Ocugen Begins Phase 1 Trial of OCU200 for Diabetic Macular Edema

In MALVERN, PA, USA, on January 16, 2025, Ocugen, Inc., a biotechnology firm, declared the commencement of its OCU200 Phase 1 clinical trial, marking a significant development in the treatment of diabetic macular edema (DME). This trial represents a potential breakthrough in managing not only DME but also diabetic retinopathy (DR) and wet age-related macular degeneration (wet AMD), due to OCU200's innovative method of action. The drug, by binding its active ingredient, tumstatin, to integrin receptors, addresses core issues of these diseases.

Dr. Shankar Musunuri, Chairman, CEO, and Co-Founder of Ocugen, highlighted the promising implications of OCU200 for DME patients, especially the 30-40% who do not respond to current anti-VEGF treatments. The aim of the ongoing Phase 1 trial is to evaluate the safety of OCU200 through a structured, multicenter, open-label study. This involves a dose-escalating strategy with three distinct groups receiving varying doses: low (0.025 mg), medium (0.05 mg), and high (0.1 mg). Each participant will have two intravitreal injections spaced six weeks apart, with monitoring continuing for up to three months post the final injection.

The prevalence of DME, DR, and wet AMD is notable, affecting approximately 12 million individuals in the United States and 130 million globally. These conditions commonly lead to symptoms like blurred vision and progressive vision loss. They are characterized by the formation of frail, leaky blood vessels that contribute to fluid build-up in the retina, ultimately harming eyesight.

Dr. David Almedia, a Vitreoretinal Surgeon and Clinician Scientist, noted an increase in vision-related issues linked to diabetes at his clinic. He expressed optimism about providing a new treatment option for patients with these conditions, given the limitations of existing anti-VEGF therapies. The demand for improved treatment options remains high.

OCU200 is distinctly formulated as a recombinant fusion protein composed of two linked components: tumstatin, which serves as an anti-inflammatory, anti-VEGF agent, and transferrin, which directs the drug to the choroid and retina. This dual-action aims to diminish vascular permeability, inflammation, and neovascularization that are common in the pathophysiology of DME, DR, and wet AMD. Importantly, this approach may achieve effectiveness at significantly lower doses than current therapies.

Dr. Huma Qamar, Chief Medical Officer at Ocugen, shared enthusiasm for the trial's progression. There is significant interest in not just the safety data but also early indications of efficacy as the trial advances. OCU200 stands as a novel biologic candidate within Ocugen's ophthalmology portfolio, with a strong focus on combating blindness-inducing diseases.

Ocugen is working towards gaining approval for OCU200 as a primary treatment option for DME, DR, and wet AMD. As a company, Ocugen is dedicated to the discovery, development, and commercialization of innovative gene and cell therapies, biologics, and vaccines. Their efforts aim to transform health outcomes and instill hope for patients worldwide through pioneering scientific advancements and strategic innovations.