Aurion Biotech Announces Positive Phase 1/2 Results for AURN001 in Corneal Edema Treatment
Aurion Biotech has released topline data from its Phase 1/2 clinical trial (CLARA), evaluating AURN001, an allogeneic cell therapy product candidate for treating corneal edema secondary to corneal endothelial dysfunction. These results highlight the therapy's potential as a groundbreaking treatment option for this debilitating condition.
About AURN001
AURN001 is a combination cell therapy that includes:
• Allogeneic human corneal endothelial cells (neltependocel).
• Rho kinase inhibitor (Y-27632).
The therapy is delivered as a one-time procedure into the anterior chamber of the eye to restore corneal endothelial function.
CLARA Phase 1/2 Trial Overview
The CLARA trial (NCT06041256) was a prospective, multicenter, randomized, double-masked, parallel-arm, dose-ranging study conducted at sites in the United States and Canada.
Key Study Details:
• Participants: 97 subjects, mean age 71.4 years, 55% female.
• Baseline Vision: Mean BCVA was 53.5 letters (20/85 Snellen); mean CCT was 676.6 microns.
• Treatment Arms:
1. High-dose AURN001: 1.0 × 10⁶ neltependocel + 100 μM Y-27632.
2. Medium-dose AURN001: 5.0 × 10⁵ neltependocel + 100 μM Y-27632.
3. Low-dose AURN001: 2.5 × 10⁵ neltependocel + 100 μM Y-27632.
4. Y-27632 only.
5. Neltependocel only: 1.0 × 10⁶ neltependocel.
Endpoints:
• Primary Endpoint:
- Proportion of responders achieving a >15-letter improvement (≥3-line gain) in BCVA at 6 months.
• Key Secondary Endpoints:
1. Change in BCVA at 6 months.
2. Change in CCT at 6 months.
3. Safety and tolerability.
Topline Results
Primary Endpoint: BCVA Response
• High-dose AURN001:
- 50% responders (P = .020).
- Compared to 14.3% in the Y-27632-only group.
• Dose-dependent improvements observed across all AURN001 arms.
Key Secondary Endpoints
Change in BCVA at 6 Months
• High-dose AURN001: Statistically significant improvement (P = .002) vs. Y-27632-only.
Change in CCT at 6 Months
• High-dose AURN001: Statistically significant improvement (P = .012) vs. Y-27632-only.
Quality of Life Improvements
• Patient-reported quality of life, measured via the Visual Function Questionnaire (VFQ-25), showed the greatest benefit in the high-dose AURN001 group.
Safety Profile
AURN001 demonstrated a favorable safety profile across all doses:
• No dose-related increase in adverse events (AEs).
• No ocular serious adverse events (SAEs) reported.
• Most common ocular treatment-emergent AEs (>3%):
- Ocular hypertension (9.3%).
- Conjunctival hemorrhage (5.2%).
- Eye pain (4.1%).
- Cystoid macular edema (3.1%).
• Most common non-ocular AE: COVID-19 (3.1%).
• Two non-ocular SAEs: hip fracture and femur fracture.
Leadership Commentary
Dr. Michael Goldstein, President and Chief Medical Officer of Aurion Biotech, expressed optimism:
“We are thrilled with the topline results of the CLARA trial. The statistically significant improvement in the high-dose AURN001 arm at 6 months, combined with the favorable safety profile, positions AURN001 for Phase 3 pivotal trials.”
Next Steps and Regulatory Milestones
The CLARA results follow Aurion Biotech’s recent achievements:
• June 2024: FDA granted Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) designation for AURN001.
• September 2024: Launch of AURN001 in Japan under the trade name Vyznova.
Based on the promising CLARA results, Aurion Biotech plans to advance the high-dose AURN001 into Phase 3 pivotal trials.
Conclusion
Aurion Biotech’s AURN001 represents a transformative step in treating corneal edema secondary to corneal endothelial dysfunction. With its robust efficacy data, favorable safety profile, and global regulatory advancements, AURN001 offers new hope for patients struggling with this vision-threatening condition.
