U.S. pivotal trial to assess efficacy and safety of AURN001 compared to placebo
Potential first-in-class cell therapy to regenerate corneal clarity and restore vision with a single procedure
SEATTLE & CAMBRIDGE, Mass. & TOKYO--(BUSINESS WIRE)--#AurionBiotech--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 that the first patients have been dosed in the U.S. ASTRA Phase 3 pivotal study of AURN001, a single-administration 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.
“Dosing the first ASTRA study participants marks an important milestone for patients, physicians, and Aurion as we work together to advance new solutions for vision loss from corneal endothelial disease,” said Edward J. Holland, MD, Chief Medical Officer, Aurion Biotech. “While modern endothelial keratoplasty procedures have transformed the treatment of the disease, there still is a need to reduce complications and improve our patient's experience. AURN001 represents the potential next evolution by moving from donor tissue-based graft surgery toward a cultured cell therapy designed to restore corneal clarity and expand access to care for patients worldwide.”
“Reaching this milestone reflects the dedication of our investigative sites, whose rigor and deep commitment to patients with corneal endothelial dystrophy have made this possible,” said Eris Jordan, OD, Chief Development Officer, Aurion Biotech. “Their enthusiasm for this program underscores a shared belief that regenerative cell therapy has the potential to fundamentally transform how we treat this disease.”
AURN001 is a first-in-class, investigational combination therapy designed to transform treatment for corneal endothelial disease, including Fuchs dystrophy. It consists of unmodified human corneal endothelial cells (neltependocel) and a rho-kinase inhibitor (Y-27632). Additionally, AURN001 is designed to regenerate corneal clarity and vision through an intracameral injection that has the potential to reduce treatment-related complications and support a fast post-operative recovery.
“This pivotal study is the foundation for our U.S. BLA submission and a critical step in our mission to address a very large global unmet patient need,” said Arnaud Lacoste, PhD, MBA, Chief Executive Officer and Chief Scientific Officer, Aurion Biotech. “By using donor corneas to create scalable, and transportable cell therapy, we are overcoming the limitations of traditional surgery. Following our success in Japan, we are now focused on providing a global solution for blindness.”
The ASTRA clinical trial has initiated in the United States. For more information on the ASTRA study, please visit: https://aurionclinicaltrials.com/ and NCT07368959.
About ASTRA Phase 3 Study
Patients will be enrolled at 12 sites across the U.S. and randomized to AURN001 + Y-27632. 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 non-contact Central Corneal Thickness (CCT) at Month 6 and change from baseline in BCVA at Month 6.
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.
Contacts
Media Contacts:
Michele Gray
(Ophthalmology Media)
917-449-9250
michele@mgraycommunications.com
Beth Keshishian
(Business / Biotech Media)
917-912-7195
beth@bethkeshishian.com
