Posted: 5 December 2023
Neuren Pharmaceuticals (ASX: NEU) today announced that enrolment of subjects into its Phase 2 clinical trial of NNZ-2591 in Pitt Hopkins syndrome has been completed. Top-line results from the trial are expected to be available in Q2 2024.
Neuren CEO Jon Pilcher commented “We are grateful to all the people who have contributed to achieving this important milestone as we strive to develop a potential first therapy for Pitt Hopkins syndrome, including the Pitt Hopkins community and the trial site teams in the United States. We now look forward to the remaining patients completing the trial and to releasing the first results of treatment with NNZ-2591 in children with Pitt Hopkins syndrome.”
The Phase 2 trial in children aged 3 to 17 years at five hospitals in the United States is examining safety, tolerability, pharmacokinetics and efficacy over 13 weeks of treatment with NNZ-2591. All subjects receive NNZ-2591 as an oral liquid dose twice daily.
Neuren is also conducting Phase 2 clinical trials of NNZ-2591 in children with three other neurodevelopmental disorders – Phelan McDermid syndrome, Angelman syndrome and Prader-Willi syndrome. All four programs have been granted Orphan Drug designation by the US Food and Drug Administration (FDA) and are being developed under Investigational New Drug (IND) applications. Each syndrome is a seriously debilitating neurological disorder that emerges in early childhood and has no or limited approved treatment options.
