The BEAT-PKD trial (NCT07282821) — a Phase 2, randomized, double-blind, placebo-controlled study of bempedoic acid in ADPKD — is approaching its estimated recruitment start date of July 2026. Funded by the U.S. Department of Defense, the trial will enroll 120 patients receiving 180mg bempedoic acid daily for 2 years.
Bempedoic acid (brand name: Nexletol) is already FDA-approved for lowering LDL cholesterol, but researchers at the University of Vermont discovered it has a second, potentially more relevant property for PKD: it's a potent AMPK activator. Unlike metformin — which activates AMPK throughout the body — bempedoic acid is a pro-drug activated specifically in the liver and kidney, potentially offering targeted benefits with fewer GI side effects.
The primary endpoint is change in Total Kidney Volume (TKV) measured by MRI. AMPK activation in PKD inhibits both mTOR (reducing cell proliferation) and CFTR chloride channels (reducing cyst fluid secretion), addressing two key drivers of cyst growth.
If successful, bempedoic acid could be prescribed off-label almost immediately since it already has FDA approval and an established safety profile. This makes it one of the faster potential paths to a new PKD treatment option. The trial is expected to complete in 2028.