Glaukos Announces Positive Phase 3 Results for Epioxa Corneal Cross-Linking Therapy

Glaukos announced that its second phase 3 confirmatory pivotal trial for Epioxa (Epi-on), a next-generation corneal cross-linking therapy for the treatment of keratoconus, has met its primary efficacy endpoint.

The trial demonstrated a statistically significant and clinically relevant improvement in maximum corneal curvature (Kmax) at 12 months compared to a placebo-controlled arm. Kmax, an FDA-accepted measure for assessing keratoconus, evaluates the steepest curvature of the cornea. The findings revealed a Kmax treatment effect of –1.0 D (P<0.0001), suggesting a reduction in the disease's progression.

“We are excited to announce these positive results, which highlight Epioxa's potential to halt or reduce the progression of keratoconus," Thomas Burns, chairman and CEO of Glaukos, said in a company news release. "We believe Epioxa could become the first FDA-approved, noninvasive treatment alternative that improves patient comfort and shortens recovery times."

The phase 3 trial enrolled 312 eyes and randomized them in a 2:1 ratio between Epioxa and a sham-controlled arm. The treatment was well-tolerated, with 91.5% of treatment patients and 90.9% of control patients completing the 12-month study. No serious adverse events were reported, and no patients discontinued the treatment due to adverse effects. There was no evidence of systemic effects or changes in corneal endothelial cell counts over the trial period.

The company plans to submit a new drug application (NDA) to the FDA by the end of 2024, using data from this second phase 3 trial in combination with results from a previously completed phase 3 study. The FDA has already confirmed that Glaukos’ first phase 3 pivotal trial, which also met its primary efficacy endpoint, would be sufficient to support an NDA submission alongside this latest trial.

Current treatments for keratoconus, such as glasses and contact lenses, address symptoms but do not halt disease progression. Glaukos’ first-generation therapy, Photrexa (Epi-off), is the only FDA-approved treatment proven to slow or stop keratoconus progression.

If approved, Epioxa could become the first FDA-approved, noninvasive cross-linking therapy.