Neuren Pharmaceuticals (ASX:NEU) sats it has received approval from the US FDA to proceed with the Phase 2 trial of NNZ-2591 in Angelman syndrome (AS).
The company said the Investigational New Drug application (IND) for the AS program is now active.
CEO Jon Pilcher said, "FDA approval of the first IND for NNZ-2591 is a huge step forward in Neuren's plan to develop NNZ-2591 for four serious neurodevelopmental disorders. The Angelman syndrome Phase 2 trial in Australia has been keenly awaited by the Angelman community and we are very excited to be able to proceed. We also now anticipate receiving clearance next month of our IND applications for similar Phase 2 trials in Phelan-McDermid and Pitt Hopkins syndromes, subject to completion of those FDA reviews."
The AS Phase 2 trial will be conducted at three hospitals in Australia, enrolling up to 20 children aged 3 to 17 years to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.
Commencement of the trial is subject to Ethics Committee approval.
The company said results from the trial are anticipated in the first half of 2023 and that it is also executing the foundational work to prepare for Phase 3 development.
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.
