Aurion’s Cell Therapy Wins RMAT, Breakthrough Therapy Designation for Corneal Edema


The CLARA trial has also completed enrollment of participants with corneal edema secondary to corneal endothelial disease.

The FDA has granted Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy (RMAT) Designation to Aurion Biotech’s AURN001 cell therapy for the potential treatment of corneal edema secondary to corneal endothelial disease.1

“Aurion Biotech is honored to receive the Breakthrough Therapy Designation and RMAT Designation for AURN001,” Sterling Chung, Vice President of Regulatory Affairs and Quality, Aurion Biotech, said in a statement.1 “These designations underscore the importance of developing a potential solution for millions of patients around the world who suffer from corneal endothelial diseases. We look forward to working closely with the FDA to expedite the development of our cell therapy.”

AURN001 is an allogeneic cell therapy) comprised of neltependocel (allogeneic human corneal endothelial cells) and Y-27632 (an inhibitor of Rho-associated, coiled-coil containing protein kinase [ROCK]). The therapy is administered via a 1-time, intracameral injection.

“The BTD and RMAT designations are important milestones for people living with corneal endothelial disease who need better, less burdensome, more accessible treatment options,” Greg Kunst, chief executive officer,Aurion added.1 “I thank the Aurion team for their hard work and dedication, and I’m grateful for the essential contributions of clinical trial patients and their ophthalmologists – all of whom have helped to make today’s news a reality.”

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Corneal edema secondary to endothelial dysfunction affects millions of people. If left untreated, corneal endothelial cell loss can cause corneal edema and loss of vision. While current standard of care corneal transplants are effective, disadvantages include limited donor organ supply and difficult post-operative recovery.

AURN-001 is currently being evaluated in the phase 1/2 CLARA clinical trial (NCT06041256). Aurion announced in April 2024 that it had completed enrollment in the trial.2

The CLARA trial is a prospective, multi-center, randomized, double masked, parallel-arm cell dose-ranging clinical trial evaluating 3 different doses of neltependocel used in combination with Y-27632. The trial has randomized 97 participants across trial sites in the Untied States and Canada.

The CLARA trial is designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction. The study is primarily assessing the percentage of participants who gain 3 lines of vision or a 15-letter improvement from baseline in best-corrected visual acuity (BCVA) at 6 months. The cell therapy product used in the CLARA trial was supplied by Aurion’s CDMO partner, Charles River Laboratories.2

“Aurion Biotech is delighted that both enrollment and dosing in our Phase 1/2 trial are now complete, and that it happened more rapidly than we expected,” Michael Goldstein, MD, MBA, President and Chief Medical Officer, Aurion Biotech, said in a statement about the completed enrollment.2 “We enrolled and dosed subjects in 6 months, which speaks to the deep interest among physicians and patients for our allogeneic cell therapy, as well as the potentially large, unmet need for our elegant, minimally invasive procedure to treat this sight-threatening disease.”

1. Aurion Biotech Receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001. News release. Aurion Biotech. June 18, 2024.
2. Aurion Biotech Announces Completion of Enrollment In Phase 1 / 2 Clinical Trial. . News release. Aurion Biotech. April 30, 2024.
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