Aurion Biotech Announces Positive Topline Data for Phase 1/2 Clinical Trial of AURN001, an Allogeneic Cell Therapy Product Candidate for the Treatment of Corneal Edema Secondary to Corneal Endothelial Dysfunction

SEATTLE & CAMBRIDGE, Mass. & TOKYO--( BUSINESS WIRE )-- Aurion Biotech , Inc. (Aurion Biotech), whose mission is to restore vision to millions of patients with life-changing regenerative therapies, today announced topline data from its Phase 1/2 clinical trial (CLARA) of AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction.

AURN001 is a combination cell therapy product candidate comprised of allogeneic human corneal endothelial cells (neltependocel) and a rho kinase inhibitor (Y-27632). AURN001 is intended to be administered to the anterior chamber of the eye as a one-time procedure.

The CLARA Phase 1/2 clinical trial ( NCT06041256 ) is a prospective, multi-center, randomized, double-masked, parallel-arm dose-ranging clinical trial designed to assess the safety, tolerability, and efficacy of AURN001 for the treatment of corneal edema secondary to corneal endothelial dysfunction. Ninety-seven subjects were randomized at US and Canadian sites to each of the following five arms:

• AURN001: 1.0 × 10⁶ neltependocel + 100 μM Y-27632 (high cell dose)
• AURN001: 5.0 × 10⁵ neltependocel + 100 μM Y-27632 (medium cell dose)
• AURN001: 2.5 × 10⁵ neltependocel + 100 μM Y-27632 (low cell dose)
• 100 μM Y-27632
• 1.0 × 10⁶ neltependocel

At baseline across all arms, the mean age was 71.4 years, with 55% being female subjects. Baseline mean BCVA was 53.5 letters, using the Early Treatment Diabetic Retinopathy Standard (ETDRS) visual acuity test (20/85 Snellen), and CCT was 676.6 microns.

The primary endpoint was the proportion of responders with a >15-letter improvement (>3-line gain) from baseline in BCVA at six months. Key secondary endpoints included (i) change from baseline in BCVA and in CCT at six months and (ii) safety and tolerability.

For the primary endpoint, a dose-dependent response was observed in the three AURN001 arms, with a statistically significant improvement in the high-dose AURN001 arm (50% of responders, p=0.020), as compared to the Y-27632-only arm (14.3%).

A key secondary endpoint, change in BCVA at six months, showed a statistically significant improvement for the high-dose AURN001 arm (p=0.002), as compared to the Y-27632-only arm (using the full analysis set population (LOCF, LS mean²)). Additionally, a dose response was observed in the three AURN001 arms.

For the key secondary endpoint, change in CCT at six months, there was a statistically significant improvement for the high-dose AURN001 arm (p=0.012), as compared to the Y-27632-only arm (using the full analysis set population (LOCF, LS Mean)).

Separately, an improvement in patient-reported quality of life, assessed using the Visual Function Questionnaire (VFQ-25), was noted with the greatest benefit being seen in the high-dose AURN001 arm.

Doses from all five treatment arms were generally well tolerated with favorable safety profiles. There was no dose relationship observed in frequency of types of adverse events (AEs). There were no ocular serious adverse events (SAEs) reported and two non-ocular SAEs (hip fracture and femur fracture). The most frequently reported (>3%) ocular TEAEs were ocular hypertension (9.3%), conjunctival hemorrhage (5.2%), eye pain (4.1%), and cystoid macular edema (3.1%) and the most frequently reported non-ocular TEAE was COVID-19 (3.1%).

“We are thrilled with the topline results of the CLARA trial,” said Michael Goldstein, M.D., M.B.A., president and chief medical officer of Aurion Biotech. “We were especially pleased that in the high-dose AURN001 arm at six months, there was a statistically significant improvement in the primary endpoint. Based on these findings, combined with the generally favorable safety profile in the CLARA trial, we look forward to bringing the high dose of AURN001 forward into our proposed Phase 3 pivotal trials.”

“We believe that today’s news is another important step forward in the clinical development of our investigational allogeneic cell therapy, AURN001, to help restore vision,” said Greg Kunst, chief executive officer of Aurion Biotech. “We look forward to presenting full results from the CLARA trial at future medical conferences.”

Topline data from the CLARA study follows Aurion Biotech’s announcement in June 2024 that the U.S. Food and Drug Administration (FDA) granted AURN001 both Breakthrough Therapy Designation (BTD) and Regenerative Medicine Advanced Therapy (RMAT) designation, and Aurion’s commercial launch of its cell therapy in Japan, under the trade name, Vyznova^TM, in September 2024. More information about Aurion Biotech’s recent progress can be found on its website: www.aurionbiotech.com/news .

About Corneal Endothelial Dysfunction

Corneal edema secondary to corneal endothelial dysfunction is a sight-threatening condition affecting millions of people throughout the world. When corneal endothelial cells die or degrade, they do not regenerate in vivo. If left untreated, corneal endothelial cell loss can cause corneal edema (swelling) and loss of vision. Although corneal transplants are used to treat corneal endothelial dysfunction, these procedures are complex, invasive and time-consuming. In addition, it is estimated that there is only one healthy donor cornea available for every 70 diseased eyes.³ As such, there remains a significant unmet need for patients with corneal endothelial disease for alternative treatment options that are effective, straightforward and minimally invasive, without being limited by donor cornea supply.

About Aurion Biotech, Inc.

Aurion Biotech’s mission is to restore vision to millions of patients with life-changing regenerative therapies. It received the prestigious Prix Galien award for best start-up in biotech in 2022. Aurion Biotech is advancing AURN001, an investigational single administration, allogeneic cell therapy to treat corneal edema secondary to corneal endothelial dysfunction. Aurion Biotech developed and has launched the first commercially available corneal endothelial cell therapy in Japan. To learn more, visit www.aurionbiotech.com .

¹ Although the study was not powered to show statistical significance, descriptive statistical analyses are provided in this topline readout.
² LOCF: Last Observed Carry Forward statistical analysis, LS Mean: Least Squares Mean statistical analysis
³ JAMA Ophthalmology. 2016;134(2):167-173. doi:10.1001/jamaophthalmol.2015.4776.

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