Aurion Biotech Gains Breakthrough and Regenerative Medicine Designations for AURN001

Aurion Biotech has achieved a significant milestone in its mission to provide regenerative therapies for vision restoration. The U.S. Food and Drug Administration (FDA) has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation to AURN001, Aurion's allogeneic cell therapy candidate, aimed at treating corneal edema secondary to corneal endothelial disease. This makes AURN001 the first allogeneic cell therapy to receive these dual designations for this specific condition.

The Breakthrough Therapy Designation is intended to accelerate the development and review process for drugs that target serious conditions and have shown preliminary clinical evidence of substantial improvement over existing therapies. RMAT, introduced as part of the 21st Century Cures Act, is reserved for investigational drugs that qualify as regenerative medicine therapies designed to treat, modify, reverse, or cure serious conditions. To be eligible for RMAT, preliminary clinical evidence must indicate that the therapy has the potential to address unmet medical needs.

Aurion Biotech stands to benefit from increased interactions with the FDA due to these designations, which aim to expedite the development process. The decision to grant these designations was based on the unmet medical needs of patients with corneal endothelial disease and the FDA’s review of clinical data from the AURN001 program, which includes multiple clinical trials and follow-ups extending to at least 12 months.

Sterling Chung, Vice President of Regulatory Affairs and Quality at Aurion Biotech, expressed gratitude for receiving these designations, emphasizing the importance of developing solutions for millions of patients worldwide suffering from corneal endothelial diseases. He looks forward to working closely with the FDA to expedite the development of their cell therapy.

This announcement comes shortly after Aurion Biotech disclosed the completion of enrollment and dosing in its Phase 1 / 2 CLARA trial in the U.S. and Canada. The CLARA trial is a prospective, multi-center, randomized, double-masked, parallel-arm study focusing on subjects with corneal edema secondary to corneal endothelial dysfunction. It evaluates three different doses of neltependocel combined with Y-27632. Ninety-seven subjects have been randomized across sites in the U.S. and Canada. The trial aims to assess the safety, tolerability, and efficacy of AURN001, with the primary endpoint being the percentage of subjects who achieve a three-line improvement in vision at six months.

Aurion Biotech has already obtained regulatory approval for AURN001 in Japan. Greg Kunst, the company's Chief Executive Officer, remarked on the significance of the BTD and RMAT designations for those suffering from corneal endothelial disease, highlighting the need for better, more accessible treatment options. He thanked the Aurion team and acknowledged the crucial contributions of clinical trial patients and their ophthalmologists.

Corneal endothelial dysfunction leads to corneal edema, a sight-threatening condition that affects millions globally. The disease occurs when corneal endothelial cells die or degrade, a process that cannot be naturally reversed. Untreated, it can result in corneal swelling and vision loss. While corneal transplants can be effective, they are hindered by a limited supply of donor organs and require patients to lie flat on their backs for up to three days post-surgery for the transplant to properly adhere.

Aurion Biotech aims to address these challenges with their innovative therapies. The company received the prestigious Prix Galien award for best start-up in biotech. Aurion’s first candidate therapy for corneal edema secondary to corneal endothelial disease is also the first clinically validated cell therapy for corneal care, having received approval in Japan. Aurion Biotech's ongoing clinical efforts in the U.S. and Canada are supported by major investors, including Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures.