Neuren Pharmaceuticals has dosed the first participant in its Koala Phase 3 trial of NNZ-2591 for Phelan-McDermid syndrome (PMS). This marks the first-ever Phase 3 study conducted for this condition, which currently lacks any approved treatments. Neuren Pharmaceuticals is a biopharmaceutical company focused on developing therapies for neurodevelopmental disorders. Their aim is to address significant unmet needs through innovative treatments.
The randomised, double-blind, placebo-controlled trial intends to enrol approximately 160 children aged between 3 and 12 years old. The trial protocol includes a four-week screening period, followed by a 13-week period of twice-daily dosing. This rigorous approach is designed to thoroughly assess the efficacy and safety of NNZ-2591.
The program has been granted Fast Track, Rare Pediatric Disease, and Orphan Drug designations by the US Food and Drug Administration (FDA). These designations highlight the urgent need for effective treatments for PMS and provide Neuren with certain benefits to expedite the development and review process. Additional participants are scheduled to begin screening and dosing throughout February and March.
Neuren anticipates activating two additional sites this month, with a further 20 sites progressing towards activation across the United States. This expansion will increase the accessibility of the trial to a larger pool of potential participants, helping to accelerate the research and development of a treatment for Phelan-McDermid syndrome.
