Aurion Biotech's AURN001 Receives Breakthrough Therapy and Regenerative Medicine Advanced Therapy Designations
Designation for AURN001 is an allogeneic cell therapy intended to treat corneal edema caused by corneal endothelial disease.
The Breakthrough Therapy Designation is aimed at accelerating the development and review of treatments for serious conditions, particularly when there is preliminary evidence suggesting significant improvement over existing therapies.
The RMAT designation applies to investigational drugs that qualify as regenerative medicine therapies, targeting serious conditions and showing potential to meet unmet medical needs.
These designations have increased interactions with the FDA, streamlining the drug development process. They reflect the pressing medical need among patients with corneal endothelial disease and the FDA’s review of clinical data from the AURN001 program, which includes multiple clinical trials with subjects.
This announcement follows patient enrollment and dosing for its Phase 1/2 CLARA trial. The CLARA trial is a randomised, double-masked study assessing three different doses of neltependocel combined with Y-27632 in subjects with corneal edema due to endothelial dysfunction.
Ninety-seven participants have been randomised, and the trial aims to evaluate the safety, tolerability, and efficacy of AURN001, with the primary endpoint being the proportion of subjects achieving a three-line improvement in vision after six months.
These designations increase the drug development process which will evaluate the safety and efficacy of the therapy in improving vision.
