Axiom Biosciences has reported results from a phase 1 clinical trial evaluating an umbilical cord-derived mesenchymal stem cell (MSC) therapy in newborns with intraventricular hemorrhage (IVH) and hypoxic-ischemic encephalopathy (HIE). According to the company, none of the treated infants died during the two-year follow-up period, and the study also recorded improvements in developmental outcomes.
The trial enrolled nine newborns, including five with severe IVH and four with HIE. IVH refers to bleeding inside the ventricles of the brain, while HIE occurs when a baby’s brain does not receive enough oxygen during or shortly after birth. The study assessed the therapy’s safety, tolerability, and preliminary efficacy.
Axiom said the therapy was co-developed with South Korea-based Medinno. Across the doses evaluated, researchers reported no treatment-related serious adverse events. The infants also did not require anti-epileptic medication. In addition, the company stated that no cases of cerebral palsy were observed among the treated patients. According to the information provided, cerebral palsy is seen in about half of patients with these conditions.
The company also reported improved neurodevelopmental delays and the absence of gross motor developmental delays among participants. Axiom noted that newborns with IVH and HIE typically face a mortality rate of nearly 50% within the first year of life.
The investigational therapy is derived from Wharton jelly, a gelatinous substance in the umbilical cord that is rich in MSCs. According to Axiom Chief Executive Officer Remo Moomiaie-Qajar, the company’s approach differs from other stem cell therapies because the cells are not engineered or edited. Instead, they are conditioned by being grown in an environment that mimics the disease state.
Moomiaie-Qajar said this process allows the cells to begin producing factors intended to repair injury associated with IVH and HIE. To address the challenge of crossing the blood-brain barrier, the stem cells were delivered directly into the ventricles of the newborns. According to the company, this approach bypasses the barrier by using the brain’s natural fluids to distribute the therapy.
“I think we solved two major potholes that have hurt a lot of other stem cell companies in the past—the approach to how you control for the product, how you control where it goes and what it produces,” Moomiaie-Qajar said in an interview.
He also said that delivering the therapy into the ventricles adds a high level of regulatory scrutiny and introduces a degree of risk that would not otherwise exist with regular stem cells.
Axiom and Medinno both focus on stem cell and regenerative therapies. Axiom, which is headquartered in San Diego, has received rare pediatric disease designations from the FDA for the Wharton jelly MSC therapy in both IVH and HIE.
Following the phase 1 findings, the company plans to advance the program into phase 2b testing. Axiom also intends to expand studies of the therapy into adult ischemic stroke. The company noted that HIE is characterized by a lack of oxygen and blood flow to the brain, a mechanism similar to that seen in adult ischemic stroke.
Axiom has announced promising Phase 1 clinical trial results for its investigational stem cell therapy designed to treat newborn brain injuries. The early-stage study primarily evaluated safety while also collecting preliminary evidence of potential therapeutic benefits. These findings represent an important milestone for Axiom as it advances innovative regenerative treatments for neonatal neurological conditions.
Axiom Announces Encouraging Phase 1 Clinical Results
The Phase 1 study showed that Axiom’s stem cell therapy was generally well tolerated among participating newborn patients. Researchers reported no major safety concerns directly related to the treatment, supporting the continued evaluation of the therapy in larger clinical studies.
Establishing a strong safety profile is a critical first step for Axiom as it progresses through the clinical development pathway.
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
- Editorial Team
