Aurion cell therapy improves vision in phase 1/2 trial

Aurion Biotech announced positive topline data from its phase 1/2 clinical trial of AURN001, an allogeneic cell therapy product candidate for the treatment of corneal edema secondary to corneal endothelial dysfunction, teeing up a phase 3 trial.

AURN001 is a combination cell therapy product candidate comprised of allogeneic human corneal endothelial cells (neltependocel) and a rho kinase inhibitor (Y-27632). The treatment is administered to the anterior chamber of the eye as a one-time procedure. AURN001 has been granted Breakthrough Therapy designation and Regenerative Medicine Advanced Therapy designation, and is approved in Japan under the trade name Vyznova.

The CLARA phase 1/2 clinical trial is a prospective, double-masked, parallel-arm dose-ranging clinical trial that included 97 subjects randomized at U.S. and Canadian sites to five arms — low, medium and high dose of AURN001, neltependocel only and Y-27632 only.

For the primary endpoint — the proportion of responders with >15 letters of improvement on the visual acuity chart — dose-dependent response was observed in the three AURN001 arms, with the high-dose AURN001 arm showing statistically significant improvement compared to Y-27632-only arm.

For the key secondary endpoint — change in central corneal thickness at six months — there was a statistically significant improvement for the high-dose AURN001 arm as compared to the Y-27632-only arm.

Corneal edema secondary to corneal endothelial dysfunction is a sight-threatening condition that if left untreated, can cause corneal swelling and loss of vision. Although corneal transplants are used to treat corneal endothelial dysfunction, the procedures are complex and invasive and are faced with a shortage of healthy donor cornea.

Based on the findings, Aurion plans to bring the high dose of AURN001 forward into phase 3 pivotal trials.