Aurion Biotech announced positive 12-month results from its Phase 1/2 CLARA trial evaluating AURN001 (neltependocel), a regenerative cell therapy for corneal endothelial disease. The double-masked, parallel-arm, dose-ranging study achieved all primary, secondary, and exploratory endpoints, with data presented at the American Academy of Ophthalmology meeting in Orlando.
Dose-Dependent Efficacy Demonstrated
The trial enrolled 97 patients across 20 sites in the U.S. and Canada, randomizing participants to receive AURN001 at high, medium, or low doses, corneal endothelial cells alone, or Y-27632 rho-kinase inhibitor alone. Patients treated with a single dose of AURN001 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 achieved a ≥15-letter best corrected visual acuity (BCVA) gain compared to 0% in the Y-27632 control group (p<0.0001). The high-dose group showed a mean change in BCVA from baseline of 12.5 letters and a mean reduction in central corneal thickness of 23.2 μm. Notably, responders' visual acuity improved from 60 to 81 letters, equivalent to improving from 20/60 to 20/25 on the Snellen chart.
Safety Profile and Patient Outcomes
The therapy demonstrated an excellent safety profile across all treatment groups. "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," said W. Barry Lee, MD, FACS, cornea specialist at Eye Consultants of Atlanta and President of the Cornea Society, who presented the data.
Patient-reported outcomes also showed dose-dependent improvement, with subjects in the high-dose group reporting the most pronounced gains in quality-of-life measures on the VFQ-25 questionnaire.
Regulatory Status and Commercial Potential
AURN001 has received both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation from the FDA for treating corneal endothelial disease. The therapy addresses a critical unmet need in treating disorders where corneal endothelial cells responsible for pumping fluid out of the cornea are damaged, leading to corneal swelling and vision loss.
Unlike current standard of care limited by donor shortages, AURN001 utilizes cultured human corneal endothelial cells. Each qualified donor line may yield up to 1,000 therapeutic doses, potentially transforming a single tissue donation into what could be the world's first mass-scale cell therapy capable of treating millions of patients.
Path Forward
"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 at Aurion Biotech.
The company plans to launch a U.S. pivotal Phase 3 trial in the first half of 2026. Aurion Biotech launched Vyznova, the first cell therapy for corneal endothelial disease commercially available in Japan in 2024, and Alcon acquired majority ownership of the company in 2025.
