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- Neuren Pharmaceuticals (NEU) submits an investigational new drug (IND) application for its NNZ-2591 treatment for Prader-Willi syndrome
- The new IND was submitted to the US Food and Drug Administration (FDA) on December 22 for approval to proceed with a phase two trial
- 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
- Clinical trials are currently ongoing in each syndrome, with a series of top-line results announcements anticipated from the second half of 2023
- Neuren is up 4.62 per cent, trading at $8.16 at 1:40 pm AEDT
Neuren Pharmaceuticals (NEU) has submitted an investigational new drug (IND) application for its NNZ-2591 treatment for Prader-Willi syndrome.
The new IND was submitted to the US Food and Drug Administration (FDA) on December 22 for approval to proceed with a phase two trial in Prader-Willi — a genetic disorder that causes obesity, intellectual disability, and shortness in height. Common symptoms of the disease include constant hunger and behavioural problems.
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.
Clinical trials are currently ongoing in each of the syndromes — the first trials of NNZ2591 in children.
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.
All subjects will receive the drug as an oral liquid dose twice daily, with escalation in two stages up to the target dose during the first six weeks of treatment, subject to tolerability.
In the Phelan-McDermid syndrome and Angelman trial, the first subject in the oldest age group has now completed the treatment period of 13 weeks, showing a “good” safety and tolerability profile, according to Neuren.
No serious adverse events were reported, and no dose modifications were required.
Neuren said it’s working closely with the patient communities and trial sites to accelerate enrolment after the holiday period in the first half of 2023, in line with younger age groups being activated.
The four trials will likely conclude at different times, with a series of top-line results announcements now anticipated from the second half of 2023, commencing with Phelan-McDermid syndrome.
Neuren was up 4.62 per cent, trading at $8.16 at 1:40 pm AEDT.
