The highest dose of Aurion Biotech’s cell therapy, the main component of which recently received approval in Japan, has improved vision in a phase 1/2 trial for a type of eye disease, leading the company to set its sights on phase 3.
The study enrolled 97 people in the U.S. and Canada with corneal edema secondary to endothelial dysfunction, a disease that can currently only be treated with invasive specialty surgery. They received one of three doses of the regenerative medicine—which comprises allogeneic human corneal endothelial cells and a rho kinase inhibitor—or the rho kinase inhibitor alone or the endothelial cells alone.
The highest dose of the therapy, dubbed AURN001, hit the study’s primary endpoint of a higher proportion of responders, 50%, seeing a more than 15-letter improvement in their vision compared to the rho kinase inhibitor cohort. Aurion described the p-value of 0.02 as a “statistically significant improvement.”
The company didn’t share specific details of how the low- and medium-dose cohorts performed, although it did say that all three AURN001 arms had shown a dose-dependent response.
When it came to the key secondary endpoint of a change in best corrected visual acuity, the high dose also showed a statistically significant improvement, with a p-value of 0.002, the company said.
All three dose cohorts were “generally well tolerated with no ocular serious adverse events,” Aurion pointed out. The most common ocular treatment-emergent adverse events were ocular hypertension (9.3%), conjunctival hemorrhage (5.2%), eye pain (4.1%) and cystoid macular edema (3.1%).
“We are thrilled with the topline results of the CLARA trial,” Aurion President and Chief Medical Officer Michael Goldstein, M.D., said in the release. “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,” Goldstein added.
AURN001 replicates cells from one healthy cornea to treat up to 100 eyes, dramatically increasing supply when compared to the typical one-donor-per-recipient ratio. The therapy doesn’t require surgery, instead being delivered through a straightforward injection that can be performed by any ophthalmologist or cataract specialist. The aim is to generate a much larger base of treatment delivery options, not to mention a more patient-friendly procedure experience.
The endothelial cell portion of AURN001, known as neltependocel, received approval in Japan in September for the treatment of bullous keratopathy of the cornea. At the time, Aurion described the regulatory win for the therapy, which is marketed as Vyznova, as the world’s first approved allogeneic cell therapy for corneal endothelial disease.