Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases, today announced that the second Phase 3 confirmatory pivotal trial for Epioxa™ (Epi-on), its next-generation corneal cross-linking iLink therapy for the treatment of keratoconus, successfully met the study’s pre-specified primary efficacy endpoint, demonstrating a clinically relevant and statistically significant improvement in maximum corneal curvature (Kmax) at 12 months from baseline between the Epioxa treated arm and the sham/placebo-controlled arm. Kmax is a U.S. FDA-accepted primary efficacy outcome for keratoconus pivotal trials and an objective measurement of the steepest corneal curvature based on corneal topography, where an increasing Kmax denotes corneal steepening and keratoconus disease progression.
“We are excited to announce these positive Phase 3 confirmatory results that met the study’s primary efficacy endpoint and once again demonstrated the potential of Epioxa to halt or reduce the advancement of keratoconus, a progressive, sight-threatening corneal disease. These results further underscore our view that Epioxa may provide the ophthalmic community and keratoconus patients with the first FDA-approved, non-invasive, bio-activated drug treatment alternative designed to reduce procedure time, improve patient comfort and shorten recovery time,” said Thomas Burns, Glaukos chairman and chief executive officer. “We appreciate the commitment and dedication of the clinical investigators, who play a vital role in bringing new innovations to patients suffering from keratoconus and at risk for significant vision loss. We expect these data to support our anticipated NDA targeted for FDA submission by the end of this year.”
Topline summary results and observations from the Phase 3 confirmatory pivotal trial are as follows:
- The Epioxa Phase 3 clinical trial successfully achieved its primary efficacy outcome by demonstrating a Kmax treatment effect of –1.0 diopter (D) (p <0.0001), determined as prospectively defined least square mean Kmax change from baseline in the Epioxa treated arm versus the sham/placebo-controlled arm at the Month 12 study endpoint.
- The treatment was generally well-tolerated, with 91.5% of enrolled treatment patients completing the 12-month trial, compared to 90.9% of enrolled control patients. No patients randomized to Epioxa treatment discontinued early due to an adverse event and there were no ocular serious adverse events reported. The majority of adverse events reported were mild and transient in nature. There was no evidence of treatment-related systemic effects reported in the study and there was no change in corneal endothelial cell counts over the 12-month evaluation period.
Read the full press release here.