Aurion Biotech’s AURN001 Shows Significant Vision Improvement in Phase 1/2 Trial for Corneal Edema

Clinical Trial Success:
Aurion Biotech's Phase 1/2 CLARA trial of AURN001, an allogeneic cell therapy for corneal edema secondary to corneal endothelial dysfunction, demonstrated significant dose-dependent efficacy, particularly in the high-dose group134.

Primary Endpoint Achievement:
The high-dose AURN001 arm showed a statistically significant improvement in the primary endpoint, with 50% of responders achieving a ≥15-letter improvement (≥3-line gain) in best-corrected visual acuity (BCVA) at 6 months (p=0.020)34.

Secondary Endpoints:
Key secondary endpoints, including change in BCVA and central corneal thickness (CCT) at 6 months, also showed statistically significant improvements in the high-dose AURN001 arm compared to the Y-27632-only arm134.

Safety Profile:
All treatment arms demonstrated favorable safety profiles, with no dose-dependent trends in adverse events (AEs) and no ocular serious adverse events (SAEs) reported134.

Regulatory Milestones:
AURN001 received Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy (RMAT) Designation from the FDA in June 2024, and it was commercially launched in Japan under the trade name Vyznova in September 202423.

Future Plans:
Based on the positive results, Aurion Biotech plans to advance the high dose of AURN001 into proposed Phase 3 pivotal trials134.

Sources:

1. https://www.optometrytimes.com/view/aurion-biotech-unveils-positive-topline-results-from-phase-1-2-trial-of-aurn001-for-corneal-edema

2. https://www.cgtlive.com/view/aurion-cell-therapy-rmat-breakthrough-therapy-designation-corneal-edema

3. https://www.businesswire.com/news/home/20241218295852/en/Aurion-Biotech-Announces-Positive-Topline-Data-for-Phase-12-Clinical-Trial-of-AURN001-an-Allogeneic-Cell-Therapy-Product-Candidate-for-the-Treatment-of-Corneal-Edema-Secondary-to-Corneal-Endothelial-Dysfunction

4. https://www.biospace.com/press-releases/aurion-biotech-announces-positive-topline-data-for-phase-1-2-clinical-trial-of-aurn001-an-allogeneic-cell-therapy-product-candidate-for-the-treatment-of-corneal-edema-secondary-to-corneal-endothelial-dysfunction