napazimone

Napazimone (KL1333) is an orally bioavailable small molecule NAD+ modulator being developed for the treatment of primary mitochondrial diseases (PMD). It acts as a substrate for the enzyme NAD(P)H:quinone oxidoreductase 1 (NQO1). By interacting with NQO1, napazimone facilitates the oxidation of NADH to NAD+, thereby increasing intracellular NAD+ levels and restoring the NAD+/NADH ratio. This mechanism is intended to improve mitochondrial function, enhance energy metabolism, and reduce oxidative stress. Originally discovered by KT&G Life Sciences, the drug is currently being developed by Abliva AB in collaboration with Pharming Group NV. It is currently in Phase 3 clinical development (the FALCON study) for patients with mitochondrial DNA-related diseases, including MELAS syndrome and Kearns-Sayre syndrome.

Other names

2-Isopropyl-3H-naphtho(1,2-d)imidazole-4,5-dione