AURN001 yields positive BCVA, corneal thickness outcomes in phase 1/2 trial

Key takeaways:

• For the primary endpoint, there was a dose-dependent response.
• The safety profile was favorable, with no ocular serious adverse events.

AURN001, an allogeneic cell therapy candidate, achieved positive results in a phase 1/2 clinical trial for the treatment of corneal edema secondary to corneal endothelial dysfunction, according to a press release from Aurion Biotech.

“Topline results of the CLARA trial illustrate that even in patients with less severe disease (typical of those in the U.S.), our product candidate AURN001 has the potential to significantly improve visual acuity and central corneal thickness,” Michael Goldstein, MD, MBA, president and chief medical officer of Aurion, told Healio.

AURN001 combines allogeneic human corneal endothelial cells, called neltependocel, and Rho kinase inhibitor Y-27632.

The prospective, multicenter, randomized, double-masked, parallel-arm dose-ranging CLARA trial randomly assigned 97 patients to treatment with a high cell dose (1 × 10⁶ neltependocel), medium cell dose (5 × 10⁵ neltependocel) or low cell dose (2.5 × 10⁵ neltependocel) and 100 µm Y-27632, 100 µm Y-27632 alone or 1 × 10⁶ neltependocel alone. The primary endpoint was the proportion of participants who experienced a best corrected visual acuity improvement of 15 letters or more at 6 months from baseline, and secondary endpoints included change in BCVA and central corneal thickness from baseline to 6 months as well as safety and tolerability.

According to the release, there was a dose-dependent response in the AURN001 arms, with 50% of patients in the high-dose arm meeting the primary endpoint (P = .020) vs. 14.3 % in the Y-27632-only arm. Additionally, there was a statistically significant improvement in BCVA at 6 months in the high-dose AURN001 arm (P = .002) vs. the Y-27632-only arm, with dose responses observed in all three AURN001 arms.

The high-dose arm also experienced a statistically significant improvement in central corneal thickness compared with the Y-27632-only arm (P = .012).

The safety profile in all five arms was favorable, with no ocular serious adverse events reported.

“These data add to our growing body of clinical data that supports the potential of our allogeneic cell therapy to positively impact the lives of patients suffering from corneal endothelial Dysfunction,” Goldstein told Healio.

As Healio previously reported, the FDA granted breakthrough therapy and regenerative medicine advanced therapy designations for AURN001 in June 2024.

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