MP-004

MP-004 is a neuroprotective small molecule developed by Miramoon Pharma to restore intracellular calcium (Ca2+) balance in stressed cells. It acts by stabilizing the interaction between the ryanodine receptor (RyR) and its regulatory protein FKBP12, thereby preventing pathological calcium leakage from the sarcoplasmic or endoplasmic reticulum. The drug also exhibits dual activity by reducing oxidative stress. Formulated as a topical eye drop for retinal indications, MP-004 is designed to reach the retina non-invasively. It has received Orphan Drug Designation from the FDA and EMA for the treatment of retinitis pigmentosa and is being explored for a variety of other conditions, including diabetic retinopathy, age-related macular degeneration, muscular dystrophies, and neurodegenerative diseases like ALS and Alzheimer's.