Aurion Biotech candidate AURN001 receives US Breakthrough Therapy Designation and Regenerative Medicine Advanced Therapy Designation

The FDA designations are meant to facilitate and expedite the development of novel therapeutics. Image credit: ©Postmodern Studio – stock.adobe.com

Aurion Biotech, a clinical-stage biotechnology firm based jointly in the United States and Japan, announced two rulings by the US Food and Drug Administration (FDA). The FDA has granted AURN001, the company’s allogeneic cell therapy candidate for the treatment of corneal oedema secondary to corneal endothelial disease, with both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) Designation.

Sterling Chung, vice president of Regulatory Affairs and Quality at Aurion Biotech, said in a news release the company was pleased to receive the BTD and RMAT designations.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 and 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.

Aurion Biotech, in its news release, explained that corneal oedema secondary to endothelial dysfunction is a sight-threatening and debilitating condition, which affects 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 oedema and loss of vision.

A BTD from the FDA 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, that investigational drugs are eligible for RMAT designation if they meet the definition of regenerative medicine therapy; are 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 US and Canada.³ The CLARA clinical trial is a prospective, multi-centre, randomised, double-masked, parallel-arm cell dose-ranging study in subjects with corneal oedema secondary to corneal endothelial dysfunction.

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

In March 2023, the company received regulatory approval from Japan's Pharmaceuticals and Medical Devices Agency.

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. 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