Neuren opens first site in US for Prader-Willi syndrome Phase 2 trial

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Neuren Pharmaceuticals (ASX:NEU) has announced that the first site in the US for its Phase 2 clinical trial of NNZ-2591 in Prader-Willi syndrome is now open.

CEO Jon Pilcher said, “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 company said the overall aim of this first clinical trial in patients is to expedite the creation of data that will enable the subsequent trial to be designed as a registration trial. 

The open-label Phase 2 trial in up to 20 children aged four to 12 is examining the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ2591. 

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 FDA and are being developed under Investigational New Drug applications.