Neuren Pharmaceuticals Limited announced that the US Food and Drug Administration (FDA) has reviewed Neuren's Investigational New Drug (IND) application for NNZ-2591 in Prader-Willi syndrome (PWS) and given approval for Neuren to proceed with the planned Phase 2 clinical trial in children with PWS. Neuren is developing NNZ-2591 for four serious neurological disorders that emerge in early childhood. Phase 2 trials are currently ongoing in children with each of Angelman, Phelan-McDermid and Pitt Hopkins syndromes, for which there are no approved medicines.

All four programs have been granted Orphan Drug designation by the FDA. Neuren previously reported positive results in the Magel2-null mouse model of Prader-Willi syndrome, in which treatment with NNZ-2591 for 6 weeks normalized fat mass, insulin levels, IGF-1 levels and all behavioural deficits.