Phase 1/2 CLARA study shows AURN001 improves BCVA compared with standard of care for corneal endothelial dysfunction
Brandon Ayres, MD, of Wills Eye Hospital, spoke with Ophthalmology 360 about the phase 1/2 data assessing investigational treatment AURN001. The 1-year results showed positive changes in best-corrected vision.
Brandon Ayres, MD:
Hi, I’m Brandon Ayres, MD, from the cornea service at Wills Eye Hospital and Ophthalmic Partners of Pennsylvania. At ASCRS 2026, I had the distinct honor of presenting Aurion Biotech’s phase 1/2 clinical trial on human corneal endothelial cell therapy. This was a first-in-class, multicenter, randomized, masked trial looking at cultured human corneal endothelial cells combined with a ROCK inhibitor as compared to cells alone and ROCK inhibitor alone. There were 3 different groups that got cultured endothelial cells with ROCK inhibitor at different concentrations, as this was a dose-ranging study: 250,000 cells/0.1 cc, 500,000 cells/0.1 cc, and 1,000,000 cells/0.1 cc. Anybody who entered the study had to have a vision of 20/50 or worse, PC IOLs in the capsular bag with corneal stromal edemas.
These were all patients who were looking to do an endothelial keratoplasty. The purpose of the study was to help show that cell injection therapy can be as good or even better than the current standard of care, which is likely endothelial keratoplasty or DMEK. The primary endpoint of the study was to look at the proportion of patients in each group who achieved a 15-letter or greater improvement in vision on the ETDRS chart at 6 months. Secondary endpoints included improvement in best-corrected vision and looking at the central corneal thickness at all time points of the study.
The initial abstract was the 6-month time point, but we had enough data to show the 1-year endpoints. This was very interesting, 65% of patients in the high-dose group achieved a 15-letter or greater improvement as compared to ROCK inhibitor alone. This same group of patients also had the best-corrected vision at 1 year. The visual improvement in that high-dose group went from approximately 20/60 at entry to 20/25 at exit. Just to give an example of a shining star patient, we had a subject who entered at 20/50, by 1 month was 20/15. He maintained that through the 12 months of the study. He also had corresponding reduction in central corneal thickness and improvement in endothelial cell density.
You always have to look at adverse events, and we did look at adverse events in this study. We really saw no serious adverse events at all, and notably, no patients had endothelial rejection. The most common adverse event was going to be elevated intraocular pressure, presumably due to steroids, and this resolved in all of the patients. Conjunctival hemorrhages were seen in 6.2% of patients, epithelial defects, CME, ocular pain, corneal abrasions were seen in about 3% of patients.
The results of the CLARA phase 1/2 study confirmed that the high dose is the best of the doses at improving corneal edema. This is the dose that will be used in the phase 3 study, which is enrolling patients right now. If the phase 3 study replicates what we saw in CLARA and replicates what we see in Japan where the procedure is approved and known as Vyznova, I’m excited to think about how this is going to change the world and how we treat endothelial disease.
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