Aurion Biotech has secured two key designations from the US Food and Drug Administration (FDA) for its allogeneic cell therapy candidate AURN001, aimed at treating corneal edema secondary to corneal endothelial disease.

AURN001 has been granted the breakthrough therapy designation (BTD) and regenerative medicine advanced therapy (RMAT) designation for the indication by the US regulator.

Aurion Biotech regulatory affairs and quality vice president Sterling Chung said: “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.”

Administered via a one-time intracameral injection, AURN001 received regulatory approval in March 2023 under the brand Vyznova in Japan for treating bullous keratopathy of the cornea.

AURN001 combines neltependocel (allogeneic human corneal endothelial cells [CECs]) with Y-27632, an inhibitor of Rho-associated, coiled-coil containing protein kinase (ROCK).

Its breakthrough therapy and RMAT designations follow the completion of enrolment and dosing in the CLARA Phase 1/2 clinical trial across sites in the US and Canada.

The early-stage trial began in late April 2024.

It aims to evaluate the safety, tolerability, and efficacy of AURN001 in treating corneal edema secondary to endothelial dysfunction, a condition that can lead to vision loss if untreated.

Corneal edema results from the loss or degradation of corneal endothelial cells, which are crucial for maintaining corneal clarity and preventing swelling.

The CLARA trial will assess three different doses of neltependocel combined with Y-27632 in 97 randomised subjects.

The primary endpoint is to determine the percentage of subjects achieving a gain of three lines of vision at six months.

Breakthrough therapy designation expedites the development and review of drugs targeting serious conditions where preliminary clinical evidence indicates potential substantial improvement over existing therapies.

RMAT designation under the 21st Century Cures Act applies to investigational drugs that qualify as regenerative medicine therapies, targeting serious conditions with potential to address unmet medical needs based on preliminary clinical evidence.

Aurion Biotech CEO Greg Kunst said: “The BTD and RMAT designations are important milestones for people living with corneal endothelial disease who need better, less burdensome, more accessible treatment options.”