- Neuren Pharmaceuticals (NEU) receives the green light to go ahead with a phase two clinical trial testing its NNZ-2591 drug on children with Prader-Willi syndrome
- It comes after the company applied for investigational new drug approval from the US FDA at the end of 2022, which has now been granted
- Neuren is developing NNZ-2591 for four serious neurological disorders which emerge in early childhood and currently have no approved medicines
- The company says it has already seen success of the drug in mouse models of Prader-Willi
- NEU shares were up 0.23 per cent, trading at $8.83 at 11:10 am AEDT
The US FDA has given Neuren Pharmaceuticals (NEU) the green light to go ahead with a phase two clinical trial to test its NNZ-2591 drug on children with Prader-Willi syndrome.
Neuren first applied for Investigational New Drug (IND) approval from the FDA for NNZ-2591 on December 22, 2022.
The FDA has now granted IND status and approved the trial to go ahead.
Neuren is developing its NNZ-2591 drug for four serious neurological disorders — Prader-Willi, Phelan McDermid, Angelman and Pitt Hopkins syndromes — which each emerge in early childhood and have no approved medicines.
The company said it has already seen the drug’s success in mouse models of Prader-Willi syndrome, with clinical trials currently ongoing in each of the syndromes.
The open-label, phase two trials will each enrol up to 20 children to examine the safety and efficacy of the drug over 13 weeks of treatment, marking the first trials of NNZ2591 in youths.
All four programs have been granted orphan drug designation by the FDA.
NEU shares were up 0.23 per cent, trading at $8.83 at 11:10 am AEDT.