- Neuren Pharmaceuticals (NEU) has commenced the first phase of its NNZ-2591 clinical trial
- The trial is being conducted in Australia and will assess the safety, tolerability and pharmacokinetics in healthy adults
- Neuren is developing this drug to treat neurodevelopment disorders such as the Phelan-McDermid, Angelman and Pitt Hopkins syndromes
- These debilitating conditions currently have no approved therapies
- Once this phase is complete, Neuren will apply to the U.S. FDA for an Investigational New Drug application to commence phase two
- Phase 2 is expected to be completed in the December quarter
- Neuren is up a slight 1.74 per cent and shares are trading for $1.75 each
Neuren Pharmaceuticals (NEU) has commenced the first phase of its NNZ-2591 clinical trial.
The trial is being conducted in Australia and will assess the safety, tolerability and pharmacokinetics in healthy adults.
The first phase will test a single oral dose of the NNZ-2591 drug at three ascending dose levels. Subject to data review and non-clinical studies currently in progress, phase two of the trial will test twice-daily oral dosing for 14 days at two ascending dose levels.
“We believe this therapy has the potential to make a real difference to the treatment of these debilitating disorders, which currently have no approved therapies,” Executive Chairman Richard Treagus said.
Neuren is developing NNZ-2591 for Phelan-McDermid, Angelman and Pitt Hopkins syndromes, each of which has received Orphan Drug designation in the U.S.
An orphan drug is developed to treat medical conditions which, because they are so rare, wouldn’t be profitable to produce without government assistance.
The Phelan-McDermid, Angelman and Pitt Hopkins syndromes are rare disorders that are caused by gene mutations. While each disorder display varied symptoms, common symptoms include speech impairment, behavioural disorders, seizures, lack of balance and range of movements.
Data from the trial will form part of the Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) in order to proceed with phase two trials in children with these neurodevelopmental disorders.
The second stage of the trial is expected to be completed in the December 2020 quarter.
Neuren is up a slight 1.74 per cent and shares are trading for $1.75 each at 10:54 am AEST.