Drug intelligence / Profile preview

Exo-KH

Development stage
Preclinical
Modality
Small Molecules, Lipid-based Nanoparticles → Nanoparticles → Drug Delivery Systems
Administration
Intravenous
01

Overview

Exo-KH is an experimental exosome-based therapeutic formulation designed for the treatment of non-small cell lung cancer (NSCLC). It consists of bovine milk-derived exosomes that are surface-conjugated with folate for targeted delivery and loaded with potassium hydroxycitrate (KH), a natural inhibitor of ATP citrate lyase (ACLY). ACLY is a key enzyme in cancer metabolism that converts mitochondria-derived citrate into acetyl-CoA and oxaloacetate in the cytosol, supporting the increased lipid and cholesterol synthesis required for rapid tumor growth (the Warburg effect). By delivering KH directly to tumor cells via folate receptor-mediated endocytosis, Exo-KH inhibits ACLY activity, thereby reducing the expression of downstream metabolic enzymes such as fatty acid synthase (FASN) and HMG-CoA reductase (HMGCR). Preclinical studies in murine models of lung adenocarcinoma have demonstrated that Exo-KH provides continuous release of its payload, reduces systemic and local ACLY activity, and significantly suppresses tumor progression compared to the free form of the drug.

Other names
potassium hydroxycitrate-loaded exosomesfolate-conjugated potassium hydroxycitrate-loaded bovine milk exosomes
02

Targets

ACLY (ATP-citrate synthase)

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