PLA2G15 inhibitor

Scenic Biotech is developing a first-in-class, brain-penetrant small-molecule inhibitor of PLA2G15 (lysosomal phospholipase A2) for the treatment of neurometabolic and neurodegenerative diseases, including Niemann-Pick Type C (NPC), Batten Disease, and Frontotemporal Lobar Degeneration (FTLD). PLA2G15 is a lysosomal enzyme that modulates the levels of bis(monoacylglycerol)phosphate (BMP), a lipid essential for lysosomal function that is often depleted or altered in neurodegenerative conditions. By inhibiting PLA2G15, the drug aims to restore BMP levels and improve lysosomal health. Preclinical data in NPC mouse models have demonstrated improvements in neurodegeneration, spleen and liver damage, and overall survival. The program is currently in the IND-enabling phase with plans to proceed to first-in-human clinical trials.

Other names

PLA2G15 inhibitor (Scenic Biotech)Scenic Biotech PLA2G15 program