• FDA grants Fast Track designation for NNZ-2591 in Phelan-McDermid syndrome
• Neuren's Koala trial is the first ever Phase 3 clinical trial for PMS
• NNZ-2591 also has Fast Track designation in Angelman syndrome and Pitt Hopkins syndrome

Neuren Pharmaceuticals (ASX: NEU) announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for NNZ-2591 for the treatment of Phelan-McDermid syndrome (PMS). Fast Track is designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Currently, there are no FDA-approved treatments for PMS. Neuren recently initiated the 'Koala' Phase 3 randomized double-blind placebo-controlled clinical trial evaluating NNZ-2591 treatment in children aged 3 to 12 years with PMS. Alignment was reached with the FDA on the single Phase 3 trial design and endpoints to support a New Drug Application. Fast Track designation has also been granted for NNZ-2591 in Angelman syndrome (AS) and Pitt Hopkins syndrome, meaning Neuren now has Fast Track for NNZ-2591 across all three of these rare neurodevelopmental disorders. Neuren CEO Jon Pilcher commented that the company is pleased to announce the Fast Track designation as it approaches Phelan-McDermid Syndrome Awareness Day, and that the Koala trial is the first ever Phase 3 clinical trial for PMS, which they hope may lead to a much-needed treatment for this community.

Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in early childhood and have no or limited approved treatment options. The company's drug candidate NNZ-2591 is in development for Phelan-McDermid syndrome, Angelman syndrome and Pitt Hopkins syndrome, with positive results achieved in Phase 2 clinical trials.