AURN001 Achieves Dose-Dependent Response at 6 Months, With Zaina Al-Mohtaseb, MD

Individuals with corneal edema achieved a dose-dependent response at 6 months in best-corrected visual acuity (BCVA) with a single dose of varied concentrations of human corneal endothelial cell (CEC) therapy in combination with Y-27632 Rho-Kinase Inhibitor (AURN001) in the ongoing Phase 1/2 CLARA trial.1

Presented at the 2025 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting, patient-reported outcomes in the trial indicated benefit for quality of life, consistent with the dose-dependent response, and a favorable safety profile, remaining well-tolerated.

“I think patients are going to love this therapy, and even in the study, it showed a dose-correlated patient-reported outcome in the high-dose arm in comparison,” presenting investigator Zaina Al-Mohtaseb, MD, an ophthalmologist at Whitsett Vision Group and chair of the ASCRS Young Eye Surgeon committee, told HCPLive. “It’s about improving, not just the vision, but what the patient feels in the process, and I think the future is pretty amazing for cornea.”

AURN001 is a combination cell therapy product candidate comprised of allogeneic human CECs (neltependocel) and a rho kinase inhibitor (Y-27632), intended to be administered as a one-time procedure to the anterior chamber of the eye.

The CLARA trial assessed the efficacy and safety of a single dose of varied concentrations of human CECs combined with Y-27632 Rho-Kinase Inhibitor, compared with CECs alone, and Y-27632 alone, for a population with corneal edema. The primary endpoint was the proportion of patients achieving a ≥15-letter improvement from baseline in BCVA at six months.

Secondary endpoints were defined as the change from baseline in BCVA and central corneal thickness (CCT) at six months, with a quality of life endpoint measured by the Visual Function Questionnaire-25 (VFQ-25). Safety was measured by treatment-emergent adverse events, graft rejection, and rescue rates.

With 5 treatment arms, a total of 97 participants with corneal edema secondary to corneal endothelial dysfunction were enrolled across 20 sites in North America. Analysis of CLARA identified a dose-dependent response for the primary and secondary endpoints and the proportion of participants requiring rescue at six months.

A statistically significant improvement in the proportion of responding patients who achieved ≥15 letters of improvement in BCVA, compared with Y-27632 alone, was recorded in the high-dose arm (50% vs 14.3%; P =.02).2

In the VFQ-25 questionnaire, patients reported dose-dependent quality of life improvement. Safety analysis found no ocular serious adverse events, treatment-emergent adverse events (TEAEs) linked to discontinuation, or graft rejections, across all 5 trial arms.

With these data marking the six-month interim data analysis, the trial is expected to be completed in the first half of 2025 for the full 12-month outcomes.

“You want to make sure that you have, at least, the same statistically significant improvement, if not more, and then the same safety data,” Al-Mohtaseb told HCPLive. “And again, seeing that dose response continues out to 12 months is going to be important, because you obviously want this [response] to last.”

Al-Mohtaseb reports relevant disclosures with Alcon, AbbVie, Bausch & Lomb, Johnson & Johnson, and others.

References

1. Al-Mohtaseb Z, Lee WB, Hester CC, Pouly SM, et al. 6-Mo Data Analysis of Clara: A Phase 1/2 Clinical Trial of Human Corneal Endothelial Cell Therapy for Corneal Edema Secondary to CED. Paper presented at the 2025 American Society of Cataract and Refractive Surgery (ASCRS) Annual Meeting. April 25-28, 2025. Los Angeles, CA.

2. McGarry J. Aurion biotech announces positive topline data for phase 1/2 clinical trial of AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction. Aurion Biotech. December 18, 2024. Accessed April 26, 2025. https://aurionbiotech.com/aurion-biotech-announces-positive-data-for-phase_1-2_trial/.