Six months ahead of its original expectations, Glaukos Corporation has concluded patient enrollment and randomization for its second phase 3 confirmatory pivotal trial of Epioxa (Epi-on) for the treatment of keratoconus.
Talk about Epioxa.
Epioxa (Epi-on) is a second-generation corneal cross-linking (CXL) therapy designed to reduce treatment time and complexity as well as improve comfort and recovery time for keratoconus patients.
It follows the first-generation corneal CXL therapy of Photrexa (Epi-off), which is currently the only FDA-approved treatment for progressive keratoconus and corneal ectasia following refractive surgery.
What’s the difference between the two?
As a topical agent, Photrexa (riboflavin 5′-phosphate ophthalmic solution) 0.146% uses photo-activation to create bonds between corneal collagen fibers.
In comparison, Epioxa uses riboflavin that has been modified to help enhance penetration through the epithelium, pulsed ultraviolet-A irradiation at increase intensity, and supplemental oxygen using “boost” goggles.
Gotcha. So what happened with this first phase 3 trial?
That trial (NCT03442751) met its pre-specified primary efficacy endpoint and was confirmed by the FDA, which gave their stamp of approval for its results to support the submission and review of an eventual new drug application (NDA).
That is, pending data from this second phase 3 trial.
Now tell me about that study.
According to Glaukos, the multi-center, randomized, placebo and sham procedure-controlled phase 3 confirmatory pivotal trial (NCT05759559) enrolled 312 eyes.
The study eyes were randomized in a 2:1 ratio to receive Epioxa or a placebo/sham procedure control treatment.
The goal: to either reduce progression of, and/or reduce the maximum corneal curvature (Kmax) in the keratoconus eyes.
What’s being measured?
The study’s primary efficacy endpoint is the mean change in Kmax from baseline to Month 12.
What determines its success?
According to Glaukos, the FDA and the company have agreed to consider the trial a success if the treatment and control arm difference of the primary efficacy endpoint is statistically significant and the difference is ≥ 1.0 diopter (D).
Then what?
The company plans to use the results from both the first and secondary phase 3 trials to support an NDA submission for Epioxa by the end of 2024.