Latest Rett Syndrome News

Neuren Pharmaceuticals reports a 16% rise in Q2 2025 royalties driven by Acadia’s DAYBUE sales growth and expanding patient base in the US. Full-year sales guidance remains strong with European market entry on the horizon.

Neurotech International reports encouraging Phase I human pharmacokinetic results for its pediatric cannabinoid drug NTI164, alongside published clinical trial data in Rett Syndrome and key regulatory progress in the US and Australia.

Neuren Pharmaceuticals has been granted a US patent for NNZ-2591 to treat Pitt Hopkins syndrome, bolstered by promising Phase 2 clinical trial results and FDA orphan drug status.

Neuren Pharmaceuticals posted record 2024 financials driven by DAYBUE royalties and advanced NNZ-2591 into Phase 3 trials, while launching a $50 million share buy-back amid volatile market sentiment.

Neuren Pharmaceuticals has approved a significant long-term equity incentive plan tied to critical clinical and regulatory milestones for its drug candidate NNZ-2591, reinforcing management’s commitment to shareholder value.

Neuren Pharmaceuticals reports a robust 11% rise in DAYBUE™ US net sales for Q1 2025, alongside a 17% jump in royalty income, setting the stage for an anticipated European market entry in early 2026.

Neurotech International has reported excellent safety and tolerability for its lead drug NTI164 in 28-day GLP toxicology studies, paving the way for regulatory submissions and longer-term clinical development.

Neurotech International has inked a development deal with RH Farma to produce a pharmaceutical-grade cannabinoid drug targeting pediatric neurodevelopmental disorders, leveraging advanced European extraction technology.

Neuren Pharmaceuticals has received a significant cash inflow of approximately US$50 million following the sale of a Priority Review Voucher linked to its Rett syndrome treatment. This milestone underscores the growing value of Neuren’s neurological drug portfolio and its partnership with Acadia Pharmaceuticals.

Neuren Pharmaceuticals has secured a pivotal FDA Type C Meeting to discuss primary efficacy endpoints for its Phase 3 trial of NNZ-2591 targeting Phelan-McDermid syndrome, advancing its rare disease drug development.

Neurotech International has secured a $2.44 million R&D tax incentive refund, strengthening its cash position as it advances clinical trials for paediatric neurological disorders and prepares for regulatory submissions in FY2025.

Neurotech International reports key clinical progress in paediatric neurological disorders, securing FDA orphan drug designation for its Rett Syndrome treatment and advancing pivotal trials for ASD and PANDAS/PANS.