Neuren Pharmaceuticals (ASX: NEU) received positive top-line results from its Phase 2 clinical trial of its NNZ-2591 candidate in children with Angelman syndrome (AS).
The NNZ-2591 was safe and well tolerated as an oral liquid dose and improvements were seen in clinically important aspects of AS.
Clinician and caregiver global efficacy measures specifically designed for Angelman syndrome showed a level of improvement from baseline that was statistically significant and considered clinically meaningful.
CEO Jon Pilcher said there are no approved treatments for AS despite its severely debilitating impact on the lives of patients, as well as their parents and siblings.
“These results provide additional confirmation that NNZ-2591 as an oral liquid dose may address the core symptoms of diverse neurodevelopmental disorders, independent of the origin of the underlying genetics,” Mr Pilcher said.
“We are very grateful to the people in the Angelman syndrome community and at the trial sites in Australia who enabled the successful completion of the trial.”
About Angelman syndrome
Angelman syndrome (AS) is a rare neurodevelopmental disorder that is estimated to occur in between one in 10,000 and 20,000 live births. It is caused by a loss of function of the UBE3A gene in chromosome 15 derived from the mother.
Angelman syndrome shares symptoms and characteristics with other disorders, which means that misdiagnosis occurs often.
Children and adults with AS typically have balance issues, motor impairments and can have debilitating seizures. Disruptive sleep is often a serious challenge. Some never walk and most do not speak. Individuals with AS have a normal life expectancy but require continuous care and are unable to live independently.
