balance problems, motor impairment, and devastating seizures. At present, there are no recognized treatments for the condition, and patients need endless care.
The Human Research Ethics Committee has authorized three locations in Australia for a clinical study of the medication NNZ-2591. These sites are located in Sydney, Brisbane, and Victoria hospitals. The phase two trial will recruit nearly 20 children aged between 3 to 17 years with the illness in order to evaluate the drug’s safety, tolerability, pharmacokinetics, and effectiveness for an oral therapy period of 13 weeks.
Jon Pilcher, Neuren CEO, expressed delight over the approval. He said, “This is another important step achieved for the first clinical trial of NNZ-2591 in patients and Neuren’s first in Australia”. He added, “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.”
Each site must now get separate research governance approval prior to initiating the study and enrolling individuals. The outcomes of the trial are expected to be released in the first half of 2023. In the meantime, Neuren is expecting FDA approval for phase two studies of NNZ-2591 in both Phelan McDermid syndrome and Pitt Hopkins syndrome.
Parallel to the studies, Neuren is completing the basic work necessary to advance NNZ-2591 through phase three development across several neurodevelopmental diseases and Prader-Willi syndrome. According to the firm, Trofinetide, the primary ingredient of NNZ-2591, gave favorable results in a phase three clinical study for Rett syndrome and also finished a phase two clinical trial for Fragile X syndrome.
