NDI-5001

NDI-5001 is a proprietary small molecule drug developed as a kidney-targeted activator of AMP‑activated protein kinase (AMPK). It is being advanced as a potential first-in-class therapy for congenital nephrogenic diabetes insipidus (NDI), particularly the X-linked form caused by mutations in the vasopressin V2 receptor gene. Unlike other AMPK activators under development for type 2 diabetes, NDI‑5001 does not affect blood glucose levels in preclinical studies and is designed to act downstream of the defective receptor pathway. The drug has received Orphan Drug Designation and Rare Pediatric Disease designation from the US FDA. Its mechanism offers an alternative to gene therapy approaches by directly activating AMPK in kidney cells to improve urine concentration ability[2][3][6][8].

Other names

Ndi5001Ndi-5001Ndi 50011,1'-(1,12-dodecanediyl)bis[cyclopropanecarboxylic acid]Cyclopropanecarboxylic acid, 1,1'-(1,12-dodecanediyl)bis-1-[12-(1 carboxycyclopropyl)dodecyl]cyclopropane-1-carboxylic acid