Axiom's stem cell therapy shows zero mortality in phase-1 trial for newborn brain injuries

Axiom Biosciences reported that its mesenchymal stem cell (MSC) therapy achieved a 0% mortality rate over two years in a phase‑1 study of newborns with severe brain injuries. The nine‑patient trial also noted improvements in neurodevelopmental milestones and no gross motor delays.

The therapy, derived from umbilical cord MSCs and co‑developed with South Korea‑based Medinno, was administered to five infants with severe intraventricular hemorrhage and four with hypoxic‑ischemic encephalopathy. Across all dose levels, no treatment‑related serious adverse events were observed, and none of the infants required anti‑epileptic medication.

These conditions typically carry a 46% mortality rate in the first year of life, and about half of affected infants develop cerebral palsy. In this study, Axiom saw no cases of cerebral palsy and no other major safety concerns.

Company leaders said the results provide broader validation for their brain‑targeting stem cell approach, though they acknowledge that larger studies will be needed to confirm efficacy and safety.