Neuren Targets Breakthrough Treatment for Newborn Brain Injury with NNZ-2591
- StockSurge Team
- Mar 27
- 2 min read
Updated: Mar 27
Neuren Pharmaceuticals (ASX: NEU) is expanding its pipeline with a new initiative: developing NNZ-2591 to treat (HIE), a severe brain injury affecting newborns.
HIE occurs when a baby’s brain is deprived of oxygen before or shortly after birth, often leading to lifelong disabilities such as developmental delays, cognitive impairments, cerebral palsy, and seizures. It remains one of the biggest causes of neonatal death and neurodevelopmental disability worldwide, affecting thousands of babies each year—2-3 per 1,000 births in high-income countries and up to 30 per 1,000 in lower-income regions.
The only current treatment is therapeutic hypothermia, which involves cooling a baby’s body to slow brain damage. While this approach reduces mortality and severe disability, up to 45% of surviving infants still suffer major developmental impairments by age two.
Neuren sees NNZ-2591 as a potential game-changer, with the ability to provide a more comprehensive treatment approach. Preclinical studies suggest the drug may help restore levels of insulin-like growth factor 1 (IGF-1), a key protein that is significantly impacted by HIE, and promote both early recovery and long-term brain development.
“We are very excited to announce HIE as a new indication for NNZ-2591,” said Neuren’s Chief Science Officer, Larry Glass. “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.”
FDA Meetings Planned
The company is planning a pre-IND (Investigational New Drug) meeting with the U.S. FDA in Q4 2025 and expects NNZ-2591 to qualify for Orphan Drug and Rare Paediatric Disease designations, which could help accelerate regulatory aspects of its development.
Dr. Brian Kalish, a neonatologist and neuroscientist at Boston Children’s Hospital and Harvard University, emphasized the need for new treatments. “There is a tremendous need for therapeutics to address both acute and chronic consequences [of HIE],” he said. “NNZ-2591 has demonstrated an ability to target early effects of brain injury as well as longer-term effects on brain development and neuroplasticity. Its role in restoring IGF-1 levels in the brain, which are significantly impacted by HIE, is very promising.”
Neuren has already had success with its first drug, DAYBUE™ (trofinetide), which won FDA approval for Rett syndrome. NNZ-2591 is currently in development for multiple neurodevelopmental disorders, including Phelan-McDermid syndrome, Pitt Hopkins syndrome, and Angelman syndrome, with promising Phase 2 results.
Neuren’s stock remained stable following the announcement, as investors assess the potential of expanding NNZ-2591’s reach into neonatal brain injuries. If successful, the company could be poised to generate the first pharmaceutical treatment for HIE.
