Neuren Pharmaceuticals starts first clinical trial for Phelan-McDermid Syndrome

The news: Neuren Pharmaceuticals has commenced its first Phase 3 clinical trial in Phelan-McDermid Syndrome (PMS), with the first participant completing a four-week screening period and beginning the 13-week dosing phase of NNZ-2591.

The context: The trial follows meeting feedback received from the US Food and Drug Administration on Thursday.

Neuren said additional participants are scheduled to begin screening the drug in February and March. The company was also named as sponsor of the 2026 Phelan-McDermid Syndrome Foundation Family Conference, The Climb We Make Together, which is set to be held in Colorado in July 2026.

What they said: "We are proud to be presenting sponsor of the PMSF family conference in July and we anticipate strong momentum for Koala as trial sites around the US progressively activate during the first half of this year," Neuren CEO Jon Pilcher said.