BGT-002: A Comprehensive Clinical and Strategic Analysis of a Novel ATP-Citrate Lyase Inhibitor for Metabolic Disease

Executive Summary

BGT-002 is an investigational, orally administered, small molecule drug being developed by Burgeon Therapeutics, also known as Bojiyuan (Shanghai) Biopharmaceutical Co., Ltd..[1] As a new molecular entity, it is classified as an inhibitor of adenosine triphosphate (ATP)-citrate lyase (ACLY), a pivotal enzyme in the metabolic pathways responsible for cholesterol and fatty acid synthesis.[3] This mechanism of action positions BGT-002 as a promising therapeutic candidate for treating prevalent metabolic disorders, primarily primary hypercholesterolemia and nonalcoholic steatohepatitis (NASH), with the drug currently in Phase 2 development for both indications.[1]

The therapeutic rationale for BGT-002 is anchored in the clinical validation of its target, ACLY, by the first-in-class inhibitor bempedoic acid. By acting upstream of HMG-CoA reductase (the target of statins), BGT-002 offers a distinct approach to lipid management. Clinical data to date support this rationale. A Phase I study in healthy volunteers demonstrated dose-proportional, clinically relevant reductions in key atherogenic lipids, with a maximal decrease in low-density lipoprotein cholesterol (LDL-C) of 22.4% after 14 days of administration.[5] This established early proof-of-concept for its utility in hypercholesterolemia.