Neuren Pharmaceuticals (ASX: NEU) announced that the US Food and Drug Administration has granted a Type C Meeting to finalise discussions on the primary efficacy endpoints for its planned pivotal Phase 3 clinical trial of NNZ-2591, aimed at treating Phelan-McDermid syndrome.
The Type C Meeting, scheduled for early April, follows a successful Type B End-of-Phase 2 Meeting where the FDA and Neuren aligned on key aspects of the trial program.
The FDA deemed a Type C Meeting the most appropriate setting for completing discussions on efficacy endpoints.
Neuren CEO Jon Pilcher stated, "We look forward to another constructive discussion with the FDA. With the meeting confirmed, we now have a clear timetable and are proceeding with preparations for a mid-year launch of the first-ever Phase 3 trial for children with Phelan-McDermid syndrome."
Neuren Pharmaceuticals focuses on developing treatments for serious neurological disorders that emerge in early childhood and currently lack effective treatment options.
The company’s drug programs have received orphan drug designations in the United States, granting development incentives for therapies targeting rare and serious conditions.