Key Takeaways

  • Aurion Biotech has completed enrollment in the US Phase 3 ASTRA pivotal study evaluating AURN001 for corneal edema secondary to corneal endothelial dysfunction
  • AURN001 combines unmodified human corneal endothelial cells, neltependocel, with the rho-kinase inhibitor Y-27632 and is administered through an intracameral injection
  • The study’s primary endpoint evaluates the proportion of patients achieving at least a 15-letter improvement in BCVA at month 6, with Aurion planning a BLA submission as the program advances

Aurion Biotech has completed patient enrollment in the US phase 3 ASTRA pivotal study evaluating AURN001, an investigational regenerative cell therapy for corneal edema secondary to corneal endothelial dysfunction, the company announced.

AURN001 is a single-administration combination therapy consisting of unmodified human corneal endothelial cells (neltependocel) and the rho-kinase inhibitor Y-27632. Administered by intracameral injection, the investigational treatment is designed to restore corneal clarity and improve vision by regenerating the corneal endothelium.

If approved, AURN001 has the potential to become the first cell therapy approved in the United States for corneal edema associated with corneal endothelial dysfunction, according to Aurion.

“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,” Eris P. Jordan, OD, chief development officer at Aurion Biotech, said in a statement.

Dr. Jordan noted that patient input was incorporated into the phase 3 study design through a dedicated patient advisory board, with feedback helping shape trial feasibility and clinically relevant endpoints.

The ASTRA study enrolled patients at 12 sites across the United States. Its primary endpoint is the proportion of participants achieving an improvement of at least 15 letters, equivalent to a 3-line gain, from baseline in best-corrected visual acuity (BCVA) at month 6. Secondary endpoints include change from baseline in central corneal thickness and BCVA at month 6.

Corneal endothelial diseases, including Fuchs dystrophy, can lead to progressive corneal edema and vision loss. Current treatment frequently relies on corneal transplantation techniques using donor tissue. Aurion is developing its cell therapy approach in part to address limitations associated with donor tissue availability and the complexity of existing surgical procedures.

“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 of Aurion Biotech.

Dr. Lacoste said the company’s regulatory, quality, manufacturing, and clinical infrastructure helped it complete enrollment on an accelerated timeline. He added that Aurion intends to build on its experience in Japan as it advances AURN001 as a potentially scalable and transportable regenerative therapy for corneal endothelial disease.