YD312 inhibits Stem Cell Factor (SCF)/c-Kit pathway that increases vascular permeability via endothelial nitric oxide synthase (eNOS). By oral administration, it could be easily delivered to the target where it showed the therapeutic effects. YD312 can ameliorate DME, DR, and AMD by normalizing vascular permeability in retina.
YD312 is a drug that treat diabetic macular edema by normalizing vascular permeability. The mechanism of action (MOA) for YD312 is through inhibiting signal transduction of SCF/c-Kit pathways, thus inhibiting the increase in vascular permeability due to eNOS. This is the first in class drug by a new mechanism, which is quite different from existing imatinib’s MOA. Because c-kit, just like VEGF and the target of YD312, is present in the endothelial wall of retinal blood vessels, orally administered YD312 can easily reach the target without having to pass through the Blood-retina barrier (BRB) resulting in therapeutic effects. It's a fundamentally different treatment from anti-VEGF drugs and steroids that are administered through intravitreal injection. Like imatinib which is orally available and 98% bioavailable, YD312 takes much better method to deliver onto our target than partially effective eye drops and intravitreal injection.
YDB101 targets for NADH-cytochrome b5 reductase 3, Diaphorase-1 (CYB5R3). It is known to be expressed in mitochondria or cytoplasm with or without flanking transmembrane.
Examples of our target can be membrane CYB5R3 and erythrocytic CYB5R3. Membrane CYB5R3 is related to elongation of fatty acids, desaturation, synthesis of cholesterol, and drug metabolism.
And Erythrocytic CYB5R3 is related to reduction of methemoglobin. When those targets go wrong and have mutations, induction of methemoglobinemias, reduction in oxygen transfer, and cyanosis can occur.