Progranulin Modulator-1 (Compound 60) is an orally active compound that promotes progranulin (PGRN) secretion. It enhances the ability of BV-2 cells to elevate PGRN levels, exhibits low cytotoxicity, and possesses an inhibitory effect on hERG. The EC50 for PGRN is 83 nM, and the IC50 for hERG is 3100 nM [1].

Progranulin modulator-1 (Compound 60) exhibits an efflux ratio (ER) value of less than 2, indicating it is not a substrate of MDR, and demonstrates high binding affinity to brain tissue along with exceptional metabolic stability [1].

Progranulin modulator-1 (Compound 60) (i.v.: 1 mg/kg, p.o.: 10 mg/kg) demonstrates a favorable pharmacokinetic profile in mice, with high bioavailability and a long half-life. It achieves an oral bioavailability of up to 99% and displays a Kpuu greater than 1, indicating significant brain accumulation within 8 hours post-administration, allowing for regulatory activity at relatively low dosages of Progranulin modulator-1 [1].