Neuren Pharmaceuticals (ASX:NEU) says it has received "clear and constructive guidance" from the US FDA Office of Neuroscience in pre-IND meetings for Phase 2 clinical trials of NNZ-2591 in Phelan-McDermid, Angelman and Pitt Hopkins syndromes.
It said all three syndromes are serious neurological disorders that emerge in early childhood and have no approved medicines.
The company said it will now proceed with preparing Investigational New Drug (IND) applications for clearance to start the trials.
"Subject to that clearance, the trials will commence as planned in H2 2021. Neuren aims to obtain top-line results from the trials in H2 2022," it said.
Neuren CEO Jon Pilcher said, “Our positive dialogue with FDA was very helpful and with only minor modifications confirmed our plans for three disorders that have such urgent unmet need. NNZ-2591 has ideal attributes – a unique mechanism of action, consistent and compelling results in the model of each disorder, a clearly identified optimum dose and proprietary manufacturing with high purity and yield. The final piece of the jigsaw is to demonstrate its impact in patients, so we are eager to expedite that data from these three Phase 2 trials.”
The Angelman syndrome trial will be conducted in Australia. The Phelan-McDermid and Pitt Hopkins syndrome trials will be conducted in the US. Each trial will test treatment of between 10 and 20 paediatric patients for 13 weeks.
The company said the primary objective will be to confirm the safety and tolerability of dosing for 13 weeks in these pediatric patients. The secondary objective will be to assess efficacy by observing the extent of change in each patient from a well-characterised baseline, using a range of efficacy measures.
"The overall aim is to generate the information that will enable the next stage to be the design of adequately powered pivotal registration trials with the optimum primary efficacy endpoints," said the company.