Emmecell Reports Positive Results from Trial of Non-surgical Cell Therapy for Corneal Edema

Emmecell, a clinical-stage biotechnology firm based in Menlo Park, California, has announced promising topline results from its U.S. multi-center Phase 1 extension study of EO2002, a non-surgical cell therapy designed to treat corneal edema. This randomized, double-masked trial has shown notable enhancements in both vision and corneal health, coupled with a strong safety profile.

In the cohort treated with 150,000 endothelial cells, patients exhibited a significant mean gain of 11 letters in Best Corrected Visual Acuity (BCVA) at the six-month mark. Impressively, 38% of these participants achieved a vision improvement of at least 15 letters, meeting an important regulatory benchmark. Across all tested doses—150,000, 500,000, and 1 million cells—subjects demonstrated enhancements in BCVA and reductions in central corneal thickness (CCT), indicating the wide-ranging efficacy of the therapy.

Jeffrey L. Goldberg, MD, PhD, co-founder of Emmecell and a professor and chair of ophthalmology at Stanford University, expressed enthusiasm about the trial outcomes. According to Goldberg, the results provide a strong foundation for advancing EO2002 into Phase 3 clinical trials, moving closer to offering a groundbreaking, non-surgical treatment option for patients suffering from corneal edema.

Emmecell's unique Magnetic Cell Delivery® technology facilitates the precise placement of endothelial cells into the posterior cornea, aiding their integration and contributing to improved visual outcomes. The safety profile of EO2002 was also positive, with no ocular or treatment-related serious adverse events (SAEs) reported among the 30 trial participants.

Beyond vision improvements, the study highlighted crucial structural benefits. Roger A. Goldberg, MD, MBA, co-founder and Chief Medical Advisor of Emmecell, pointed out that EO2002 not only achieved robust functional gains but also indicated anatomic markers of efficacy, such as decreased central corneal thickness and enhanced endothelial cell density. Remarkably, some patients treated at an advanced center experienced nearly a 50% reduction in central guttae, suggesting potential disease modification for conditions like Fuchs dystrophy.

EO2002 stands out as a pioneering therapy with the potential to meet a significant unmet need. Currently, corneal edema often necessitates invasive and risky surgical procedures, including corneal transplants, which are constrained by donor availability and other limitations. EO2002's non-surgical method offers the potential to treat numerous eyes from a single donor cornea, potentially changing the standard approach to care.

Ellen Koo, MD, a professor of ophthalmology at the Bascom Palmer Eye Institute, University of Miami, and a study investigator, emphasized the revolutionary impact of EO2002. She noted that the ability to treat corneal edema without surgery could significantly increase access to treatment and improve outcomes for a larger patient population.

In the United States alone, around 50,000 corneal transplants are performed each year, leaving many patients untreated due to the invasive nature of the surgery or limited availability of donor tissue. EO2002 offers a novel alternative, combining safety, efficacy, and scalability to meet this considerable need.

Emmecell intends to present detailed findings at upcoming medical conferences and is eager to advance EO2002 into pivotal Phase 3 trials. The company is dedicated to pioneering advancements in the treatment of corneal and retinal diseases, utilizing its expertise to enhance vision and quality of life for patients globally.

Emmecell, established as a clinical-stage biotechnology enterprise, focuses on developing advanced cell therapies for eye diseases. Through proprietary methods like Magnetic Cell Delivery®, the company is reshaping the treatment landscape for conditions such as corneal edema and retinal disorders, offering safe, effective, and non-surgical solutions.