- Neuren Pharmaceuticals (ASX:NEU) enrols the last patient for its phase two trial using NNZ-2591 in Angelman syndrome
- The trial opened to children aged 3-17 and is taking place across three Australian hospitals over 13 weeks of treatment
- The drug already performs well in children suffering from Phelan-McDermid syndrome
- AS affects one in 12,0000-24,000 people, and Neuren’s trial is expected to bring topline results by the third quarter of 2024
- NEU shares last traded at $23.10
Neuren Pharmaceuticals (ASX:NEU) has entered the last phase of screening for its phase two clinical trial using NNZ-2591 in Angelman syndrome (AS), with all subjects now enrolled.
The phase two trial opened to children aged 3-17 and is taking place across three Australian hospitals. The trial aims to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.
The drug will be administered twice daily in an oral liquid dose and has already performed well in a study of children suffering from Phelan-McDermid syndrome. Furthermore, the company is awaiting the results of a clinical trial using the same drug for children with Pitt-Hopkins syndrome and Prader-Willi syndrome.
All four programs have been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and are being developed under Investigational New Drug (IND) applications.
“We are grateful to the Angelman community in Australia including both FAST Australia and the ASAA, together with the trial site teams, who have enabled this important milestone to be achieved,” Neuren CEO Jon Pilcher said.
AS is a severe developmental delay and learning disability that becomes apparent between the ages of 6-12 months. Children and adults with AS exhibit balance issues as well as motor impairment and they can have seizures.
The are no medications for the disorder, which renders some individuals unable to walk, unable to speak and left struggling to sleep. Individuals have a normal life expectancy, but they require continuous care and are unable to live independently.
Characterised by a loss of function to the UBE3A gene located on chromosome 15, the disorder affects one in 12,0000-24,000 people, and Neuren’s trial is expected to bring topline results by the third quarter of 2024.
NEU shares last traded at $23.10.