Axiom Biosciences Reports Positive Phase 1 Results for Investigational Stem Cell Therapy in Newborns with Severe Brain Injury

• Investigational WJ-MSC therapy associated with no deaths at 12 months and statistically significant improvements in long-term cognitive and motor outcomes
• Results support planned expansion of the regenerative platform into adult ischemic stroke and additional neurological indications
  
  

SAN DIEGO, July 14, 2026 (GLOBE NEWSWIRE) -- Axiom Biosciences, a clinical-stage biotechnology company developing next-generation regenerative therapies and targeted biologics, today announced positive Phase 1 results for its investigational Wharton’s Jelly-derived mesenchymal stem cell (WJ-MSC) therapy for neonatal brain injury. The therapy, co-developed with South Korea-based Medinno Inc., is being developed to treat intraventricular hemorrhage (IVH) and hypoxic-ischemic encephalopathy (HIE), two severe neurological conditions in newborns associated with high rates of mortality and long-term disability.

The investigational therapy consists of conditioned WJ-MSCs administered directly into the central nervous system. The Phase 1 dose-escalation study evaluated the safety, tolerability, and preliminary efficacy of the therapy across a range of doses in 9 newborns diagnosed, 5 with severe IVH and 4 with HIE.

Key Clinical Trial Findings

• Achieved a 0% mortality rate at 12 months compared to the historical natural mortality rate of approximately 46% within the first year of life for infants with severe IVH.
• Demonstrated a favorable safety profile, with no treatment-related serious adverse events (SAEs) observed across all dose cohorts.
• Produced promising improvements in long-term functional outcomes at 24 onths, including key cognitive and motor development measures.
  
  
  

“These Phase 1 results represent an important milestone for a patient population with tragically limited treatment options,” said Dr. Remo Moomiaie-Qajar, Founder and Chief Executive Officer of Axiom Biosciences. “The fact that all patients survived for a year and showed promising long-term improvement reveals the platform’s regenerative potential. These results give us a solid launchpad for our next phase of clinical development.”

IVH and HIE are among the leading causes of mortality and long-term neurological disability in newborns, frequently resulting in cerebral palsy, developmental impairment, and lifelong medical complications. There are currently no approved regenerative therapies that directly address the underlying neurological injury associated with these conditions.

In 2023, the company received Rare Pediatric Disease Designations from the U.S. Food and Drug Administration for both IVH and HIE. The FDA’s Rare Pediatric Disease Priority Review Voucher program is currently authorized through September 30, 2029; if a product is approved while the program remains in effect, these designations may make the company eligible to receive Priority Review Vouchers.

Building on these results, Axiom is evaluating the expansion of the platform into adult ischemic stroke. Because HIE and ischemic stroke share common mechanisms involving ischemic tissue injury and subsequent inflammatory response, the company believes the platform may have broader applications across neurological disorders. Ischemic stroke affects approximately 700,000 adults annually in the United States and remains a leading cause of death and long-term disability worldwide.

“With time being of the essence in stroke care, patients lose 1.9 million neurons every single minute,” added Dr. Moomiaie-Qajar. “Our platform aims to offer a first-of-its-kind regenerative approach that goes beyond mitigating damage to potentially restoring function.”

Axiom plans to advance the neonatal program into Phase 2 development and to evaluate expansion into adult ischemic stroke and additional neurological indications.

About Axiom Biosciences
Axiom Biosciences (formerly Cytonus Therapeutics) is a clinical-stage biotechnology company developing next-generation regenerative therapies and targeted biologics. Through advanced cellular engineering and translational science, the company is building a pipeline of therapies designed to address some of the most challenging diseases across neurology, inflammation, and regenerative medicine. Founded in 2018 and headquartered in San Diego, California, Axiom is advancing programs from rare pediatric conditions toward larger indications, including adult ischemic stroke. To learn more, please visit www.axiombio.co.

About Medinno Inc.
Medinno Inc. is a South Korea-based biotechnology company specializing in stem cell technologies and regenerative medicine platforms, with a focus on clinical translation and global partnerships.

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