Neuren Pharmaceuticals (ASX:NEU) has received US FDA approval to proceed with the Phase 2 trial of NNZ-2591 in Pitt Hopkins syndrome (PTHS).
The company said the Investigational New Drug application (IND) for the PTHS program is now active.
CEO Jon Pilcher said, “We are excited to proceed with this groundbreaking trial in Pitt Hopkins syndrome and look forward to working with the community in the US. Neuren now has three INDs active for NNZ-2591 and we are advancing our plan to address multiple neurodevelopmental disorders that have such high unmet need.”
The Phase 2 trial will be conducted at four hospitals in the US. It will enrol up to 20 children aged 3 to 17 years with PTHS to examine safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591.
The company said top-line results from the trial are anticipated in the first half of 2023.
Neuren also recently received FDA approval to proceed with Phase 2 trials of NNZ-2591 in Angelman syndrome and Phelan-McDermid syndrome.
"In parallel with the three Phase 2 trials, Neuren is executing the foundational work to prepare for Phase 3 development of NNZ-2591 across multiple indications," said the company.