Aurion Biotech, a clinical-stage regenerative medicine company with a mission to restore vision to millions of patients,  announced positive 12 month results from the company’s Phase 1/2 CLARA trial, which evaluated the safety, efficacy and tolerability of AURN001 (neltependocel [human corneal endothelial cells] in combination with Y-27632 rho-kinase inhibitor) in patients with corneal edema secondary to corneal endothelial dysfunction. The findings were presented at Cornea Day during the American Academy of Ophthalmology (AAO) meeting in Orlando, FL.

In this Phase 1/2 double-masked, parallel-arm, dose-ranging study (NCT06041256), patients treated with a single dose of AURN001 corneal endothelial cell (CEC) therapy demonstrated a clear dose-dependent response, with the greatest efficacy observed in the high-dose group. At 12 months, 65% of subjects in the high-dose AURN001 group vs. 0% of the Y-27632 group achieved a ≥15-letter best corrected visual acuity (BCVA) gain (p<0.0001). Also, in the high-dose group, the mean change in BCVA from baseline was 12.5 letters, mean reduction in central corneal thickness (CCT) was 23.2 µm, and responders’ visual acuity improved from 60 to 81 letters (20/60 to 20/25, Snellen equivalent). A dose-dependent improvement was also demonstrated in patient-reported outcomes (VFQ-25), with subjects in the high-dose group reporting the most pronounced gains in quality-of-life measures.

"The results of our Phase 1/2 CLARA trial add to the large body of data on AURN001 and confirm, across different geographies and patient populations, the success of the corneal endothelial cell therapy that Aurion is already commercializing in Japan after approval from PMDA in 2024," said Edward J. Holland, MD, Chief Medical Officer, Aurion Biotech. “As we prepare to launch a U.S. pivotal Phase 3 trial in the first half of 2026, we are incredibly optimistic that our cell therapy will one day restore vision to millions of people with corneal endothelial blindness.”

“It is impressive that this trial was able to successfully achieve all primary, secondary, and exploratory endpoints,” said W. Barry Lee, MD, FACS, cornea specialist at Eye Consultants of Atlanta and President of the Cornea Society, who presented the data at AAO. “In particular, the data showed that AURN001 was safe and well-tolerated across all treatment groups, with no cases of graft rejections and no treatment-related serious adverse events. This will provide clinicians with a high level of confidence in the safety of CEC therapy as the clinical trial program continues.”

In the U.S., Aurion Biotech has obtained both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation (BTD) for AURN001 for its potential to treat corneal endothelial disease, a group of disorders where a critical layer of cells responsible for pumping fluid out of the cornea is damaged. This leads to corneal swelling and vision loss. Unlike the current standard of care, which is limited by donor shortages, AURN001 utilizes cultured human CECs. Subject to manufacturing specifications and quality controls, each qualified donor line may yield up to 1,000 therapeutic doses, transforming a single tissue donation into what could be the world’s first mass-scale cell therapy with the ability to treat millions of patients.