CAMBRIDGE, Mass.– Aurion Biotech has dosed the first patients in its U.S. Phase 3 ASTRA trial evaluating AURN001, an investigational cell therapy for corneal edema caused by corneal endothelial dysfunction.
The pivotal study will assess the efficacy and safety of AURN001 compared to placebo and marks a key step toward potential regulatory approval. The therapy is being developed as a single-administration treatment designed to restore corneal clarity and improve vision.
“Dosing the first ASTRA study participants marks an important milestone for patients, physicians, and Aurion as we work together to advance new solutions for vision loss from corneal endothelial disease,” said Edward J. Holland, M.D., Chief Medical Officer of Aurion Biotech. “While modern endothelial keratoplasty procedures have transformed the treatment of the disease, there still is a need to reduce complications and improve our patient’s experience. AURN001 represents the potential next evolution by moving from donor tissue-based graft surgery toward a cultured cell therapy designed to restore corneal clarity and expand access to care for patients worldwide.”
AURN001 is a first-in-class investigational therapy combining human corneal endothelial cells, known as neltependocel, with a rho-kinase inhibitor, Y-27632. The treatment is administered through an intracameral injection and is intended to regenerate corneal tissue, potentially reducing complications associated with traditional transplant procedures and enabling faster recovery.
“Reaching this milestone reflects the dedication of our investigative sites, whose rigor and deep commitment to patients with corneal endothelial dystrophy have made this possible,” said Eris Jordan, O.D., Chief Development Officer of Aurion Biotech. “Their enthusiasm for this program underscores a shared belief that regenerative cell therapy has the potential to fundamentally transform how we treat this disease.”
The ASTRA trial is being conducted at 12 sites across the United States. Patients will be randomized to receive AURN001 plus Y-27632, with the primary endpoint measuring the proportion of participants achieving at least a 15-letter improvement in best corrected visual acuity at six months. Secondary endpoints include changes in corneal thickness and visual acuity over the same period.
Aurion Biotech Chief Executive Officer and Chief Scientific Officer Arnaud Lacoste, Ph.D., MBA, said the study is a critical step toward bringing the therapy to patients globally.
“This pivotal study is the foundation for our U.S. BLA submission and a critical step in our mission to address a very large global unmet patient need,” Lacoste said. “By using donor corneas to create scalable, and transportable cell therapy, we are overcoming the limitations of traditional surgery. Following our success in Japan, we are now focused on providing a global solution for blindness.”
Aurion is advancing corneal endothelial cell therapy as a regenerative approach to replace damaged cells that do not naturally regenerate. The therapy uses human cells derived from donated corneas, without synthetic materials or genetic modification, with the goal of restoring normal corneal function and vision.
