Neuren Pharmaceuticals (ASX:NEU) is focused on neurodevelopmental disorders and rare disease drug development.
In this presentation, CEO Jon Pilcher highlights the success of DAYBUE, the first FDA-approved therapy for Rett Syndrome, with US sales reaching $445m in 2024 and projected to grow further. Acadia Pharmaceuticals, Neuren’s US partner, has also gained approval in Canada and plans to launch sales in Q3 2025, with European approvals expected in Q1 2026.
Meanwhile, Neuren’s second drug, NNZ-2591, has positive Phase 2 results across multiple syndromes and is advancing toward Phase 3 trials for Phelan-McDermid syndrome mid-year.
The company has a strong cash position of $359m, providing self-sufficiency in funding its clinical pipeline. Future catalysts include expanding DAYBUE into new markets and progressing NNZ-2591 towards commercialisation, with a long-term vision of developing a market-dominant therapy for multiple neurodevelopmental disorders.
