Neuren Pharmaceuticals (ASX:NEU) announced that results from its Phase 2 clinical trial of NNZ-2591 in children and adolescents with Phelan-McDermid syndrome have been published in Neurology Genetics, a peer-reviewed, open-access journal of the American Academy of Neurology.
The publication, titled "NNZ-2591 in Children and Adolescents With Phelan-McDermid Syndrome," details a 13-week, open-label Phase 2 study in 3- to 12-year-old patients. NNZ-2591 was well-tolerated, and both clinicians and caregivers reported meaningful improvements in key symptoms of PMS.
The Phase 2 findings have informed the design of Neuren's ongoing Phase 3 Koala trial, which is currently recruiting children and adolescents in the US.
Koala mirrors the Phase 2 study in age range, treatment duration, and dosing. Neuren CEO Jon Pilcher said, "We are pleased that Neurology Genetics has published our Phase 2 results, which provide a strong scientific basis for further evaluation of NNZ-2591 in our Koala Phase 3 study."