At the end of March 2025, Neuren Pharmaceuticals publicly shared their intent to explore their current approved neurodevelopmental NNZ-2591 compound for an indication in neonatal Hypoxic Ischemic Encephalopathy.
Hope for HIE has been engaged to provide patient-family input to all parts of the development process — from study design to longitudinal follow-up.
“This is a very exciting time for therapeutic advancement for HIE. We are excited to bring our organizational consulting and longitudinal patient-family engagement expertise to this project. HIE babies can’t wait, and we need more safe and effective therapies, and improved care and outcomes, for this important community to accelerate,” stated Betsy Pilon, Executive Director.
About Neuren Pharmaceuticals
Neuren is developing new drug therapies to treat multiple serious neurological disorders that emerge in
early childhood and have no or limited approved treatment options. Recognising the urgent unmet
need, all programs have been granted “orphan drug” designation in the United States. Orphan drug
designation provides incentives to encourage development of therapies for rare and serious diseases.
DAYBUE™ (trofinetide) is approved by the US Food and Drug Administration (FDA) and Health Canada for
the treatment of Rett syndrome. Neuren has granted an exclusive worldwide licence to Acadia
Pharmaceuticals Inc. for the development and commercialisation of trofinetide.
Neuren’s second drug candidate, NNZ-2591, is in development for multiple neurodevelopmental
disorders, with positive results achieved in Phase 2 clinical trials in Phelan-McDermid syndrome, Pitt
Hopkins syndrome and Angelman syndrome.
To read the full announcement, please click below:
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