Aurion Biotech receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for AURN001

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Aurion Biotech announced that the FDA has granted both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation for AURN001, the company’s allogeneic cell therapy candidate for the treatment of corneal edema secondary to corneal endothelial disease.

Sterling Chung, vice president of Regulatory Affairs and Quality at Aurion Biotech, said in a news release the company was pleased to receive the Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for AURN001.¹

“These designations underscore the importance of developing a potential solution for millions of patients around the world who suffer from corneal endothelial diseases,” Chung said in the release. “We look forward to working closely with the FDA to expedite the development of our cell therapy.”

Greg Kunst, CEO of Aurion, pointed out the BTD and RMAT designations are important milestones for people living with corneal endothelial disease who need better, less burdensome, more accessible treatment options.

“I thank the Aurion team for their hard work and dedication, and I’m grateful for the essential contributions of clinical trial patients and their ophthalmologists - all of whom have helped to make today’s news a reality,” he said.

According to the news release, corneal edema secondary to endothelial dysfunction is a sight-threatening and debilitating condition affecting millions of people throughout the world. When corneal endothelial cells die or degrade, they do not regenerate. If left untreated, corneal endothelial cell loss can cause corneal edema (swelling) and loss of vision.

According to the company’s news release, the FDA’s Breakthrough Therapy Designation is designed to expedite the development and review of drugs that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).¹

RMAT is part of the 21st Century Cures Act.² The FDA noted in a news release investigational drugs are eligible for RMAT designation if it meets the definition of regenerative medicine therapy; is intended to treat, modify, reverse, or cure a serious condition; and preliminary clinical evidence indicates that the regenerative medicine therapy has the potential to address unmet medical needs for such conditions.²

Companies that earn BTD and RMAT designation can benefit from increased interactions with the FDA with the goal of expediting drug development. The dual designations granted to Aurion Biotech are based on the criteria, the unmet medical need among patients with corneal endothelial disease, and FDA’s review of clinical data from the AURN001 program, which includes multiple clinical trials with subjects having completed at least 12 months of follow-up.¹

This news comes on the heels of Aurion’s announcement that it has completed enrollment and dosing of its Phase 1 / 2 CLARA trial in the U.S. and Canada.3 The CLARA clinical trial is a prospective, multi-center, randomized, double-masked, parallel-arm cell dose-ranging study in subjects with corneal edema secondary to corneal endothelial dysfunction.

According to the company, the study will evaluate 3 different doses of neltependocel used in combination with Y-27632. Ninety-seven subjects have been randomized at US and Canadian sites. The CLARA trial is designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction. The primary endpoint is the percentage of subjects who gain 3 lines of vision at 6 months.

The company has already received regulatory approval in Japan.

References:

1. Aurion Biotech Receives Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation for Its Drug Candidate AURN001. Businesswire.com. Published June 19, 2024. Accessed June 19, 2024. https://www.businesswire.com/news/home/20240619102413/en/Aurion-Biotech-Receives-Breakthrough-Therapy-Designation-and-Regenerative-Medicine-Advanced-Therapy-Designation-for-Its-Drug-Candidate-AURN001

2. Center for Biologics Evaluation and Research. Regenerative Medicine Advanced Therapy Designation. U.S. Food and Drug Administration. Published 2019. https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/regenerative-medicine-advanced-therapy-designation

3. wordpress@pint.com. Aurion Biotech Announces Completion of Enrollment In Phase 1 / 2 Clinical Trial. Aurion Biotech. Published April 30, 2024. Accessed June 19, 2024. https://aurionbiotech.com/aurion-biotech-announces-completion-of-enrollment-in-phase-1-2-clinical-trial