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Neuren Pharmaceuticals Limited (ASX:NEU)

ALERT: Price-sensitive ASX Announcement for NEU
Current share price for NEU : $16.460 -0.71 (-4.14%)+
Announcement
Release
25 Aug 2025 9:27AM
Price at Release
$17.170
Full Release
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Summary
Neuren Pharmaceuticals Limited announced the initiation of the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome (PMS). The trial, which has received Institutional Review Board (IRB) approval, is the first-ever Phase 3 trial for PMS, a neurodevelopmental disorder with no approved treatments. The 13-week trial will involve approximately 160 children aged 3-12, with a potential 12-month open-label extension. The trial is fully funded by Neuren's current cash reserves, following regulatory alignment on trial design with the US FDA. Neuren focuses on developing therapies for serious neurological disorders, with its programs granted orphan drug designation in the US to encourage development for rare diseases.
Key Points
  • First investigational site initiated in the US for Phase 3 trial of NNZ-2591 for PMS.
  • Received IRB approval for the trial.
  • First-ever Phase 3 trial in Phelan-McDermid syndrome.
  • Approximately 160 children aged 3-12 will participate in a 13-week trial.
  • Participants may continue treatment in a 12-month open-label extension.
  • Trial is fully funded by Neuren's cash reserves.
  • Regulatory alignment achieved with US FDA on trial design.
  • Neuren's programs have orphan drug designation in the US.
IMPORTANT NOTE: This information is autogenerated and has not been reviewed for accuracy or completeness. You should refer to the full announcement here for further information.