Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on innovative therapies for glaucoma, corneal disorders, and retinal diseases, has announced positive results from the second Phase 3 confirmatory trial for Epioxa™ (Epi-on). This next-generation corneal cross-linking therapy for keratoconus successfully met its primary efficacy endpoint, showing a clinically relevant and statistically significant improvement in maximum corneal curvature (Kmax) after 12 months.
Kmax, an objective measurement of the steepest corneal curvature derived from corneal topography, is an FDA-accepted primary efficacy outcome for keratoconus pivotal trials. An increase in Kmax indicates corneal steepening and disease progression in keratoconus.
“We are thrilled to share these positive Phase 3 results, which met the study’s primary efficacy endpoint and demonstrated Epioxa’s potential to halt or reduce the progression of keratoconus, a progressive and sight-threatening corneal disease,” said Thomas Burns, Glaukos CEO. “These findings reinforce our belief that Epioxa could offer the ophthalmic community and keratoconus patients the first FDA-approved, non-invasive, bio-activated drug treatment that reduces procedure time, enhances patient comfort, and shortens recovery time. We are grateful to the clinical investigators for their commitment to advancing treatment options for keratoconus patients facing significant vision loss. We expect these results to support our planned NDA submission to the FDA by year-end.”
Trial Results Summary:
- The Phase 3 trial achieved its primary efficacy endpoint, showing a Kmax treatment effect of –1.0 diopter (D) (p < 0.0001) based on the mean change from baseline in the Epioxa-treated group compared to the sham/control group at the 12-month mark.
- The treatment was generally well-tolerated, with 91.5% of patients in the Epioxa group completing the trial, compared to 90.9% in the control group. No patients discontinued due to adverse events, and no serious ocular adverse events were reported. Most adverse events were mild and transient.
- The multi-center, randomized, placebo- and sham-controlled trial included 312 eyes and aimed to evaluate the safety and efficacy of Epioxa in slowing or reducing Kmax in progressive keratoconus. The study was designed to be considered successful based on a special protocol assessment (SPA) agreement with the FDA, requiring a statistically significant difference of at least 1.0 D between the treatment and control arms.
The results from this second Phase 3 trial, combined with those from the earlier Phase 3 trial, are expected to support Glaukos’ NDA submission for Epioxa by the end of 2024. The FDA has confirmed that the data from the first trial would be adequate for submission alongside the new results.
Glaukos recently held a successful pre-NDA meeting with the FDA regarding Epioxa. The meeting aimed to confirm the proposed NDA content and ensure that the clinical data package, including both completed Phase 3 trials, was sufficient for review.
Glaukos’ corneal cross-linking iLink therapies utilize proprietary, bio-activated, single-use drug formulations to strengthen corneal tissue and halt keratoconus progression. Typically diagnosed in teenagers, keratoconus leads to thinning and weakening of the cornea, which, if untreated, can result in vision loss and is a leading cause of corneal transplants in the U.S. Approximately 90% of keratoconus cases are bilateral, with up to 20% of patients ultimately requiring a corneal transplant. Traditional treatments like glasses and contact lenses only address symptoms, whereas Glaukos’ first-generation iLink therapy, Photrexa® (Epi-off), is the first FDA-approved treatment shown to slow disease progression.
Epioxa aims to preserve the corneal epithelium, reduce procedure times, enhance patient comfort, and shorten recovery periods. Its proprietary formulation is designed to penetrate the epithelial layer, utilize a stronger UV-A irradiation protocol, and deliver increased supplemental oxygen for enhanced cross-linking. If approved, Epioxa would become the first FDA-approved, non-invasive corneal cross-linking therapy that does not require removal of the corneal epithelium.
About Glaukos
Glaukos (www.glaukos.com) is an ophthalmic pharmaceutical and medical technology company focused on developing innovative therapies for glaucoma, corneal disorders, and retinal diseases. Glaukos pioneered Micro-Invasive Glaucoma Surgery (MIGS) as an alternative to traditional glaucoma treatments, launching its first MIGS device in 2012. In 2024, Glaukos began commercial activities for iDose® TR, a long-duration, intracameral procedural pharmaceutical for continuous glaucoma treatment. The company also markets the only FDA-approved corneal cross-linking therapy using a proprietary bio-activated pharmaceutical for keratoconus. Glaukos continues to advance a robust pipeline of novel technologies aimed at improving care and outcomes for patients with chronic eye diseases.
