Innovative Delivery Method Overcomes the Blood-Brain Barrier

Axiom Biosciences and Medinno have jointly developed HIEstem™, a stem cell therapy for neonatal brain injury, which has demonstrated remarkable safety in a Phase 1 clinical trial. The trial involved nine newborns suffering from intraventricular hemorrhage (IVH) and hypoxic-ischemic encephalopathy (HIE), and it achieved a 0% mortality rate over a two-year period, confirming its high safety profile. Unlike existing therapies that struggle to cross the blood-brain barrier (BBB), HIEstem™ employs a direct intraventricular administration method, maximizing drug delivery efficiency to the brain. This is achieved through an ingenious design that utilizes the cerebrospinal fluid flow to establish a drug delivery pathway.

Environment Control Technology Mimics Natural Cells

The persistent challenges of cell survival and functionality, which have plagued conventional stem cell therapies, have been addressed through Axiom's proprietary environment control technology. HIEstem™ does not involve artificial genetic manipulation; instead, it utilizes Wharton's Jelly-derived stem cells, cultured under conditions similar to the disease environment using a thrombin pretreatment technique. These stem cells, trained in an environment that mimics the disease state, exhibit enhanced survival rates and maximized release of therapeutic factors upon reaching the damaged brain tissue. Consequently, not only were there no serious adverse events observed over the two-year period, but the treated newborns also showed significant improvement in brain function, allowing them to discontinue the use of anticonvulsant medications.

Addressing Unmet Needs and Receiving Orphan Drug Designation

Currently, IVH and HIE pose life-threatening risks to infants under one year of age, with a natural mortality rate of approximately 46%. Furthermore, even among survivors, about half experience lifelong sequelae such as cerebral palsy, highlighting the urgent need for effective therapies. The HIEstem™ clinical trial showed no signs of cerebral palsy and no delayed development of gross motor skills, effectively addressing the unmet medical need. Recognizing its value, HIEstem™ has received Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for both conditions.

Scalability to the Adult Brain Disease Market

Based on the success of this clinical trial, Axiom Biosciences is rapidly preparing for a Phase 2b trial and plans to expand the indications to include adult ischemic stroke. The underlying mechanisms of HIE in newborns, which involve hypoxia and reduced blood flow, are very similar to those of adult stroke, allowing for cross-validation. This demonstrates a venture capital-driven growth strategy, aiming to rapidly scale up from the rare pediatric disease market to the multi-billion dollar adult brain disease market using the platform technology acquired in the rare pediatric disease field. Axiom's ability to overcome the regulatory hurdles faced by leading companies such as Mesoblast, which experienced delays in regulatory approvals, is garnering significant attention.