Aurion Biotech Reports Positive Phase 1/2 Results for AURN001 Cell Therapy for Corneal Edema

Aurion Biotech, Inc., operating out of Seattle, Cambridge, and Tokyo, has announced promising results from its Phase 1/2 clinical study, known as CLARA, which investigates a novel therapy for corneal edema stemming from corneal endothelial dysfunction. This innovative treatment, named AURN001, combines allogeneic human corneal endothelial cells with a rho kinase inhibitor, Y-27632. The aim is to administer this therapy to the eye's anterior chamber in a single procedure.

The CLARA trial is a robustly designed, multi-center, randomized, double-masked, parallel-arm study conducted across various sites in the United States and Canada. It evaluates the safety, tolerability, and effectiveness of AURN001 in treating the specified eye condition. A total of 97 patients were randomized into five different treatment groups. These groups received varying doses of AURN001, with the highest dose containing 1.0 × 10⁶ neltependocel cells and 100 μM of Y-27632.

Participants in the trial had an average age of 71.4 years, and 55% were female. At the onset of the study, the average best-corrected visual acuity (BCVA) score, measured using the ETDRS test, was 53.5 letters, equivalent to a 20/85 Snellen score. The central corneal thickness (CCT) was recorded at 676.6 microns.

The primary endpoint for this trial was to measure the percentage of patients who experienced more than a 15-letter improvement in BCVA after six months. Secondary endpoints included changes in BCVA and CCT after six months, along with safety and tolerability assessments. The data revealed a dose-dependent response in the three AURN001 arms, with the high-dose group showing significant improvement, with 50% of patients responding positively compared to just 14.3% in the Y-27632-only group.

A closer examination of secondary endpoints showed that the high-dose AURN001 group had a significant improvement in BCVA at the six-month mark compared to the Y-27632-only group. Similarly, these patients exhibited significant changes in CCT, indicating potential benefits in vision restoration. Furthermore, an enhancement in the quality of life, assessed via the Visual Function Questionnaire, was most pronounced in the high-dose group.

Safety profiles across all five treatment arms were generally favorable, with no significant dose relationship observed regarding adverse events. Ocular side effects reported included ocular hypertension, conjunctival hemorrhage, eye pain, and cystoid macular edema. The most common non-ocular adverse event was COVID-19 infection.

Aurion Biotech sees these results as a significant milestone. Dr. Michael Goldstein, the company's president and chief medical officer, expressed excitement over the positive outcomes, particularly in the high-dose group. He emphasized the promising safety profile of AURN001. CEO Greg Kunst echoed this sentiment, highlighting the study as an essential step in developing this cell therapy aimed at vision restoration.

This study's findings come after Aurion Biotech received Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the U.S. FDA in June 2024. Furthermore, Aurion has launched its cell therapy in Japan under the brand name Vyznova.

Corneal endothelial dysfunction poses a significant threat to vision worldwide, as the cells involved do not regenerate naturally. Current solutions like corneal transplants are complex and restricted by donor availability. Aurion Biotech's approach offers a less invasive and more accessible treatment alternative, addressing a critical unmet need for patients with this condition. The company's commitment to advancing regenerative therapies continues to drive its mission to restore vision to millions globally.