.
Neuren’s broader financial position provides a solid foundation as the European process plays out. For 2025, the company reported royalty income of $65 million and profit after tax of $30 million. Cash and short-term investments stood at $296 million at 31 December 2025, supporting both pipeline investment and capital management initiatives.
The board has completed a $50 million on-market share buy-back and has initiated a new buy-back program from 2 March 2026, reflecting its view that the current share price materially undervalues Neuren’s assets. Management has emphasised that NNZ-2591 development programs for Phelan-McDermid syndrome, Pitt Hopkins syndrome and hypoxic-ischemic encephalopathy remain fully funded alongside the buy-back.