- Neuren Pharmaceuticals (NEU) has approval from the US Food and Drug Administration (FDA) to proceed with Phase 2 trials of its NNZ-2951 drug, which is used to treat Pitt Hopkins syndrome
- Neuren has also recently received FDA approval to proceed with Phase 2 trials of NNZ-2591 in Angelman syndrome and Phelan-McDermid syndrome.
- Commencement is subject to ethics approval from the Institutional Review Board and top-line results from the trial are anticipated in H1 2023
- Commencement of Phase 2 trial treatment of a fourth neurodevelopmental disorder, Prader-Willi syndrome, is targeted for H2 2022
- NEU shares are down 2.61 per cent, trading at $4.11
Neuren Pharmaceuticals (NEU) has received approval from the US Food and Drug Administration (FDA) to proceed with Phase 2 trials of its NNZ-2951 drug, which is used to treat Pitt Hopkins syndrome (PTHS) among other conditions.
The Phase 2 trial will be conducted at four hospitals in the US, enrolling 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.
PTHS is a neurodevelopmental condition caused by the loss of one copy or a mutation of the TCF4 gene on chromosome 18, and currently has no treatment approved.
Commencement is subject to ethics approval from the Institutional Review Board and top-line results from the trial are anticipated in H1 2023.
Neuren has 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 preparing foundation work to prepare for Phase 3 development of NNZ-2591 across multiple indications.
Neuren CEO Jon Pilcher said the company is “advancing our plan to address multiple neurodevelopmental disorders that have such high unmet need”.
Commencement of Phase 2 trial treatment of a fourth neurodevelopmental disorder, Prader-Willi syndrome, is targeted for H2 2022 with results anticipated in H2 2023.
NEU shares were down 2.61 per cent, trading at $4.11 as of 11:28 am AEDT.