Neuren Pharmaceuticals (ASX: NEU) has announced that its Phase 2 clinical trial of NNZ-2591 in Angelman syndrome (AS) is open for enrolment.
The company said the first subjects are expected to imminently enter the trial 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 (IND) with US FDA. Top-line results are anticipated for the first half of 2023.
CEO Jon Pilcher said, “We are excited to commence this trial in Australia to assess the potential for NNZ-2591 to make a difference in Angelman syndrome, 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.”
The open-label Phase 2 trial (NCT05011851) will enrol a single group of up to 20 children aged 3 to 17 years with AS to examine the safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.
The company said all subjects will receive NNZ-2591 as an oral liquid dose twice daily, with titration up to the target mg/kg dose during the first six weeks of treatment, subject to safety and tolerability. It said the overall aim of the trial is to generate information to inform the design of a subsequent registration trial.
There are currently no approved medicines for AS, which is characterised by severe developmental delay and learning disabilities that become noticeable by the age of 6 – 12 months. Children and adults with AS typically have balance issues, motor impairment and can have debilitating seizures. Some individuals never walk, most do not speak and disruptive sleep also can be a serious challenge. Individuals have a normal life expectancy, but they require continuous care and are unable to live independently. AS is caused by a loss of function of the UBE3A gene on chromosome 15, with incidence estimated at between 1 in 12,000 and 1 in 24,000 people.