Aurion Biotech Reports Positive 12-Month Results from Phase 1/2 CLARA Trial of AURN001

Aurion Biotech has announced encouraging 12-month clinical results from its ongoing Phase 1/2 CLARA trial, which is evaluating AURN001, a corneal endothelial cell therapy for patients with corneal edema secondary to endothelial dysfunction. The findings were presented during Cornea Day at the American Academy of Ophthalmology (AAO) annual meeting in Orlando, Florida.

AURN001 Demonstrates Strong Efficacy and Safety in Treating Corneal Edema

The CLARA study (NCT06041256) is a double-masked, parallel-arm, dose-ranging Phase 1/2 trial assessing the safety, efficacy, and tolerability of AURN001 (neltependocel), a combination of human corneal endothelial cells and Y-27632, a rho-kinase inhibitor.

A clear dose-dependent efficacy was observed in the 12-month analysis. In the high-dose group, 65% of patients achieved a ≥15-letter gain in best corrected visual acuity (BCVA), compared to 0% in the control group treated only with Y-27632 (P<0.0001). The mean BCVA improvement in the high-dose cohort was 12.5 letters, and central corneal thickness (CCT) was reduced by 23.2 µm. Among responders, visual acuity improved from 60 to 81 letters, equivalent to a change from 20/60 to 20/25 (Snellen).

The therapy also produced a dose-dependent improvement in quality-of-life scores, with participants in the high-dose group reporting the most notable gains on the VFQ-25 questionnaire.

Expert Commentary and Next Steps

“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. “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.”

The trial’s success was also highlighted by W. Barry Lee, MD, FACS, cornea specialist at Eye Consultants of Atlanta and President of the Cornea Society, who presented the data at AAO. “It is impressive that this trial met all primary, secondary, and exploratory endpoints,” he stated. “AURN001 was safe and well-tolerated across all dose groups, with no 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.”

Regulatory Designations Support U.S. Development

In the United States, AURN001 has been granted both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation (BTD) by the FDA, recognizing its potential to address significant unmet needs in treating corneal endothelial diseases.