NEUREN Koala Phase 3 clinical trial of NNZ-2591 PMS UPDATE

Neuren Pharmaceuticals reported today, February 6, that the first participant in its Koala Phase 3 clinical trial of NNZ-2591 for Phelan-McDermid syndrome (PMS) has completed the 4-week screening period and commenced the 13-week period of twice-daily dosing.

Koala is a Phase 3, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of NNZ-2591 in approximately 160 children aged 3 to 12 years with PMS. A screening period of up to 4 weeks is followed by treatment with NNZ-2591 or placebo for 13 weeks. The program has Fast Track, Rare Pediatric Disease and Orphan Drug designations from the US Food and Drug Administration.

More participants are scheduled to start dosing or start screening in February and March. To date, 25 families have been referred to the two activated trial sites. At this early stage, 37 more families are already on waitlists for a trial site in a convenient geographical location, with two sites expected to activate in February and 20 more across the US progressing towards activation.

For further study details and clinical trial information, click here.