Company Interview / Acadia fuels Neuren's growth despite "old news" EU hurdle

Key points:

European regulator’s negative decision viewed as expected, with hopes for change after re-examinationUS market remains central, with robust sales and new product launch by Acadia PharmaceuticalsStrong guidance for royalties and profits in 2026, with lean cost base and substantial cash reservesOngoing progress on phase three clinical trials for NNZ-2001, targeting further growth

Neuren Pharmaceuticals’ Jon Pilcher outlines the latest developments regarding the European approval process for the company’s therapy for Rett syndrome, partnered with Acadia Pharmaceuticals. Pilcher states that the recent negative decision by the European regulator’s committee (CMP) is not unexpected and attributes the outcome to the committee’s focus on older clinical trial data rather than the broader evidence, including real-world experience from the US market, where the drug has been approved for three years. Pilcher highlights that Acadia will request a re-examination with new rapporteurs, bringing the opportunity for additional evidence to be considered, and hopes for a positive outcome by mid-year, primarily for the benefit of the Rett syndrome community in Europe.

Despite the setback in Europe, Pilcher emphasises the US market as the most significant driver for Neuren Pharmaceuticals (ASX:NEU) and notes strong sales growth for the therapy in the US, Canada, and Israel. Acadia has introduced a powder formulation, Debut Stix, to address patient preferences, potentially expanding the patient base. Growth guidance for sales in 2026 sits between 17% and 25%, further reinforcing the US market’s importance to Neuren.

Pilcher points to continued progress on the company’s second drug, NNZ-2001, now in phase three trials, with solid financials backing further research. Pilcher stresses a long-term approach in response to market volatility and short-term news-driven share price movements.