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- Neuren Pharmaceuticals (NEU) receives the all clear from the US Food and Drug Administration (FDA) to commence phase two trials of its drug, NNZ-2591, on Phelan-McDermid syndrome (PMS)
- The trial will be conducted across four hospitals in United States on children between the ages of three and 12 with PMS
- The aim is to examine the drug over 13 weeks and test its safety, tolerability, pharmacokinetics, and efficacy
- Neuren is now preparing for phase three development of the drug for multiple treatments
- Shares were trading 1.67 per cent higher today at $4.25 each
Neuren Pharmaceuticals (NEU) has received US Food and Drug Administration (FDA) approval for its phase two trial of its drug, NNZ-2591, on Phelan-McDermid syndrome (PMS).
PMS is a rare genetic condition that causes developmental and speech delays, behavioural problems, ow muscle tone, motor delays, and epilepsy.
The trial will be conducted on 20 children with PMS, who are between the ages of three and 12, across four hospitals in United States.
The aim of the trial is to examine the drug over 13 weeks and assess the safety, tolerability, pharmacokinetics, and efficacy.
However, the trial is subject to ethics approval from the Institutional Review Boards before it can commence.
Additionally, Neuren is also undertaking phase two trials of the drug in other disorders such as Angelman syndrome and Pitt Hopkins, with plans on a trial for Prader-Willi syndrome.
“We are excited to be able to proceed with this eagerly anticipated trial following FDA approval of our second investigational new drug application (IND) for NNZ-2591 and we look forward to advancing in partnership with the Phelan-McDermid community in the US,” CEO Jon Pilcher said.
“We also anticipate a response from the FDA for our third IND in Pitt Hopkins syndrome in coming days.”
Neuren is now preparing for phase three development of the drug on multiple treatments
Shares were trading 1.67 per cent higher today at $4.25 each at 12:09 pm AEDT.
