- Neuren Pharmaceuticals (NEU) opens the first site in the United States for its phase two clinical trial in Prader-Willi syndrome
- Neuren’s open-label, phase two trial will be conducted in up to 20 children with the condition to examine the efficacy of its NNZ-2591 drug over 13 weeks of treatment
- In parallel with conducting the phase two trial, Neuren will execute additional development work ahead of its next phase of development
- Concurrently, Neuren is conducting phase two trials of NNZ-2591 in children with three additional neurodevelopmental disorders
- Neuren Pharmaceuticals is down 0.58 per cent, trading at $12.19 at 3:57 pm AEST
Neuren Pharmaceuticals (NEU) has opened the first site in the United States for its phase two clinical trial in Prader-Willi syndrome.
Prader-Willi syndrome is a highly debilitating neurodevelopmental disorder, which is estimated to occur once in every 30,000 people. Along with very low muscle tone and feeding difficulties in infancy, the syndrome also causes an unregulated appetite, learning disabilities, sleep disturbances and gastrointestinal complications.
Neuren’s open-label, phase two trial will be conducted on up to 20 children aged between four and 12 years old with the condition, to examine the safety, tolerability, and efficacy of NEU’s NNZ-2591 drug over 13 weeks of treatment.
Each subject will receive two daily doses of the drug as an oral liquid.
In parallel with conducting the phase two trial, Neuren has planned to execute additional development work ahead of its next phase of trials.
“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,” NEU CEO Jon Pilcher said.
“We are eager to assess the potential impact of NNZ-2591, having observed highly encouraging effects in the pre-clinical model.”
Concurrently, Neuren is conducting phase two trials of NNZ-2591 in children with three other neurodevelopmental disorders: Phelan-McDermid, Pitt Hopkins and Angelman syndromes.
Each of the four programs has been granted orphan drug designation by the US Food and Drug Administration (FDA) and are being developed under Investigational New Drug (IND) applications.
NEU was down 0.58 per cent, trading at $12.19 at 3:57 pm AEST.