Neuren Pharmaceuticals (ASX:NEU) has announced it will develop its drug candidate NNZ-2591 to treat hypoxic-ischemic encephalopathy (HIE), a severe brain injury affecting newborns due to lack of oxygen or blood flow during or shortly after birth.
HIE is a leading cause of neonatal death and long-term neurological disability worldwide, with up to 45% of surviving children experiencing significant developmental impairment by age two—even with current standard-of-care treatment, therapeutic hypothermia. Neuren believes NNZ-2591 may offer a new approach by targeting both the acute injury phase and long-term neurological outcomes.
Based on strong preclinical data, the company expects the program to qualify for Orphan Drug and Rare Pediatric Disease designations from the US Food and Drug Administration. A pre-IND meeting with the FDA is planned for Q4 2025 ahead of initiating clinical trials.
Chief Science Officer Larry Glass said HIE was a natural extension of Neuren’s legacy in brain injury: “Neuren is now targeting a potential new paradigm in treatment to improve long-term outcomes for children and their families.”
Neonatologist and Harvard neuroscientist Dr Brian Kalish, who serves on Neuren’s advisory board, called the move promising: “NNZ-2591 has demonstrated an ability to target early effects of brain injury as well as longer-term effects on brain development and neuroplasticity.”
NNZ-2591 is already in Phase 2 development for three rare neurodevelopmental conditions—Phelan-McDermid, Pitt Hopkins, and Angelman syndromes—with positive early results. Neuren’s other asset, trofinetide (marketed as DAYBUE™), is approved in the US and Canada for Rett syndrome.
Shares are trading 1.55% higher at $12.12.
