Emmecell announces topline results from Phase 1 extension study of EO2002 for corneal edema

(Image credit: Adobe Stock/Anastasiia)

Emmecell announced promising topline results from its U.S. multicenter Phase 1 extension study evaluating EO2002, a nonsurgical cell therapy for corneal edema.

The company noted that its randomized, double-masked trial demonstrated improvements in vision and corneal health, along with a strong safety profile.¹

Patients who received 150,000 endothelial cells exhibited an average improvement of 11 letters in Best Corrected Visual Acuity (BCVA) at 6 months. Notably, 38% of these patients achieved a vision gain of 15 letters or more, meeting a key regulatory benchmark.

Across all tested dose levels—150,000, 500,000, and 1 million cells—participants experienced BCVA improvements and reductions in central corneal thickness (CCT), underscoring the therapy's broad efficacy.¹

Jeffrey L. Goldberg, MD, PhD, co-founder of Emmecell and professor and chair of ophthalmology at Stanford University, expressed enthusiasm about the trial results.

“These findings strongly support advancing EO2002 into Phase 3 trials, bringing us closer to providing a transformative, nonsurgical treatment for patients with corneal edema,” he said in a statement.

Emmecell's Magnetic Cell Delivery technology enables precise placement of endothelial cells onto the posterior cornea, promoting integration and enhancing visual outcomes. The therapy also demonstrated a favorable safety profile, with no ocular or treatment-related serious adverse events (SAEs) reported among the 30 trial participants.¹

Potential for eye health

Roger A. Goldberg, MD, MBA, co-founder and chief medical advisor of Emmecell, highlighted that, in addition to vision improvements, the study showed critical structural benefits.

“EO2002 not only delivered robust functional improvements but also demonstrated anatomic biomarkers of efficacy, such as reduced central corneal thickness and increased endothelial cell density,” Goldberg said in a statement. “Remarkably, a subset of patients treated at an advanced center showed a nearly 50% reduction in central guttae—suggesting true disease modification for conditions like Fuchs dystrophy."

EO2002 is a first-in-class therapy designed to address a significant unmet need in corneal care. Currently, treating corneal edema often necessitates invasive and high-risk procedures such as corneal transplants, which are limited by donor availability. By offering a nonsurgical alternative, EO2002 can potentially treat hundreds of eyes from a single donor cornea, representing a transformative advancement in the standard of care.

"The ability to treat corneal edema without surgery is a game-changer for patients," said Ellen Koo, MD, professor of ophthalmology at the Bascom Palmer Eye Institute, University of Miami, and an investigator in the study. "With EO2002, we could significantly expand access to treatment and improve outcomes for a larger population."

A growing need

In the United States, approximately 50,000 corneal transplants are performed annually, yet many patients remain untreated due to the invasive nature of surgery and the scarcity of donor tissue. EO2002 offers a revolutionary alternative, combining safety, efficacy, and scalability to address this substantial need.¹

Emmecell plans to share detailed results at upcoming medical conferences and is preparing to advance EO2002 into pivotal Phase 3 trials. The company remains committed to driving innovation in the treatment of corneal and retinal diseases, leveraging its expertise to enhance vision and improve quality of life for patients worldwide.

Reference

1. Emmecell. Emmecell Announces Positive Topline Results from Randomized, Double-masked Trial of Groundbreaking Non-surgical Cell Therapy for Corneal Edema. Prnewswire.com. Published November 18, 2024. Accessed November 18, 2024. https://www.prnewswire.com/news-releases/emmecell-announces-positive-topline-results-from-randomized-double-masked-trial-of-groundbreaking-non-surgical-cell-therapy-for-corneal-edema-302307751.html