Neuren Pharmaceuticals (ASX:NEU) has announced it has received approval from the Human Research Ethics Committee for its Phase 2 clinical trial of NNZ-2591 in Angelman syndrome (AS).
The company said the approval covers the three sites in Australia that will conduct the trial - Queensland Children’s Hospital/Centre for Children’s Health Research, Sydney Children’s Hospital and Austin Health in Victoria.
Neuren CEO Jon Pilcher said, “This is another important step achieved for the first clinical trial of NNZ-2591 in patients and Neuren’s first in Australia. We are very excited to be working closely with the local Angelman syndrome community and are eager to accelerate development of this potential therapy which has shown such promise to date.”
The company said the trial will enrol up to 20 children aged 3 to 17 years to examine the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with orally administered NNZ-2591. Results from the trial are expected in the first half of 2023.