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A review traces how CETP-inhibitor design evolved from first-generation scaffolds plagued by off-target toxicity to optimized late-generation clinical candidates (Eur J Med Chem 2026)

Original title: Structural optimization of cholesteryl ester transfer protein inhibitors: From early scaffolds to advanced clinical candidates

Eur J Med Chem · · 6

Abdollahzadeh M, Ramezani M, Hatamipour M

This review examines the structural optimization of CETP inhibitors, a long-sought dyslipidemia target due to its central role in remodeling lipoprotein particles and reverse cholesterol transport. Over the past two decades, intensive research generated a wide variety of CETP inhibitors spanning diverse primary chemical scaffolds, from early discovery compounds to late-stage clinical candidates. Despite promising beginnings, first-generation CETP inhibitors were found to carry clear flaws, including off-target toxicities, high lipophilicity, suboptimal pharmacokinetic properties, and inconsistent clinical trial outcomes, leading to termination of several late-stage candidates. The review discusses how a next-generation medicinal chemistry approach, optimizing pharmacokinetic and physicochemical properties and refining ligand efficiency across primary scaffolds, enabled development of later-generation CETP inhibitors with substantially improved pharmacokinetic profiles, analyzing this evolution across the diverse bioactive scaffolds explored throughout the class's history.

Read the paper (DOI)PubMed

Original abstract

Cholesteryl ester transfer protein has been a long-sought target for the treatment of dyslipidemia because of its prime function in the remolding of lipoprotein particles and the reverse transport of cholesterol. During the past couple of decades, a huge variety of CETP inhibitors has been generated from intensive biomedical research, taking on diverse primary scaffolds from discovery to late-stage development. Despite their promising beginnings, the first-generation CETP inhibitors have been realized to possess a couple of obvious flaws, such as the existence of off-target toxicities, high lipophilicity, sub-optimized pharmacokinetic properties, and inconsistent results in the outcomes of clinical studies, leading to the termination of a couple of candidates in late-stage development. Therefore, the need for the next-generation approach that considered optimizations in the pharmacokinetics and physiological-chemical properties and the reduction of ligand efficiency and primary scaffolds allowed for the development of pharmacokinetic and late-stage CETP inhibitors with enhanced properties. Here, a thorough discussion to analyze the leveraging of biomedical principles to further develop late-generation CETP inhibitors with major improvements from the pharmacokinetics and results of the primary clinical studies characterizing a huge variety of the first-generation CETP inhibitors with diverse primary bioactive scaffolds will be undertaken.

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Summary written by cetpinhibition.org from the published abstract; figures as published. Page updated 18 August 2026. Methods.