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Related Experiment Video

Updated: Jun 10, 2026

Differential Effects of Lipid-lowering Drugs in Modulating Morphology of Cholesterol Particles

Differential Effects of Lipid-lowering Drugs in Modulating Morphology of Cholesterol Particles

Published on: November 10, 2017

Mipomersen as a potential adjunctive therapy for hypercholesterolemia.

Neeraj Patel¹, Robert A Hegele

¹University of Western Ontario, Robarts Research Institute, London, Ontario, Canada.

Expert Opinion on Pharmacotherapy

|August 17, 2010

Summary

Mipomersen effectively lowers LDL cholesterol in patients with homozygous familial hypercholesterolemia. This antisense oligonucleotide shows promise as an adjunctive therapy, though longer-term safety requires further study.

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Differential Effects of Lipid-lowering Drugs in Modulating Morphology of Cholesterol Particles

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Synthesis of Monocyte-targeting Peptide Amphiphile Micelles for Imaging of Atherosclerosis

Synthesis of Monocyte-targeting Peptide Amphiphile Micelles for Imaging of Atherosclerosis

Published on: November 17, 2017

Area of Science:

Cardiovascular Medicine
Pharmacology
Genetics

Background:

Homozygous familial hypercholesterolemia (HoFH) is a rare genetic disorder (1:1,000,000) leading to extremely high LDL cholesterol levels.
HoFH patients face a severe risk of cardiovascular events and show limited response to conventional lipid-lowering therapies.
Apolipoprotein B-100 (apoB) is a key component of LDL cholesterol, making it a target for novel therapies.

Purpose of the Study:

To evaluate the safety and efficacy of mipomersen as an adjunctive treatment for lowering LDL cholesterol in HoFH patients.
To assess the impact of mipomersen on other lipid parameters, including lipoprotein (a).

Main Methods:

A Phase III, double-blind, randomized, controlled trial involving 34 HoFH patients.
Subcutaneous administration of mipomersen (200 or 160 mg) for 26 weeks.
Monitoring of LDL cholesterol, lipoprotein (a), liver enzymes (ALT), and intrahepatic triglycerides.

Main Results:

Mipomersen significantly reduced LDL cholesterol by 24.7% from baseline.
Mipomersen also demonstrated a reduction in plasma lipoprotein (a) levels.
The most common adverse events were injection-site reactions and influenza-like symptoms. Four patients experienced elevated ALT levels, with one meeting stopping criteria.

Conclusions:

Short-term data suggest mipomersen is a safe and effective adjunctive therapy for reducing LDL cholesterol in HoFH.
Further investigation is necessary to determine the long-term safety and efficacy profile of mipomersen in this patient population.