Aurion Biotech’s ABA-1 Clinical Trial for Corneal Edema Cell Therapy AURN001 Doses First Canadian Patient

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AURN001 consists of allogeneic human corneal endothelial cells referred to as “neltependocel” and Y-27632, a small molecule drug.

Aurion Biotech has dosed the first Canadian patient in its phase 1/2 ABA-1, CLARA clinical trial (NCT06041256) evaluating AURN001 (marketed as Vyznova in Japan), a combination cell therapy product, for the treatment of corneal edema secondary to corneal endothelial dysfunction.1

AURN001 consists of allogeneic human corneal endothelial cells referred to as “neltependocel” and Y-27632, a small molecule drug that inhibits Rho-associated, coiled-coil containing protein kinase. AURN001, which is delivered to the eye via intracameral injection, is meant to be a 1-time treatment for certain corneal conditions.

In the multicenter, randomized, double-masked, dose-ranging ABA-1, CLARA trial, approximately 100 patients will be randomly assigned to receive either AURN001, neltependocel alone, or Y-27632 alone. Patients who receive AURN001 will receive product with either a low dose, medium dose, or high dose of neltependocel, depending on their assigned arm. All patients who received neltependocel alone will receive the same dose of neltependocel as patients in the high dose AURN001 arm. The primary outcome measure for the study is the percentage of participants who achieve a 3-line gain from baseline in best-corrected visual acuity at 6 months posttreatment. The trial is taking place at sites in both the United States and Canada.

“We are very pleased to have begun dosing subjects in this trial in Canada,” Michael Goldstein, MD, MBA, the president and chief medical officer of Aurion Biotech, said in a statement.1 “Treating subjects in both the US and Canada in our CLARA trial is an important milestone in our clinical development program. We believe that our cell therapy has the potential to revolutionize the treatment of corneal patients throughout the world.”

Notably, AURN001, which is intended to treat multiple indications that traditionally call for corneal transplant, was approved for the treatment of bullous keratopathy of the cornea in Japan in March 2023.2 Later that year, in October, Aurion announced that the first patient in ABA-1, CLARA had been dosed at a site in the US.3

“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,” Goldstein said at the time.3

Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech

Arnaud Lacoste, PhD

In April 2023, a poster entitled “AURN001, a Cell Therapy with Immunomodulatory Properties Required for Long-term Maintenance of the Immunological Homeostasis of the Eye” was presented by Aurion at the Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting, held April 23-27, 2023, in New Orleans, Louisiana.4 Around this time, CGTLive® interviewed Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech, who co-authored the poster. Lacoste explained the rationale behind treating corneal dystrophies with a cell therapy like AURN001.

“It seems to us that a cell therapy is a much better option than typical corneal transplant for both patients and ophthalmic surgeons,” Lacoste told CGTLive. “For patients, the procedure required to deliver cells is much less invasive than typical corneal transplantation—and our clinical studies suggest that the cell therapy restores vision at least to the level that corneal transplants do currently. For ophthalmic surgeons, cell therapy is also advantageous because the procedure is less complex to perform [which] makes it much more likely that more ophthalmic surgeons will be able to provide the treatment and that improves, of course, patient access.”

REFERENCES
1. Aurion Biotech announces first Canadian subject dosed in phase 1/2 clinical trial. News release. Aurion Biotech. March 28, 2024. Accessed April 8, 2024. https://aurionbiotech.com/aurion-biotech-announces-first-canadian-subject-dosed-in-phase-1-2-clinical-trial/
2. Aurion Biotech receives approval from Japan’s PMDA for new drug application. News release. Aurion Biotech. March 23, 2023. Accessed April 25, 2023. https://aurionbiotech.com/aurion-biotech-receives-approval-from-japans-pmda-for-new-drug-application/
3. Aurion Biotech announces first subject dosed in U.S. Phase 1/2 clinical trial. Aurion Biotech. October 24, 2024. Accessed April 8, 2024. https://aurionbiotech.com/aurion-biotech-announces-first-subject-dosed-in-us-clinical-trial/
4. Littlefield C, Berenshteyn F, Yasin J. AURN001, a cell therapy with immunomodulatory properties required for long-term maintenance of the immunological homeostasis of the eye. Presented at: Association for Research in Vision and Ophthalmology (ARVO) 2023 Annual Meeting. April 23-27, 2023; New Orleans, LA. Abstract #779 – C0380
 
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