Aurion Biotech’s AURN001 achieves all endpoints at 12 months, paving way for phase III trial
Aurion Biotech has reported positive 12-month results from its phase I/II CLARA trial evaluating AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction, showing that AURN001 met all primary, secondary, and exploratory endpoints in patients with corneal endothelial disease.
The findings, presented during Cornea Day at the American Academy of Ophthalmology (AAO) meeting in Orlando, Florida (October 17-20, 2025) support advancing AURN001 into a pivotal phase III trial planned for the first half of 2026.
In the double-masked, dose-ranging CLARA trial (NCT06041256), patients received a single dose of AURN001 – a therapy consisting of human corneal endothelial cells (CECs) combined with Y-27632 rho-kinase inhibitor. At 12 months, 65% of subjects in the high-dose AURN001 group gained ≥15 letters of best-corrected visual acuity (BCVA), compared to 0% in the control group. The high-dose cohort showed a 12.5-letter mean BCVA improvement, a 23.2-µm reduction in central corneal thickness (CCT), and improvement from 20/60 to 20/25 (Snellen equivalent) among responders.
“It is impressive that this trial was able to successfully achieve all primary, secondary, and exploratory endpoints,” said W. Barry Lee, President of the Cornea Society who presented the data at AAO. “In particular, 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.”
Aurion’s Chief Medical Officer, Edward J. Holland, added: "The results of our phase I/II 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 the Pharmaceuticals and Medical Devices Agency (PMDA) in 2024… We are incredibly optimistic that our cell therapy will one day restore vision to millions of people with corneal endothelial blindness.
The company holds both Breakthrough Therapy Designation (BDT) and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA for AURN001 — designed to address a global shortage of donor corneal tissue. Each qualified donor line can produce up to 1,000 therapeutic doses, positioning the therapy as a potential mass-scale regenerative solution for corneal endothelial blindness.
Aurion Biotech has reported positive 12-month results from its phase I/II CLARA trial evaluating AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction, showing that AURN001 met all primary, secondary, and exploratory endpoints in patients with corneal endothelial disease.
The findings, presented during Cornea Day at the American Academy of Ophthalmology (AAO) meeting in Orlando, Florida (October 17-20, 2025) support advancing AURN001 into a pivotal phase III trial planned for the first half of 2026.
In the double-masked, dose-ranging CLARA trial (NCT06041256), patients received a single dose of AURN001 – a therapy consisting of human corneal endothelial cells (CECs) combined with Y-27632 rho-kinase inhibitor. At 12 months, 65% of subjects in the high-dose AURN001 group gained ≥15 letters of best-corrected visual acuity (BCVA), compared to 0% in the control group. The high-dose cohort showed a 12.5-letter mean BCVA improvement, a 23.2-µm reduction in central corneal thickness (CCT), and improvement from 20/60 to 20/25 (Snellen equivalent) among responders.
“It is impressive that this trial was able to successfully achieve all primary, secondary, and exploratory endpoints,” said W. Barry Lee, President of the Cornea Society who presented the data at AAO. “In particular, 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.”
Aurion’s Chief Medical Officer, Edward J. Holland, added: "The results of our phase I/II 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 the Pharmaceuticals and Medical Devices Agency (PMDA) in 2024… We are incredibly optimistic that our cell therapy will one day restore vision to millions of people with corneal endothelial blindness.
The company holds both Breakthrough Therapy Designation (BDT) and Regenerative Medicine Advanced Therapy (RMAT) designations from the FDA for AURN001 — designed to address a global shortage of donor corneal tissue. Each qualified donor line can produce up to 1,000 therapeutic doses, positioning the therapy as a potential mass-scale regenerative solution for corneal endothelial blindness.
