Neuren initiates development of NNZ-2591 to treat HIE

• Neuren to develop NNZ-2591 to treat hypoxic-ischemic encephalopathy (HIE), a devastating brain injury in newborns
• NNZ-2591 aims to target both acute effects and chronic impairments of HIE, going beyond current standard of care
• Neuren plans to seek Orphan Drug and Rare Pediatric disease designations from the FDA

Neuren Pharmaceuticals (ASX: NEU) announced the initiation of development of NNZ-2591 to treat hypoxic-ischemic encephalopathy (HIE), a devastating type of brain injury caused when a baby's brain does not receive enough oxygen or blood flow before or shortly after birth. HIE is one of the leading causes of neonatal death and neurodevelopmental disability worldwide, affecting 2-3 in 1,000 births in high-income countries and 10-30 per 1,000 births in low- and middle-income countries. The current standard of care for HIE is temporary hypothermia (cooling the head or whole body), which provides a modest decrease in mortality and severe neurodevelopmental disability. However, even with hypothermia, 40-45% of children who survive HIE have significant neurodevelopmental impairment at 2 years of age. Based on its therapeutic properties and compelling data from preclinical models, Neuren believes that NNZ-2591 can potentially provide a highly differentiated form of treatment, continuing beyond acute treatment in the neonatal intensive care unit to target both the acute effects and chronic impairments resulting from HIE. Neuren anticipates that NNZ-2591 in HIE will qualify for Orphan Drug and Rare Pediatric disease designations from the US Food and Drug Administration (FDA).Leveraging the platform of clinical, non-clinical and manufacturing data that Neuren has built for NNZ-2591, the company is targeting a pre-IND meeting with the FDA in Q4 2025 before initiating a clinical trial in HIE patients.

Neuren is very excited to announce HIE as a new indication for NNZ-2591, targeting a potential new paradigm in treatment to improve long-term outcomes for children and their families. The company is looking forward to working with experts in the field to make this program successful.