Hypoxic Ischemic Encephalopathy

In the United States, HIE affects approximately one newborn every hour, or approximately 8,000 newborns each year.

Globally, it is implicated in more than 23% of neonatal deaths, with disproportionate burden in regions with limited access to advanced perinatal care. Worldwide, HIE remains a leading cause of neonatal mortality and long-term neurological disability, including cerebral palsy, epilepsy, and cognitive impairment.

Therapeutic options for HIE are extremely limited. The current standard of care utilizes therapeutic hypothermia with cooling caps or blankets, which aim to reduce metabolic demand and limit secondary injury. While hypothermia provides a modest reduction in mortality, its impact on long-term neurocognitive outcomes remains inconsistent, leaving a substantial unmet medical need for these babies.

Pegtarazimod is uniquely suited for development as a therapy for HIE due to its dual-targeting mechanism addressing key drivers of brain injury cascade. In preclinical models, pegtarazimod has demonstrated a marked reduction in cerebral infarction and meaningful improvements in brain function. Disease-modifying potential is supported by histological findings, neuroimaging (including MRI), and neurocognitive outcome measures, positioning pegtarazimod as a differentiated therapeutic approach in a setting with profound unmet need.