Aurion Biotech Announces First Subject Dosed in U.S. Phase 1 / 2 Clinical Trial

Allogeneic Cell Therapy for the Treatment of Corneal Edema Secondary to Corneal Endothelial Dysfunction

SEATTLE & CAMBRIDGE, Mass. & TOKYO--(BUSINESS WIRE)--Aurion Biotech, whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced that it has dosed the first subject in its Phase 1 / 2 U.S. clinical trial (ABA-1, CLARA) of AURN001, a cell therapy for the treatment of corneal edema secondary to corneal endothelial dysfunction.

“On the heels of our recent approval in Japan, we are very pleased to have begun dosing subjects in this trial in the United States,” said Michael Goldstein, MD, MBA, President and Chief Medical Officer of Aurion Biotech. “The start of this trial is another important milestone in our goal of bringing this corneal endothelial cell therapy to millions of patients in need throughout the world. We believe that cell therapy has the potential to revolutionize the treatment of corneal patients around the world.”

AURN001 is a combination cell therapy product (biologic/drug) comprised of neltependocel (allogeneic human corneal endothelial cells (CECs]) and Y-27632 (an inhibitor of Rho-associated, coiled-coil containing protein kinase [ROCK]). AURN001 is intended to be administered to the eye as a one-time, intracameral injection.

The Phase 1 / 2 clinical trial (ABA-1, CLARA) is a prospective, multi-center, randomized, double masked, parallel-arm cell dose-ranging clinical trial in subjects with corneal edema secondary to corneal endothelial dysfunction. The study will evaluate three different doses of neltependocel used in combination with Y-27632. Approximately 100 subjects will be randomized at US sites.

This 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 percentage of subjects who gain 3 lines of vision at 6 months.

About Corneal Endothelial Dysfunction

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. Although corneal transplants are effective, there are disadvantages with these procedures – PKP/DMEK/DSAEK – including limited donor organ supply. Transplants require a supply of donor corneas in a 1:1 ratio (one healthy donor cornea to treat each diseased one), yet it’s estimated that there is only one donor cornea available for every 70 diseased eyes.¹ In addition, post-operative recovery for corneal transplant patients requires that they like flat on their backs for up to three days, in order for the transplant to adhere to the corneal stroma.

About Aurion Biotech

With offices in Seattle, Cambridge and Tokyo, Aurion Biotech is a clinical-stage biotech company, whose mission is to restore vision to millions of patients with life-changing regenerative therapies. Aurion Biotech is the recipient of the prestigious Prix Galien award for best start-up in biotech. The Company’s first candidate is for the treatment of corneal edema secondary to corneal endothelial dysfunction, and is the first off-the-shelf, clinically validated cell therapy for corneal care, having received regulatory approval in Japan. The Company has initiated clinical trials in the U.S. Privately held, Aurion Biotech is backed by leading investors that include Deerfield, Alcon, Petrichor, Flying L Partners, Falcon Vision / KKR, and Visionary Ventures. To learn more about Aurion Biotech, please visit www.aurionbiotech.com.

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1} https://jamanetwork.com/journals/jamaophthalmology/fullarticle/2474372