Aurion Biotech completes enrollment in phase 3 ASTRA study of AURN001 for corneal edema secondary to corneal endothelial dysfunction

Aurion Biotech, Inc. announced in a press release that patient enrollment has been completed in the 12 US sites of the ASTRA phase 3 pivotal study (NCT07368959) of the cell therapy AURN001 designed to treat corneal edema associated with corneal endothelial dysfunction. This treatment, an investigational regenerative therapy, may be the first such therapy approved in the US to treat this condition, according to the company.

The phase 3 study is assessing the efficacy and safety of AURN001. The primary endpoint is the percentage of patients who gain a minimum of 15 letters or more (three lines of vision) in the best-corrected visual acuity (BCVA) at month 6 compared with baseline. Two secondary endpoints are the changes in the central corneal thickness and BCVA at month 6 compared with baseline.

Arnaud Lacoste, PhD, MBA, chief executive officer and chief scientific officer of Aurion Biotech, said, "Completing enrollment in our U.S. pivotal study marks a major milestone for Aurion and an important step toward our planned BLA submission."

Eris P. Jordan, OD, the company’s Chief Development Officer, commented, “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.”

About AURN001

The treatment is described as a potential first-in-class investigational combination therapy for corneal endothelial disease, including Fuchs dystrophy. It is comprised of unmodified human corneal endothelial cells (neltependocel, Vyznova) from donated corneas with a rho-kinase inhibitor (Y-27632). AURN001 is administered once via intracameral injection and is designed to restore corneal clarity and vision with the single procedure, according to the press release.

The therapy is designed to replace lost or dysfunctional endothelial cells that cannot regenerate naturally, causing progressive vision loss. The cells used in the therapy come from donated corneas that are intended to repopulate the endothelium and help rebuild the corneal structure.

The company cited the significant global burden of corneal endothelial disease, the lack of available donor tissue and the complexity of current surgical approaches, all of which underscore the need for development of effective treatments. The efficacy of AURN001 may potentially broaden access to care for patients who have limited options.