OCU200 is a biologic product candidate in preclinical development for treating severely sight-threatening diseases like Diabetic Macular Edema (DME), Diabetic Retinopathy (DR), and Wet Age-Related Macular Degeneration (Wet-AMD). Patients affected by these diseases share common symptoms, such as blurriness in vision and progressive vision loss as the disease progresses. The formation of fragile and leaky new blood vessels leads to fluid accumulation in and around the retina, causing damage to vision.
OCU200 is a novel fusion protein consisting of two human proteins, tumstatin and transferrin, that are already present normally in retinal tissues. OCU200 possesses unique features which enable it to (a) efficiently target leaky blood vessels, (b) regress the existing abnormal blood vessels, and (c) inhibit the growth of new blood vessels in the retina and choroid. Tumstatin, which acts as an anti-VEGF, anti-inflammatory and anti-oxidative agent, is the active component of OCU200. It binds to integrin receptors, which play a crucial role in disease pathogenesis. Transferrin facilitates the targeted delivery of tumstatin into the retina and choroid and potentially helps increase the interaction between tumstatin and integrin receptors.
In preclinical studies, OCU200 demonstrated efficacy in different animal models of neovascularization. In an animal model for DME and DR (Oxygen-induced retinopathy in mice), OCU200 demonstrated comparable efficacy at significantly lower dose compared to existing approved anti-VEGF therapy in preventing disease manifestation and progression. In animal models for wet AMD (laser induced CNV in mice and rats), OCU200 demonstrated comparable or slightly better activity compared to anti-VEGF control groups in preventing the formation and growth of new leaky blood vessels and subsequent disease symptoms.
OCU200 Demonstrated Efficacy in Animal Models for DME, DR, and Wet-AMD
About Diabetic Macular Edema and Diabetic Retinopathy
Diabetic macular edema and diabetic retinopathy are the most common vision-threatening diseases occurring in diabetic patients. Approximately 7.7 million people are affected with DR and approximately 745,000 with DME in the United States. These numbers are expected to further increase as the number of diabetic people due to disease management and life-style-related changes increase.
There are limited treatment options available for DR and DME patients and there is a need for the development of safe and effective therapy. Currently, approved anti-VEGF therapy does not work effectively in approximately 50% of DME patients. These approved therapies target only one pathway associated with DR and DME, either angiogenesis with anti-VEGF therapy or inflammation in case of steroid therapy. The development of a therapeutic which targets a multiple causative pathway of DR and DME, such as angiogenesis, oxidation, and inflammation, would offer the best treatment option for all these patients.
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.
Current FDA approved therapeutics for wet AMD include intravitreal injection of either Lucentis® or Eylea®, which target VEGF. Bevacizumab (Avastin™), the parent antibody from which ranibizumab was derived, is also used as an off-label treatment. Though these products have been effective in mitigating the disease symptoms, they have substantial limitations as demonstrated in clinical studies. For example, a significant percentage of patients do not respond to therapy and experience continuous deterioration of their vision. Additionally, the long-term, repeated dosing of anti-VEGF therapy results in reduced effectiveness and approximately 30-50% patients continue to show fluid persistence in the subretinal space, even after 1-2 years of treatment.
OCU200 possess unique characteristics to target pathways in all three of these disease areas and has the potential to offer better treatment options for all patients.
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