UMIP-67 – A Novel Anti-Angiogenic Therapy for Neovascular Diseases

Problem Proliferativeretinopathies, such as diabetic retinopathy (DR) and retinopathy of prematurity(ROP), are attributed to the abnormal proliferation of vasculature within theretina; the abnormal vasculature also lacks the architecture and structuralintegrity of normal vasculature. DRaffects approximately 8 million individuals in the United States, with 80% ofpatients with a 20+ year history of diabetes being afflicted. About 10% of DR patients progress to diabetivcmacular edema, causing a loss in visual acuity, and ultimately irreversibleblindness. Current therapies for proliferativeretinopathies include anti-VEGF therapies to inhibit vascularization within theretina. Such therapies have seenmoderate benefits, but since VEGF is strongly associated with normalvascularization, indiscriminate inhibition of all vascularization has led toadverse clinical outcomes, particularly in infant ROP patients who require VEGFfor proper retinal development. Pharmaceuticaldevelopment of therapies against targets that selectively inhibit pathologic,but not normal, vascularization would be greatly beneficial for retinopathies,and could be extended into other areas, such as oncology. Technology We recently discovered secretogranin III (Scg3)as a highly disease-selective angiogenic factor; Scg3 preferentially bound toand stimulated angiogenesis of diseased but not normal vessels, whereas VEGFbound to and promoted angiogenesis of both diseased and healthy vessels. We further developed a humanized, monoclonalantibody against Scg3 and demonstrated its high therapeutic efficacy in a mousemodel of proliferative retinopathy (Fig. 1).Importantly, whereas the retinopathy-approved anti-VEGF drug afliberceptaffected kidney development in the mouse model, anti-Scg3 did not,demonstrating its selective therapeutic effect.Moreover, we discovered that Scg3 and VEGF act through at leastpartially distinct mechanisms, and combinatorial therapy exhibited asynergistic effect, which could be exploited clinically (Fig. 2). Opportunity ·Strong, worldwide intellectual property position dueto the novelty of Scg3 and anti-Scg3 ·Worldwide diabetic retinopathy market estimated at$10B by 2025 ·Potential for orphan drug and rare pediatric diseasedesignations for retinopathy of prematurity ·Potential extension of anti-Scg3 into oncology market · Looking for partner to license and develop anti-Scg3 through clinical trials and marketing Bin Yan byan@med.miami.edu 305-243-4035