Publications

Aegerion conducts both preclinical and clinical investigational studies on its product candidates. When appropriate, Aegerion may provide results of these studies to educate the research, clinical and medical communities on both the mechanisms of disease states as well as clinical results from its therapies. There are also other important scientific publications relating to the disease state and mechanism of action of our therapies from renowned authors and institutions that provide the basis for our clinical research.

Through the years, we have published and presented results relating to the efficacy and safety of our investigational therapies in full, peer-reviewed publications as well as presentations at society meetings. We have published, with our investigators, the results of our clinical trial work in world-renowned journals such as the New England Journal of Medicine(NEJM), and Nature, Clinical Practice Cardiovascular Medicine. We have presented at major society meetings such as the AHA, ADA, EAS, DALM, ISA, AALSD, and ATVB, among others. This site contains a bibliography of publications that should help educate and provide background on HoFH, FC, MTP-I and the clinical trial research of lomitapide.

HoFH

Goldstein JL, Hobbs HH, Brown MS. Familial hypercholesterolemia. In: Scriver CR, Beaudet Al, Sly WS, Valle D, eds. The Metabolic and Molecular Bases of Inherited Disease. 8th ed. New York: McGraw-Hill Information Services Company; 2001:2863-913

Kolansky DM, Cuchel M, Clark BJ, et al. Longitudinal evaluation and assessment of cardiovascular disease in patients with homozygous familial hypercholesterolemia. Am J Cardiol. 2008; 102:1438-1443.

MTP and Mechanisom of Action

Hussain MM, Shi J, Dreizen P. Microsomal triglyceride transfer protein and its role in apoB-lipoprotein assembly. J Lipid Res. 2003; 44:22-32.

Liao W, Hui TY, Young SG, Davis RA. Blocking microsomal triglyceride transfer protein interferes with apoB secretion without causing retention or stress in the ER. J Lipid Res. 2003; 44:978-985.

Wetterau JR, Gregg RE, Harrity TW, et al. An MTP inhibitor that normalizes atherogenic lipoprotein levels in WHHL rabbits. Science 1998;282: 751-4.

Lomitapide

Cuchel M, Bloedon LT, Szapary PO, Kolansky DM, Wolfe ML, Sarkis A, Millar JS, Ikewaki K, Siegelman ES, Gregg RE, Rader DJ: Inhibition of microsomal triglycerides transfer protein in familial hypercholesterolemia. N Engl J Med 356: 148-156, 2007. Free Download from NEJM

Samaha FF, McKenney J, Bloedon LT et al. Inhibition of triglyceride transfer protein alone or with ezetimibe in patients with moderate hypercholesterolemia. Nature Clinical Practice, Cardiovascular Medicine 2008; 5: 497-505.

Scientific Presentations and Posters

	ISA 2009 PDF Efficacy, Safety and Tolerability of the MTP--Inhibitor, AEGR--733 (Lomitapide) Combined with Atorvastatin Michael Davidson1, Thomas Littlejohn2, Sheila Rodstein3, Carol Price4, Matthew Parris4, William Sasiela4, Daniel Rader5 1Radiant Research, Chicago, IL; 2Piedmont Medical Research, Winston-Salem, NC; 3Radiant Research, Minneapolis, MN; 4Aegerion Pharmaceuticals, Bridgewater, NJ; 5University of Pennsylvania, Philadelphia, PA
	ISA 2009 PDF Impact of high doses of the MTP-Inhibitor, AEGR-733, on the single dose Pharmacokinetics of Atorvastatin and Rosuvastatin Richard L Dunbar1,LeAnne T Bloedon1,2, Ram Gadi1, Danielle Duffy1, Amanda Baer1, William J Sasiela2, Daniel J Rader1, Marina Cuchel1 1Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, PA, USA; 2Aegerion Pharmaceuticals, Bridgewater, NJ, USA
	ISA 2009 PDF Impact of High Doses off the MTP--Inhibitor Lomitapide (AEGR--733) on the Single--Dose Pharmacokinetics of Extended--Release Niacin Richard L. Dunbar1, LeAnne T. Bloedon1,2, Ramprasad Gadi1, Raj Movva1, Amanda Baer1, Matthew Parris2, William J. Sasiela2, Daniel J. Rader, Marina Cuchel1 1Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, PA, USA; 2Aegerion Pharmaceuticals, Bridgewater, NJ, USA
	ATVB 2009 PDF Microsomal Triglyceride Transfer Protein Jeffrey Billheimer, Debra Cromley, William Sasiela^ and Daniel J. Rader* *University of Pennsylvania, Institute for Translational Medicine and Therapeutics, Philadelphia, PA ^ Aegerion Pharmaceuticals, Bridgewater, NJ
	AHA 2009 PDF A Phase 3 Study of Microsomal Triglycerides Transfer Protein Inhibitor Lomitapide (AEGR-733) in Patients with Homozygous Familial Hypercholesterolemia: Interim Results at 6 Months Marina Cuchel1, Emma Meagher1, A David Marais2, Dirk J Blom2, Hendrick du Toit Theron3, Amanda L Baer1, LeAnne Bloedon4, William J Sasiela4, Daniel J Rader1 1Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, PA, USA; 2University of Cape Town, Cape Town, South Africa; 3Netcare Private Hospital, Bloemfontein, South Africa; 4Aegerion Pharmaceuticals, Bridgewater, NJ
	AASLD 2009 PDF Prevalence and Analysis of Hepatic Steatosis in Patients with Hypercholesterolemia who Lack Traditional Risk Factors Associated with Steatosis Scott Grundy, Michael Davidson, Harold Bays, Mark Rosen, Emma Meagher, LeAnne Bloedon, Carol Price, Matthew Parris, William Sasiela, Daniel Rader From The University of Texas Southwestern, Dallas, TX (SG); Radiant Research, Chicago, IL (MD); Louisville Metabolic and Atherosclerosis Research Center, Louisville, KY (HB); University of Pennsylvania, Philadelphia, PA (MR, EM, LB, DR), and Aegerion Pharmaceuticals, Bridgewater, NJ (LB, CP, MP, WS)
	ADA 2008 PDF Body Weight Reductions with Low Dose Lomitapide (AEGR-733), a Microsomal Triglyceride Transfer Protein Inhibitor: Results from Three Phase 2 Trials Harold E. Bays1, MD, Michael Davidson2, MD, Sherwyn L. Schwartz3, MD, Carol Price4, RN, BS, William J. Sasiela4, PhD, Matthew Parris4, BS, Emma Meagher5, MD, Daniel Rader5, MD Pharmaceuticals, Inc. Aegerion 1. Louisville Metabolic and Atherosclerosis Research Center, Louisville, Kentucky, 40213, 2. The University of Chicago Pritzker School of Medicine, Preventive Cardiology,Chicago, Illinois, 60637, 3. Diabetes and Glandular Disease Research, San Antonio, Texas, 78229 4. Aegerion Pharmaceuticals, Bridgewater, New Jersey, 08807, 5. University of Pennslvania, Institute for Translational Medicine and Therapeutics, Philadelphia, Pennsylvania, 19104
	AHA 2008 PDF Impact of the MTP-Inhibitor AEGR-733 as Monotherapy and in Combination with Ezetimibe on Lipid Subfractions as Measured by NMR Spectroscopy Frederick F. Samaha, James McKenney, LeAnne T. Bloedon, William J. Sasiela, Daniel J. Rader From The University of Pennsylvania, Philadelphia, PA (FFS, LTB, DJR); National Clinical Research, Richmond, VA (JM); and Aegerion Pharmaceuticals, Bridgewater, NJ (LTB, WJS)
	DALM 2007 PDF Impact of the MTP Inhibitor AEGR-733 on Pharmacokinetics of Statins Danielle Duffy¹, LeAnne T. Bloedon¹, Richard L. Dunbar¹, Ram Gadi¹, Rajesh Movva¹, William J. Sasiela², Daniel J. Rader¹, Marina Cuchel¹ Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, PA, USA; 2. Aegerion Pharmaceuticals, Bridgewater, NJ, USA
	DALM 2007 PDF Microsomal Triglyceride Transfer Protein Inhibitor AEGR-733 Reduces Body Weight in Patients with Hypercholesterolemia – a Phase 2 Trial Federick F. Samaha1, James McKenney2, LeAnne T. Bloedon3, William J. Sasiela4, Daniel J. Rader3 1. Division of Cardiology, the University of Pennsylvania Health System; 2. National Clinical Research, Richmond, VA; 3. Institute for Translational Medicines Therapeutics, UPenn, Philadelphia, PA; 4. Aegerion Pharmaceuticals, Bridgewater, NJ.
	DALM – 2007 PDF Efficacy and Safety of the MTP-Inhibitor AEGR-733 as Monotherapy and in Combination with Ezetimibe–a Phase 2 Trial Federick F. Samaha1, James McKenney2, LeAnne T. Bloedon3, William J. Sasiela4, Daniel J. Rader3 1. Division of Cardiology, the University of Pennsylvania Health System; 2. National Clinical Research, Richmond, VA; 3. Institute for Translational Medicines Therapeutics, UPenn, Philadelphia, PA; 4. Aegerion Pharmaceuticals, Bridgewater, NJ.
	DALM – 2007 PDF Impact of the MTP Inhibitor AEGR-733 on Pharmacokinetics of Fenofibrate Richard L. Dunbar¹, LeAnne T. Bloedon¹, Danielle Duffy¹, Ram Gadi¹, Rajesh Movva¹, William J. Sasiela², Daniel J. Rader¹, Marina Cuchel¹ 1. Institute for Translational Medicine and Therapeutics, University of Pennsylvania, Philadelphia, PA, USA; 2. Aegerion Pharmaceuticals, Bridgewater, NJ, USA

Publications

HoFH

MTP and Mechanisom of Action

Lomitapide

Scientific Presentations and Posters

What isHoFH?

What is
HoFH?