Aurion Biotech, Inc. (“Aurion Biotech”), a commercial-stage regenerative cell therapy company dedicated to restoring vision and working toward a future where blindness is eliminated, today announced the completion of patient enrollment in the U.S. ASTRA Phase 3 pivotal study of AURN001, a single-administration investigational regenerative cell therapy for corneal edema secondary to corneal endothelial dysfunction. AURN001 has the potential to be the first cell therapy approved in the U.S. to treat this condition.

“Completing enrollment in ASTRA is a shared achievement. Dosing our final patient in this study reflects the work of our investigators and site teams who executed with conviction, the patients who trusted us with their vision, and the cross-functional teams here at Aurion who cleared every obstacle along the way,” said Eris P. Jordan, OD, Chief Development Officer, Aurion Biotech. “The Phase 3 protocol was built to two standards at once: the regulatory expectations for a pivotal study, and the lived expectations of the patients it is designed to serve. Through a dedicated patient advisory board, we invited the community into the design process, and their input shaped both trial feasibility and the endpoints that matter most to patients living with this disease.”

The significant global burden of corneal endothelial disease, combined with the limited availability of donor tissue and the complexity of current surgical approaches, highlights the need for treatment options that can scale more effectively. AURN001 is a potential first-in-class investigational combination therapy for corneal endothelial disease, including Fuchs dystrophy. By addressing the barriers of surgical complexity, donor tissue scarcity, and post-operative complications, AURN001 has the potential to broaden access to care for patients who currently have limited options. Consisting of unmodified human corneal endothelial cells (neltependocel) and a rho-kinase inhibitor (Y-27632), AURN001 is designed to regenerate corneal clarity and vision through an intracameral injection.

“Completing enrollment in our U.S. pivotal study marks a major milestone for Aurion and an important step toward our planned BLA submission,” said Arnaud Lacoste, PhD, MBA, Chief Executive Officer and Chief Scientific Officer, Aurion Biotech. “This achievement reflects the strength of the foundation established across our regulatory, quality, manufacturing, and clinical teams from the outset of the program. Their collective expertise and execution enabled us to reach full enrollment on an accelerated timeline and brings us closer to delivering a scalable, transportable regenerative cell therapy for patients affected by corneal endothelial blindness. Building on our success in Japan, we remain committed to advancing a global solution for this significant unmet medical need.”

About ASTRA Phase 3 Study

Patients were enrolled at 12 sites across the U.S. The primary endpoint is the percentage of participants who achieve a ≥ 15-letter improvement (3-line gain) from baseline in Best Corrected Visual Acuity (BCVA) at Month 6. Secondary endpoints are changes from baseline in central corneal thickness (CCT) and best corrected visual acuity (BCVA) at Month 6. For more information on the ASTRA study, please visit: https://aurionclinicaltrials.com/ and NCT07368959 on clinicaltrials.gov.

About Corneal Endothelial Cell Therapy (CECT)

Aurion Biotech is advancing Corneal Endothelial Cell Therapy (CECT), a regenerative treatment that aims to replace lost or dysfunctional endothelial cells. Because endothelial cells cannot naturally regenerate, patients experience progressive vision loss. The cells delivered to patients are human corneal endothelial cells obtained from donated corneas. They do not contain any synthetic materials or artificial scaffold. The cells do not undergo genetic reprogramming. Aurion’s cells are intended to repopulate the endothelium and help re-establish a corneal structure that is virtually indistinguishable from a healthy cornea with healthy fluid balance.

About Aurion Biotech

Aurion’s mission is to restore vision to millions of patients with life-changing regenerative therapy. Aurion is a commercial stage company in Japan and a clinical stage company in Phase 3 development in the U.S. The company is developing AURN001, an investigational, single-administration, allogeneic cell therapy for corneal endothelial disease, a condition that causes progressive vision loss in millions of patients worldwide. In 2024, Aurion launched Vyznova™, the first cell therapy for corneal endothelial disease commercially available in Japan. Aurion received the prestigious Prix Galien award for Best Start-Up in Biotech in 2022 and was named to Fast Company’s annual list of Most Innovative Companies in 2025. In 2025, Alcon acquired majority ownership of Aurion Biotech. For more information, visit www.aurionbiotech.com and follow us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding the clinical development, potential benefits, manufacturing capacity, regulatory status, and timing of future clinical trials of AURN001. Forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results to differ materially, including those related to clinical trial design, patient enrollment, safety and efficacy results, regulatory review and approval, manufacturing, supply, and other factors. Aurion undertakes no obligation to update any forward-looking statements to reflect events or circumstances after the date of this release.

Any product/brand names and/or logos are trademarks of Aurion Biotech, Inc.

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