In 2023 Neuren became the market darling of the ASX healthcare...

In 2023 Neuren became the market darling of the ASX healthcare sector after receiving US FDA approval via its US marketing partner for Trofinetide, selling under the name Daybue, to treat Rett’s Syndrome.Power says the countdown is on for another key catalyst for NEU. The company is expecting positive clinical trial results of NNZ-2591 into another rare disease called Angelman Syndrome within a month.“The market is keenly watching the soon to-be-released Angelman syndrome Phase 2 results,” he says.Neuren receives Rare Paediatric Disease Designation from FDAAhead of release of clinical trial results for Angelman Syndrome Neuren Pharmaceuticals (ASX:NEU) has received Rare Paediatric Disease (RPD) designation from the US Food and Drug Administration (FDA) for NNZ-2591 – developed to treat Phelan-McDermid Syndrome (PMS), known to be a rare disease that, primarily, affects some children.
An RPD designation is granted by the FDA for serious or life-threatening diseases affecting fewer than 200,000 patients in the US.“If the product is ultimately approved, NEU may now qualify for a priority review voucher that can be redeemed to receive priority review for a different product or sold,” Power says.“These PRVs can be sold for up to US$100m, so potentially a nice win fall for NEU.”Neuren says it is now is preparing for an end of Phase 2 meeting with the FDA in Q3 2024 to discuss the development program for NNZ-2591 in PMS, for which there is currently no FDA approved drug.The company previously reported results from a Phase 2 trial in which significant improvement was assessed by both clinicians and caregivers across multiple efficacy measures.