The Food and Drug Administration (FDA) has granted Orphan Drug designation to PIC1-dPEG24 (ReAlta Life Sciences) for the treatment of hypoxic-ischemic encephalopathy (HIE) in neonates.

Hypoxic-ischemic encephalopathy is a brain injury that occurs when healthy infants suffer from oxygen deprivation to the brain. The disease can be caused by several events, including placental tear or injury from umbilical cord complications. PIC1-dPEG24 is believed to decrease reperfusion injury via complement activation, myeloperoxidase activity, neutrophil extracellular trap formation and antioxidant activities.

The designation is supported by data from preclinical studies in HIE animal models that demonstrated PIC1-dPEG24 decreased the area of brain infarction by 50% and improved neurocognitive outcomes. Additionally, PIC1-dPEG24 improved neurocognitive function in hypoxia-induced, treated rats.

The Company plans to submit an Investigational New Drug (IND) application to the FDA for HIE later this year, with first-in-man clinical studies expected in late 2020 and  phase 2 studies in neonates in 2021.

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“There is an urgent need for new therapeutic options to treat hypoxic-ischemic encephalopathy, a devastating condition that profoundly impacts newborns and their families,” said Ulrich Thienel, MD, PhD, ReAlta’s CEO. “The orphan drug designation for our RLS-0071 compound is an important milestone in ReAlta’s strategic plan to advance our PIC1 platform to address life-threatening medical needs.”

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