Neuren Pharmaceuticals Advances PMS Treatment Trials

Neuren Pharmaceuticals Limited (AU:NEU) has released an update.

Neuren Pharmaceuticals is set to begin Phase 3 trials for NNZ-2591, a potential first-ever treatment for Phelan-McDermid syndrome (PMS), following a constructive FDA meeting. The trial will be a 13-week, randomized, double-blind study on children aged 3 to 12, with continuity in an open-label extension study until commercial launch. The company is finalizing efficacy endpoints with the FDA and proceeding with trial preparations, including site selection and drug manufacturing.

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