Latest Rett Syndrome News

Neuren’s partner Acadia secures positive CHMP opinion for DAYBU® in Rett syndrome, paving the way for first EU treatment approval and triggering significant milestone payments for Neuren.

Neuren Pharmaceuticals reported a 15% rise in 2025 royalties from DAYBUE®, boosted by strong US sales and a new powder formulation launch. The company is progressing its pivotal Phase 3 trial for NNZ-2591 in Phelan-McDermid syndrome and reassessing capital management amid solid cash reserves.

Neuren Pharmaceuticals saw a 20% jump in Q1 2026 DAYBUE net sales to US$101 million, with royalties up 23%. The Japan trofinetide trial has been fast-tracked, and the new powder formulation DAYBUE STIX gains early traction.

Neurotech International has opened its first Phase 3 clinical site for NTI164 targeting Autism Spectrum Disorder, reported positive 90-day toxicology results, and launched a new Rett syndrome research collaboration.

Neuren Pharmaceuticals’ partner Acadia Pharmaceuticals has broadened availability of DAYBUE STIX, a new FDA-approved powder formulation of trofinetide for Rett syndrome, in the US. Early caregiver feedback and expert consensus highlight its potential role in personalised patient care.

Neuren Pharmaceuticals’ partner Acadia Pharmaceuticals is seeking a re-examination of the European Medicines Agency’s refusal to approve trofinetide for Rett syndrome, aiming to overturn the setback and bring the treatment to European patients.

Neurotech International reports a dramatic reduction in half-year losses alongside significant clinical and regulatory progress for its lead cannabinoid therapy NTI164. The company also strengthens its cash position through a $4 million placement and a $4.7 million R&D tax refund.

Neuren Pharmaceuticals reports a 15% rise in 2025 royalties from its partner Acadia’s DAYBUE sales, with strong 2026 guidance signalling further growth. The launch of a new powder formulation and ongoing regulatory efforts in Europe and Japan add to the momentum.

Neurotech International has reported positive long-term safety results for its lead drug candidate NTI164, bolstering its path towards regulatory approval and chronic use in paediatric neurological disorders.

Neuren Pharmaceuticals has dosed the first patient in its groundbreaking Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome, a rare genetic disorder with no approved therapies. The trial marks a significant step forward for the PMS community and rare disease drug development.

The US Congress has reauthorized the Rare Pediatric Disease Priority Review Voucher program through September 2029, a move that could significantly benefit Neuren Pharmaceuticals as it advances its rare disease drug candidates.

Acadia Pharmaceuticals has received a negative vote from the European Medicines Agency’s committee on its application to market trofinetide for Rett syndrome in the EU, prompting plans for a re-examination request.