Neuren Pharmaceuticals advances NNZ-2591 for Phelan-McDermid syndrome to Phase 3 clinical trial

Neuren Pharmaceuticals advances NNZ-2591 for Phelan-McDermid syndrome to Phase 3 clinical trial

Neuren Pharmaceuticals has secured approval from the United States Food and Drug Administration (FDA) to progress into the first-ever pivotal Phase 3 clinical trial of NNZ-2591, a potential treatment for Phelan-McDermid syndrome (PMS). This landmark development is the outcome of a constructive End of Phase 2 meeting with the FDA, during which critical design aspects […]

Neuren Pharmaceuticals achieves breakthrough in Pitt Hopkins syndrome treatment

Neuren Pharmaceuticals achieves breakthrough in Pitt Hopkins syndrome treatment

Neuren Pharmaceuticals (ASX: NEU) has announced promising top-line results from its Phase 2 clinical trial of NNZ-2591, aimed at treating children with Pitt Hopkins syndrome (PTHS), a neurodevelopmental disorder with no approved treatments to date. The trial demonstrated statistically significant improvements across all four specifically designed efficacy measures, much to the optimism of clinicians and […]