Aurion Announces Positive 12-Month Results from Phase 1/2 CLARA Trial of AURN001 Corneal Endothelial Cell Therapy
Aurion Biotech announced positive 12-month results from its phase 1/2 CLARA clinical trial. The study 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) annual meeting in Orlando, Florida.
In this double-masked, parallel-arm, dose-ranging phase 1/2 trial (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—compared to 0% in the Y-27632 control group—achieved a ≥15-letter gain in best corrected visual acuity (BCVA) (P<0.0001). In the high-dose cohort, the mean change in BCVA from baseline was 12.5 letters, while central corneal thickness (CCT) decreased by 23.2 µm. Responders’ visual acuity improved from 60 to 81 letters (Snellen equivalent 20/60 to 20/25). A dose-dependent improvement was also observed in patient-reported outcomes (VFQ-25), with high-dose participants reporting the most significant 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 PMDA approval 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.”
“It is impressive that this trial was able to successfully achieve all primary, secondary, and exploratory endpoints,” added W. Barry Lee, MD, FACS, cornea specialist at Eye Consultants of Atlanta and President of the Cornea Society, who presented the data at AAO. “AURN001 was safe and well-tolerated across all treatment groups, with no cases of graft rejection and no treatment-related serious adverse events. This provides clinicians with high confidence in the safety profile of CEC therapy as the program advances.”
In the United States, Aurion Biotech has been granted both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation (BTD) by the FDA for AURN001.