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- Neuren Pharmaceuticals (NEU) opens its Phase Two clinical trial of its NNZ-2591 drug in Angelman syndrome (AS) for enrollment
- The trial is being conducted at three hospitals in Brisbane, Melbourne and Sydney under an investigational new drug application with the US Food and Drug Administration
- Neuren says the trial will aim to examine the safety, tolerability, pharmacokinetics and efficacy of NNZ-2591 over 13 weeks of treatment
- In parallel with the Phase Two trial, Neuren is also executing the foundational work to prepare for Phase Three development across multiple indications
- Shares in Neuren are trading grey at $3.95 per share at 12:22 pm AEST
Neuren Pharmaceuticals (NEU) has opened its Phase Two clinical trial of its NNZ-2591 drug in Angelman syndrome (AS) for enrollment.
The first subjects are expected to enter the trial “imminently” at the Centre for Clinical Trials in Rare Neurodevelopmental Disorders at Children’s Health Queensland Hospital.
The trial is being conducted at three hospitals in Brisbane, Melbourne and Sydney under an investigational new drug application with the US Food and Drug Administration.
Neuren said the aim of the trial was to examine the safety, tolerability, pharmacokinetics and efficacy of NNZ-2591 over 13 weeks of treatment under which all subjects will receive an oral liquid dose twice daily.
CEO Jon Pilcher said Angelman syndrome was a “seriously debilitating” condition with no approved medicines.
“In the ube3a knockout mouse model of Angelman, treatment with NNZ-2591 normalized all the deficits, so we are now eager to observe the effects of treatment in children,” Mr Pilcher said.
In parallel with the Phase Two trial, Neuren is also executing the foundational work to prepare for Phase Three development across multiple indications.
Angelman syndrome is characterized by severe developmental delay and learning disabilities that become noticeable by the age of six to 12 months.
Shares in Neuren were trading grey at $3.95 per share at 12:22 pm AEST.
