MA-01

MA-01 is a novel small molecule derivative of the cysteinyl leukotriene receptor 1 (CysLTR1) antagonist montelukast. Developed for the treatment of early-stage diabetic retinopathy (DR), MA-01 exhibits higher binding affinity for the CysLTR1 receptor compared to its parent compound. In preclinical studies, it has demonstrated the ability to reduce inflammation-driven effects in human retinal microvascular endothelial cells (HREC), specifically lowering levels of inflammatory markers such as IL1β, CXCL10, VCAM1, and CCL2. In vivo mouse models of streptozotocin-induced diabetes have shown that MA-01 provides sustained neuroprotection and prevents visual impairment over a three-month period by stabilizing the blood-retinal barrier and reducing endothelial permeability. The drug is being evaluated for both systemic and local intravitreal delivery.

Other names

montelukast analog MA-01montelukast derivative MA-01