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KHK4951 is an investigational ophthalmic formulation of tivozanib, a small-molecule vascular endothelial growth factor receptor (VEGFR) tyrosine kinase inhibitor. It is developed as a novel nano-crystalized eye drop designed to efficiently deliver the drug to posterior ocular tissues. The primary indications under investigation are neovascular (wet) age-related macular degeneration (nAMD) and diabetic macular edema (DME). The mechanism of action involves inhibition of VEGFR-1, -2, and -3 signaling pathways, thereby reducing abnormal blood vessel growth and leakage in the retina[2][4][7]. This approach aims to provide a less invasive alternative to current standard-of-care intravitreal injections.
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