Neuren Pharmaceuticals (ASX:NEU) has initiated the development of NNZ-2591 to treat hypoxic-ischemic encephalopathy (HIE).
HIE is a type of brain injury caused when a baby’s brain does not receive enough oxygen or blood flow before or shortly after birth. About two to three in every 1,000 births in high-income countries and 10 to 30 per 1,000 births in low- and middle-income countries will be affected by HIE.
HIE is one of the leading causes of neonatal death and neurodevelopmental disability worldwide. It can lead to a range of symptoms in surviving children, including developmental delays, cognitive impairment, cerebral palsy, and seizures. Some children develop serious long-term complications that can affect them well into adulthood.
Currently, the only approved treatment for HIE is temporary hypothermia, which is cooling the head or whole body to lower the baby’s metabolic rate and give the brain some time to recover from the hypoxic event. Hypothermia provides a modest decrease in mortality and severe neurodevelopmental disability. However, even with hypothermia, 40 to 45 per cent of children who survive HIE have significant neurodevelopmental impairment at 2 years of age.
Neuren said it believes that NNZ-2591 can potentially provide a highly differentiated form of treatment continuing beyond acute therapy in the neonatal intensive care unit to target both the acute effects and chronic impairments resulting from HIE. It said it anticipates that NNZ-2591 in HIE will qualify for Orphan Drug and Rare Pediatric disease designations from the US FDA.
Neuren chief science officer Larry Glass said, “We are very excited to announce HIE as a new indication for NNZ-2591. Neuren has a long heritage in brain injury, dating back to our inception at the University of Auckland and scientists at the University also played a major role in the development of hypothermia as the current standard of care for HIE. Neuren is now targeting a potential new paradigm in treatment to improve long-term outcomes for children and their families.”