Neuren Pharmaceuticals Secures FDA Type C Meeting for Phelan-McDermid Syndrome Trial

Melbourne, Australia – Neuren Pharmaceuticals (ASX: NEU) has announced that the US Food and Drug Administration (FDA) has granted a Type C Meeting to discuss the primary efficacy endpoints for its upcoming pivotal Phase 3 trial of NNZ-2591 in Phelan-McDermid syndrome (PMS).

This marks a key step in Neuren’s clinical development strategy, following a successful Type B End of Phase 2 Meeting, where the company aligned with the FDA on all other critical aspects of the Phase 3 trial design. The face-to-face Type C Meeting is scheduled for early April, paving the way for the first-ever Phase 3 trial for PMS to commence mid-year.

CEO Jon Pilcher on the FDA Milestone

“We look forward to another constructive discussion with the FDA. Having a confirmed Type C meeting now establishes a clear timetable, and in parallel, we are continuing all our preparations,” said Neuren CEO Jon Pilcher.

About NNZ-2591 and Neuren’s Rare Disease Focus

Neuren is dedicated to developing therapies for serious neurological disorders that emerge in early childhood and currently have no or limited treatment options.

Key Developments:

  • NNZ-2591 has shown positive Phase 2 results in Phelan-McDermid syndrome, Pitt Hopkins syndrome, and Angelman syndrome.
  • All programs have received orphan drug designation in the US, providing regulatory incentives to support drug development for rare diseases.
  • Neuren’s first approved drug, DAYBUE™ (trofinetide), is FDA- and Health Canada-approved for Rett syndrome and is exclusively licensed to Acadia Pharmaceuticals (NASDAQ: ACAD) for global development and commercialization.

Next Steps for NNZ-2591 in Phelan-McDermid Syndrome

  • Early April: Type C Meeting with the FDA.
  • Mid-2025: Anticipated commencement of Phase 3 trials.
  • Ongoing regulatory and trial preparations to ensure a smooth trial launch.

With this FDA engagement, Neuren is progressing toward a groundbreaking Phase 3 trial for Phelan-McDermid syndrome, potentially delivering a much-needed first-ever treatment option for children with this rare neurological condition.