Glaukos Announces Positive Topline Outcomes in Phase 3 Confirmatory Trial for Epioxa™, Achieving Primary Efficacy Endpoint and Demonstrating Favorable Tolerability and Safety

Rhea-AI Summary

Glaukos (NYSE: GKOS) announced positive topline results from its Phase 3 confirmatory trial for Epioxa™, a next-generation corneal cross-linking therapy for keratoconus. The trial met its primary efficacy endpoint, demonstrating a clinically relevant and statistically significant improvement in maximum corneal curvature (Kmax) at 12 months. Key findings include:

1. A Kmax treatment effect of –1.0 diopter (p <0.0001)
2. 91.5% of treated patients completed the 12-month trial
3. No treatment-related serious adverse events reported
4. No evidence of systemic effects or changes in corneal endothelial cell counts

Glaukos plans to submit a New Drug Application (NDA) to the FDA by the end of 2024. If approved, Epioxa could become the first FDA-approved, non-invasive drug therapy for keratoconus that preserves the corneal epithelium.

Positive

• Phase 3 confirmatory trial met primary efficacy endpoint with statistically significant improvement in Kmax
• High patient retention rate of 91.5% in the treatment arm
• No serious adverse events or systemic effects reported
• FDA agreed that the clinical data package is sufficient for NDA submission
• Potential to be the first FDA-approved, non-invasive drug therapy for keratoconus

Negative

• None.

Insights

Medical Research Analyst

This Phase 3 trial for Epioxa in keratoconus treatment is highly significant. The study met its primary efficacy endpoint with a -1.0 diopter treatment effect (p<0.0001) in maximum corneal curvature (Kmax) at 12 months. This demonstrates Epioxa's ability to halt or reduce keratoconus progression.

Key points:

• High patient retention (91.5% completion rate)
• No treatment discontinuations due to adverse events
• No ocular serious adverse events reported
• Majority of adverse events were mild and transient
• No evidence of systemic effects or changes in corneal endothelial cell counts

If approved, Epioxa could become the first FDA-approved, non-invasive drug therapy for keratoconus that preserves the corneal epithelium. This could significantly improve patient comfort and recovery time compared to current treatments. The positive results support Glaukos' plan to submit a New Drug Application (NDA) by the end of 2024, potentially bringing a novel treatment option to market for this progressive, sight-threatening disease.

Financial Analyst

These positive Phase 3 results for Epioxa are a significant milestone for Glaukos, potentially opening up a lucrative new market. Keratoconus affects approximately 1 in 2,000 people, representing a sizeable patient population. As the first non-invasive, FDA-approved treatment, Epioxa could capture a substantial market share.

Key financial implications:

• Potential for high-margin drug sales upon FDA approval
• Expanded addressable market beyond current invasive treatments
• Possible increase in recurring revenue from ongoing patient treatments
• Strengthened competitive position in the ophthalmology market

Investors should note that while the NDA submission is planned for end of 2024, the approval process and potential market launch will take additional time. However, this news significantly de-risks the Epioxa program and could drive near-term stock appreciation. Long-term, successful commercialization could materially impact Glaukos' revenue growth and profitability.

Phase 3 Confirmatory Trial Met Pre-Specified Primary Efficacy Endpoint and Demonstrated Excellent Tolerability and a Favorable Safety Profile Through 12 Months, Supporting Anticipated New Drug Application (NDA) Submission by End of 2024

Data Suggest Epioxa Has the Potential to Provide the Ophthalmic Community and Patients with the First FDA-Approved, Non-Invasive Drug Therapy Alternative for Keratoconus

ALISO VIEJO, Calif.--(BUSINESS WIRE)-- 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.

The multi-center, randomized, placebo and sham controlled Phase 3 confirmatory pivotal trial randomized 312 eyes and was designed to evaluate the safety and efficacy of Glaukos’ Epioxa therapy in impeding the progression of, and/or reducing Kmax, in eyes with progressive keratoconus. The study eyes were randomized in a 2:1 ratio to receive Epioxa therapy or placebo and sham procedure control treatment. The study’s primary efficacy endpoint was the mean change in Kmax from baseline to Month 12. Based on a special protocol assessment (SPA) agreement with the U.S. FDA, the study was to be considered a success if the difference between the treatment and control arm in the primary efficacy endpoint is statistically significant and the difference is ≥ 1.0 D.

Results from this second Phase 3 confirmatory pivotal trial together with the already-completed first Phase 3 pivotal trial are expected to support Glaukos’ anticipated NDA submission for Epioxa by the end of 2024. As a reminder, the U.S. FDA has confirmed Glaukos’ first Phase 3 pivotal trial for Epioxa, which met the pre-specified primary efficacy endpoint, would be adequate to support the submission and review of an eventual NDA, in conjunction with this second Phase 3 trial.

Glaukos recently completed a successful clinical pre-NDA meeting with the U.S. FDA regarding Epioxa as a novel treatment for keratoconus. The purpose of the meeting was to obtain agreement from the FDA on the content of the proposed NDA, and in particular, to confirm that the company’s clinical data package, including the two completed Phase 3 pivotal studies, would be sufficient to support an NDA submission and review. As an outcome of this pre-NDA meeting, the FDA agreed that the proposed clinical data package is sufficient to support an NDA submission and review.

Glaukos’ corneal cross-linking iLink therapies use proprietary, bio-activated, single-use drug formulations to strengthen corneal tissue and halt progression of keratoconus. Typically diagnosed in a patient’s teenage years, keratoconus is a debilitating eye condition characterized by progressive thinning and weakening of the cornea. If left untreated, keratoconus can lead to loss of vision and even blindness and is one of the leading causes of corneal transplant (penetrating keratoplasty) in the United States. Approximately 90% of cases of keratoconus are bilateral and as many as 20% of patients ultimately require a corneal transplant. Conventional keratoconus treatments such as eyeglasses or contact lenses address symptoms but Glaukos’ first-generation iLink therapy, known as Photrexa^®, or Epi-off, is the first and only FDA-approved therapy that has been shown to slow or halt disease progression. There are more than 300 peer-reviewed publications supporting the performance and safety of Glaukos’ iLink therapy.

Epioxa, which is designed to preserve the corneal epithelium, reduce procedure times, improve patient comfort and shorten recovery time, utilizes a proprietary, novel drug formulation designed to penetrate the epithelial layer of the cornea, a stronger UV-A irradiation protocol and the ability to deliver increased levels of supplemental oxygen to enhance cross-linking. If approved, the company anticipates Epioxa would be the first FDA-approved, non-invasive corneal cross-linking therapy that does not require removal of the corneal epithelium, the outermost layer of the front of the eye.

About Glaukos

Glaukos (www.glaukos.com) is an ophthalmic pharmaceutical and medical technology company focused on developing and commercializing novel therapies for the treatment of glaucoma, corneal disorders and retinal diseases. Glaukos first developed Micro-Invasive Glaucoma Surgery (MIGS) as an alternative to the traditional glaucoma treatment paradigm, launching its first MIGS device commercially in 2012. In 2024, Glaukos commenced commercial launch activities for iDose® TR, a first-of-its-kind, long-duration, intracameral procedural pharmaceutical designed to deliver 24/7 glaucoma drug therapy inside the eye for extended periods of time. Glaukos also markets the only FDA-approved corneal cross-linking therapy utilizing a proprietary bio-activated pharmaceutical for the treatment of keratoconus, a rare corneal disorder. Glaukos continues to successfully develop and advance a robust pipeline of novel, dropless platform technologies designed to meaningfully advance the standard of care and improve outcomes for patients suffering from chronic eye diseases.

Forward-Looking Statements

All statements other than statements of historical facts included in this press release that address activities, events or developments that we expect, believe or anticipate will or may occur in the future are forward-looking statements. Although we believe that we have a reasonable basis for forward-looking statements contained herein, we caution you that they are based on current expectations about future events affecting us and are subject to risks, uncertainties and factors relating to our operations and business environment, all of which are difficult to predict and many of which are beyond our control, that may cause our actual results to differ materially from those expressed or implied by forward-looking statements in this press release. These potential risks and uncertainties include, without limitation, the extent to which we may obtain regulatory approval for Epioxa or other investigational products, our ability to successfully commercialize such products, and the continued efficacy and safety profile of our products when commercially marketed as compared to their pre-approval clinical trial results. Historical, current and forward-looking sustainability-related statements may be based on standards for measuring progress that are still developing, internal controls and process that continue to evolve, and assumptions that are subject to change in the future. The information included in, and any issues identified as material for purposes of this document may not be considered material for SEC reporting purposes. In the context of this disclosure, the term “material” is distinct from, and should not be confused with, such term as defined for SEC reporting purposes. These and other risks, uncertainties and factors related to Glaukos, and our business are described in detail under the caption “Risk Factors” and elsewhere in our Annual Report on Form 10-Q for the quarter ended June 30, 2024, which was filed with the Securities and Exchange Commission (SEC) on August 2, 2024. Our filings with the SEC are available in the Investor Section of our website at www.glaukos.com or at www.sec.gov. In addition, information about the risks and benefits of our products is available on our website at www.glaukos.com. All forward-looking statements included in this press release are expressly qualified in their entirety by the foregoing cautionary statements. You are cautioned not to place undue reliance on the forward-looking statements in this press release, which speak only as of the date hereof. We do not undertake any obligation to update, amend or clarify these forward-looking statements whether as a result of new information, future events or otherwise, except as may be required under applicable securities law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20241016373490/en/

Media Contact:

Michele Gray

(917) 449-9250

michele@mgraycommunications.com

Investor Contact:

Chris Lewis

Vice President, Investor Relations & Corporate Affairs

(949) 481-0510

clewis@glaukos.com

Source: Glaukos Corporation

FAQ

What was the primary efficacy endpoint in Glaukos' Phase 3 trial for Epioxa (GKOS)?

The primary efficacy endpoint was the mean change in maximum corneal curvature (Kmax) from baseline to Month 12, with a treatment effect of –1.0 diopter (p <0.0001) achieved.

When does Glaukos (GKOS) plan to submit the New Drug Application for Epioxa?

Glaukos plans to submit the New Drug Application (NDA) for Epioxa to the FDA by the end of 2024.

What are the potential advantages of Epioxa over current keratoconus treatments (GKOS)?

Epioxa is designed to preserve the corneal epithelium, reduce procedure times, improve patient comfort, and shorten recovery time compared to current treatments. It could be the first FDA-approved, non-invasive drug therapy for keratoconus.

How did the safety profile of Epioxa look in the Phase 3 trial (GKOS)?

Epioxa demonstrated a favorable safety profile with no serious adverse events reported, no evidence of treatment-related systemic effects, and no change in corneal endothelial cell counts over the 12-month evaluation period.