STG-001

STG-001 is an investigational small molecule drug developed by Stargazer Pharmaceuticals for the treatment of Stargardt disease, a rare inherited retinal degenerative disorder that leads to progressive central vision loss and legal blindness. The drug acts as an indirect visual cycle modulator by reducing plasma concentrations of retinol binding protein 4 (RBP4) and vitamin A, thereby modulating the visual cycle and decreasing the accumulation of cytotoxic retinoids in the eye. This mechanism aims to reduce retinal damage and preserve vision in patients with Stargardt disease. STG‑001 has received Orphan Drug Designation in both the US and EU, and has reached Phase II clinical trials[2][3][4][5][7][8].

Other names

3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-pyrazol-1-yl)propanoic acidIK88NC17ODIK-88NC17ODIK 88NC17ODUNII-IK88NC17ODUNII-IK-88NC17ODUNII-IK 88NC17ODCHEMBL4650305CHEMBL-4650305CHEMBL 4650305