Neuren Pharmaceuticals (ASX:NEU) has announced that the US FDA has reviewed Neuren’s Investigational New Drug (IND) application for NNZ-2591 in Prader-Willi syndrome (PWS).
The company said the FDA had approved Neuren to proceed with the planned Phase 2 clinical trial in children with PWS.
PWS is a highly debilitating neurodevelopmental disorder, caused by defects in the 15q11-q13 region of chromosome 15. Infants with PWS have very low muscle tone and suffer from feeding difficulties. An unregulated appetite and easy weight gain characterize the later stages of PWS, which can lead to morbid obesity.
The company 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, PhelanMcDermid and Pitt Hopkins syndromes, for which there are no approved medicines. The FDA has granted all four programs Orphan Drug designation.
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.