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Emerging small molecule drugs

Sophie Colin1, Giulia Chinetti-Gbaguidi, Jan A Kuivenhoven

  • 1Université Lille 2, F-59000, Lille, France.

Handbook of Experimental Pharmacology
|December 20, 2014
PubMed

Insights

Emerging small molecules aim to improve cardiovascular health by targeting HDL-C levels and function, especially for patients with type 2 diabetes mellitus who have persistent risks despite statin therapy.

Area of Science:

  • Cardiovascular Medicine
  • Pharmacology
  • Metabolic Diseases

Background:

  • Dyslipidemia is a key risk factor for cardiovascular diseases (CVD).
  • Statins effectively lower LDL-C but residual risk remains, particularly in type 2 diabetes mellitus (T2DM).
  • High-density lipoprotein cholesterol (HDL-C) levels are inversely associated with CVD risk, prompting interest in therapies to raise HDL-C or improve its function.

Purpose of the Study:

  • To review emerging small molecules in clinical trials for modulating HDL-C levels and functionality.
  • To explore novel therapeutic strategies as complementary treatments for cardiovascular diseases.

Main Methods:

  • Review of clinical trial data for novel small molecules targeting HDL-C.
  • Focus on emerging drug classes including CETP inhibitors, PPAR agonists, LXR agonists, and other agents like RVX-208.

Main Results:

  • Most previous therapies targeting HDL-C have failed in clinical trials due to side effects or lack of clinical benefit.
  • This chapter highlights specific emerging small molecules currently undergoing clinical evaluation.

Conclusions:

  • Novel small molecules targeting HDL-C and functionality represent a promising area for complementary cardiovascular disease therapy.
  • Further clinical evaluation is crucial to determine the efficacy and safety of these emerging agents, especially in high-risk populations like T2DM patients.

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