Aurion Biotech releases positive 12-month results from phase 1/2 CLARA trial

Aurion Biotech has released positive 12-month results from its phase 1/2 CLARA trial evaluating the safety, efficacy, and tolerability of AURN001 in patients with corneal edema secondary to corneal endothelial dysfunction.

In the CLARA trial, a double-masked, parallel-arm, dose-ranging study, 97 patients were enrolled across 20 sites in the US and Canada. Patients were randomized to AURN001 high, medium, or low dose; corneal endothelial cell (CEC) alone; or Y-27632 alone. The primary endpoint was the proportion of patients with a ≥15-letter improvement from baseline in BCVA at 6 months, while secondary endpoints were change from baseline in BCVA and CCT.

AURN001 is a combination cell therapy product candidate comprised of allogeneic human corneal endothelial cells (neltependocel) and a rho kinase inhibitor (Y-27632).

According to the company, patients treated with a single dose of AURN001 CEC therapy demonstrated a “clear dose-dependent response, with the greatest efficacy observed in the high-dose group.”

Additionally, at 12 months 65% of patients in the high-dose AURN001 group achieved a ≥15-letter best corrected visual acuity (BCVA) gain (p<0.0001), while 0% achieved this in the Y-27632 group. In the high-dose group the mean change in BCVA from baseline was 12.5 letters, the mean reduction in central corneal thickness (CCT) was 23.2 µm, and responders’ visual acuity improved from 60 to 81 letters (20/60 to 20/25, Snellen equivalent).

Edward J Holland, MD, Chief Medical Officer of Aurion Biotech, and W. Barry Lee, MD, FACS, cornea specialist at Eye Consultants of Atlanta and President of the Cornea Society, commented on the results in a press release from the company.

"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 Holland.

While Lee added, “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. This will provide clinicians with a high level of confidence in the safety of CEC therapy as the clinical trial program continues.”

Aurion has received both Regenerative Medicine Advanced Therapy (RMAT) and Breakthrough Therapy Designation (BTD) for AURN001 in the US.

Earlier in 2025, Alcon announced it has acquired a majority interest in Aurion Biotech. The company plans to launch a pivotal phase 3 trial in the US in the first half of 2026.

References:

1. Aurion Biotech achieves all primary, secondary, and exploratory endpoints in AURN001 phase 1/2 CLARA trial at 12 months. Published October 18, 2025. Accessed October 20, 2025. https://www.businesswire.com/news/home/20251018009011/en/Aurion-Biotech-Achieves-All-Primary-Secondary-and-Exploratory-Endpoints-in-AURN001-Phase-12-CLARA-Trial-at-12-Months

2. Alcon acquires majority interest in Aurion Biotech. Published March 27, 2025. Accessed October 20, 2025. https://www.ophthalmologytimes.com/view/alcon-acquires-majority-interest-in-aurion-biotech