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."
Koala is a randomised, double-blind, placebo-controlled Phase 3 study evaluating the safety and efficacy of NNZ-2591 in approximately 160 children aged 3 to 12 years with PMS.
After a screening period of up to four weeks, participants receive either NNZ-2591 or placebo for 13 weeks.
At the time of reporting, Neuren Pharmaceuticals' share price was $18.35.