Aurion Biotech’s Cell Therapy AURN001 Demonstrates Efficacy and Safety in Phase 1/2 ABA-1, CLARA Clinical Trial

Aurion Biotech’s AURN001 (marketed as Vyznova in Japan), a combination cell therapy and small molecule product that remains investigational in the United States, has demonstrated efficacy and safety in data from the phase 1/2 ABA-1, CLARA clinical trial (NCT06041256) assessing it as an alternative to corneal transplant for 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. In ABA-1, CLARA, patients are assigned to receive either a low (2.5x10⁵), medium (5.0x10⁵), or high (1.0x10⁶) dose of neltependocel within the combination product. In an additional arm, participants receive neltependocel alone at a dose equivalent to that used in the high dose AURN001 arm and in yet another arm participants are treated with Y-27632 alone.

Notably, a statistically significant improvement in the proportion of responding patients who achieved at least 15 letters of improvement in best corrected visual acuity (BCVA) compared to the Y-27632-only arm, which constituted the primary end point, was recorded in the high dose arm (50% of responding patients versus 14.3%, P = .02). Furthermore, using the full analysis set population (LOCF, LS mean), the high dose arm showed a statistically significant improvement in change in BCVA at 6 months compared to the Y-27632-only arm (P = .002) and in change in central corneal thickness (CCT) at 6 months compared to the Y-27632-only arm (P = .012). These constituted key secondary end points. The greatest improvement in the Visual Function Questionnaire (VFQ-25) was also observed in the high dose arm.

“We are thrilled with the topline results of the CLARA trial,” Michael Goldstein, MD, MBA, the president and chief medical officer of Aurion Biotech, said in a statement.¹ “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.”

With regard to safety, no dose relationship was seen with regard to the frequency of types of adverse events (AEs). There were no ocular serious AEs (SAEs) in any arm. The only nonocular SAEs were a hip fracture and femur fracture. Ocular hypertension (9.3%), conjunctival hemorrhage (5.2%), eye pain (4.1%), and cystoid macular edema (3.1%) were the most common ocular treatment-emergent AEs (TEAEs). The most common nonocular TEAE was COVID-19, which occurred in 3.1% of patients.

“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,” Greg Kunst, the chief executive officer of Aurion, added to the statement.¹ “We look forward to presenting full results from the CLARA trial at future medical conferences.”

CGTLive® has previously interviewed Arnaud Lacoste, PhD, the chief scientific officer of Aurion Biotech, about AURN001 and its potential to address unmet needs.

“It seems to us that a cell therapy is a much better option than typical corneal transplant for both patients and ophthalmic surgeons. 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 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. News release. Aurion Biotech, Inc. December 18, 2024. Accessed December 23, 2024. https://aurionbiotech.com/aurion-biotech-announces-positive-data-for-phase_1-2_trial/