PLX-100

**PLX-100** is a preclinical oral combination therapy developed by Polaryx Therapeutics for neuronal ceroid lipofuscinosis, also known as Batten disease. It combines gemfibrozil, a peroxisome proliferator-activated receptor alpha agonist, with all-trans retinoic acid, a retinoid-pathway agonist. The program is intended to enhance transcription factor EB-mediated lysosomal biogenesis, increase lysosomal function including tripeptidyl peptidase 1 expression, reduce neuroinflammation, and promote neuronal survival. In murine CLN2 and CLN3 models, PLX-100 showed neuroprotective effects, reduced brain lipofuscin accumulation, and extended survival in a CLN2 model. The FDA granted orphan drug designation for PLX-100 for neuronal ceroid lipofuscinoses. ([polaryx.com](https://polaryx.com/our-pipeline/))

Other names

all-trans retinoic acid + gemfibrozil