OCU200 is a novel fusion protein consisting of two naturally occurring molecules that are present normally in retinal tissues. Tumstatin is the active component of OCU200 and plays a pivotal role in regulating ocular neovascularization through the inhibition of several key signaling pathways, including VEGF. Importantly, tumstatin selectively targets the actively dividing epithelial cells of expanding blood vessels, leaving healthly existing vasculature unharmed. Transferrin is a protein involved in regulating iron levels and it binds to a receptor that is highly expressed in the retina, particularly by deeper retinal pigment epithelial cells and in the sub-retinal choroidal layer, where wet-AMD first occurs before spreading inward. The fusion protein containing transferrin and tumstatin enables the selective anti-neovascular activity of tumstatin to penetrate and become concentrated within the areas of the retina that are primarily impacted by early neovascular disease.
In preclinical studies, OCU200 demonstrated superior efficacy compared to tumstatin alone and anti-VEGF therapy in reducing new blood vessel formation. These encouraging results highlight the potential for OCU200 to deliver true disease modification for wet-AMD and other high-need ocular neovascular diseases.
Ocugen is continuing to advance OCU200 in late-preclinical studies and expects to commence human clinical trials as a treatment for wet-AMD in 2019.
About 10-15% of patients with age-related macular degeneration (AMD) progress to the advanced “wet” form, which is characterized by penetration of abnormal blood vessels in the retina that leak blood and proteins. The result can be irreversible damage to photoreceptor cells and rapid, severe vision loss, particularly in the center of the field of vision, causing significant functional impairment. Wet-AMD accounts for 90% of all AMD-related blindness.
Market-leading drugs used to treat wet-AMD include anti-VEGF injections Eylea® and Lucentis®, to which only about a third of wet-AMD patients respond initially. Such treatments are also associated with rebound neovascularization, macular atrophy, and in some cases, severe adverse reactions. There remains significant need for new therapies that are rapidly effective in a larger patient population and can have a significant and lasting impact on the progression of wet-AMD.
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