Posted: 28 June 2023
Neuren Pharmaceuticals (ASX: NEU) today announced that the first site in the United States for its Phase 2 clinical trial of NNZ-2591 in Prader-Willi syndrome is now open.
Neuren CEO Jon Pilcher commented “The Neuren team is very excited to be working with the community to complete this important first study of NNZ-2591 in young children with Prader-Willi syndrome. We are eager to assess the potential impact of NNZ-2591, having observed highly encouraging effects in the pre-clinical model.”
The overall aim of this first clinical trial in patients is to expedite the generation of data that will enable the subsequent trial to be designed as a registration trial. In parallel with conducting the Phase 2 trial, Neuren is executing the additional development work required to be ready for Phase 3 development.
Neuren is also conducting Phase 2 trials of NNZ-2591 in children with three other neurodevelopmental disorders – Phelan-McDermid, Pitt Hopkins and Angelman syndromes. All four programs have been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and are being developed under Investigational New Drug (IND) applications. Each syndrome is a seriously debilitating disorder that emerges in early childhood and has no or limited approved treatment options.
