When Axiom Biosciences' stem cell therapy demonstrated a 0% mortality rate over two years in potentially fatal newborn neurological diseases this week, the biotech's leadership saw a broader validation for their approach to targeting the brain.
The phase 1 clinical trial dosed nine newborns with the umbilical cord-derived mesenchymal stem cell (MSC) therapy and observed improved neurodevelopmental delays and no gross motor developmental delays, Axiom reported on Tuesday.
The therapy was co-developed by South Korea-based Medinno to treat intraventricular hemorrhage (IVH), a term for bleeding inside the ventricles of the brain, as well as hypoxic-ischemic encephalopathy (HIE), where the baby's brain doesn't receive enough oxygen during or shortly after birth. The mortality rate for newborns with these diseases is nearly 50% in the first year of life, according to the release.
The study evaluated the safety, tolerability and preliminary efficacy of the therapy in five newborns with severe IVH and four with HIE. Across the doses evaluated, there were no treatment-related serious adverse events and no need for anti-epileptic medication. Additionally, Axiom didn't observe cerebral palsy, which is seen in half of patients with these diseases.
Although MSC therapies have been researched for decades, hurdles still exist for these treatments. Mesoblast’s Ryoncil, an MSC-based therapy to treat steroid-refractory acute graft versus host disease, only clawed its way to FDA approval in 2024 after two rejections.
“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,” Axiom CEO Remo Moomiaie-Qajar, M.D., told Fierce in an interview.
Axiom’s investigational therapy is derived from Wharton jelly, which is an MSC-rich gelatinous substance in the umbilical cord. The jelly happens to house “the youngest cells in the human body,” Moomiaie-Qajar explained.
Unlike other stem cell therapies, Axiom’s Wharton jelly MSCs are not engineered or edited. Instead, they are “conditioned” or grown in an environment that mimics the disease state. This allows the cells to begin producing certain factors to repair injury, in this case IVH and HIE.
To overcome the difficulty of getting the therapy to cross the blood-brain barrier, the stem cells were delivered into the ventricles of the newborns. This technique allows for bypassing of the barrier by using the natural fluids of the brain to deliver the therapy.
The approach “adds a very high level of scrutiny from a regulatory perspective, but also introduces a level of risk that otherwise wouldn't be there with regular stem cells,” said Moomiaie-Qajar.
Both Axiom and Medinno specialize in developing stem cell and regenerative therapies. Headquartered in San Diego, Axiom has already received rare pediatric disease designations from the FDA for the Wharton jelly MSC therapy for both IVH and HIE.
Due to the positive clinical results, Axiom plans to move into phase 2b trials while also expanding the studies to test the therapy in adult ischemic stroke. The so-called HIE insult is characterized by a lack of oxygen and blood flow to the brain, a similar mechanism to that seen in adult ischemic stroke.
“That to me is really exciting, because it's a demonstration of how you can start as a small company in rare diseases, and very rapidly scale to one of the biggest socioeconomic problems in healthcare today,” Moomiaie-Qajar said.