Hypercholesterolemia is a disorder characterised by abnormally increased levels of cholesterol in the blood. The high levels of cholesterol increase a person's risk of developing cardiovascular diseases such as coronary artery disease, which involve build-up of plaque in the coronary arteries, in turn putting a person across the risk of suffering a heart attack. The main causes of hypercholesterolemia include heredity, diet high in saturated fats and cholesterol, other diseases such as diabetes mellitus, renal diseases and obesity, along with lifestyle changes including little exercise, smoking, alcoholism and stress. Lifestyle changes, dietary changes, and medications can control the disease to some extent. The main class of drugs prescribed to lower the increased cholesterol levels is called statins or HMG-CoA reductase inhibitors, and the other classes of drugs are in development phase. The hypercholesterolemia therapeutic pipeline is expected to witness growth mainly due to technological advancements in the field, along with increasing awareness regarding different cardiovascular diseases and the availability of their treatment options. Increasing prevalence of diabetes and obesity disorders and changing lifestyle of people are further contributing to the incidence of hypercholesterolemia, globally.
In April 2017, Regeneron Pharmaceuticals, Inc.’s evinacumab received breakthrough therapy designation for the treatment of homozygous familial hypercholesterolemia (HoFH) from the U.S. Food and Drug Administration. Evinacumab is an investigational monoclonal antibody to angiopoietin-like protein 3 (ANGPTL3), which acts as an inhibitor of lipoprotein lipase and endothelial lipase, and appears to play a central role in lipoprotein metabolism. After reporting positive interim Phase II results, Regeneron is planning a Phase III trial for evinacumab in HoFH patients. In April 2017, Alnylam Pharmaceuticals partnered with The Medicines Company and RNA Biotech, for a Phase III trials that will randomize 3,000 subjects with ASCVD (atherosclerotic cardiovascular disease) and FH (familial hypercholesterolemia) to either inclisiran or placebo, with a primary endpoint of LDL cholesterol.
In April 2016, Viking Therapeutics, Inc. announced positive Phase Ib clinical data on VK2809 in hypercholesterolemic subjects. The results of the trial showed that VK2809 treatment showed reduced levels of LDL-C, ranging from 15.2% at the 5.0 mg dose (p=0.026) to 41.2% at the 20 mg dose (p<0.0001).
In July 2014, Abeome Corporation received funding for a Small Business Innovative Research (SBIR) grant focused on generating a large number of monoclonal antibodies to human PCSK9, an emerging drug target for the treatment of high cholesterol.
The major companies having drugs in pipeline for the treatment of hypercholesterolemia include 3SBio Inc., Abeome Corporation, Aegerion Pharmaceuticals, Inc., AFFiRiS AG, Alnylam Pharmaceuticals, Inc., Alvogen Korea Co., Ltd., AtheroNova Inc., BioLingus AG, Catabasis Pharmaceuticals, Inc., Cerenis Therapeutics Holding SA, Chong Kun Dang Pharmaceutical Corp., CymaBay Therapeutics, Inc., Daewon Pharm Co. Ltd., Eli Lilly and Company, Esperion Therapeutics, Inc., F. Hoffmann-La Roche Ltd., Hanmi Pharmaceuticals, Co. Ltd., Immune Response BioPharma, Inc., Johnson & Johnson, Kadmon Corporation, LLC, Kastle Therapeutics, LLC, Kowa Company, Ltd., Kyorin Pharmaceutical Co., Ltd., Merck & Co., Inc., Omeros Corporation, Pfizer Inc., Planet Biotechnology Inc., Progenra, Inc., Regeneron Pharmaceuticals, Inc., RegenxBio Inc., Serometrix, LLC, Shanghai Fudan-Zhangjiang Bio-Pharmaceutical Co., Ltd., Thetis Pharmaceuticals LLC, Viking Therapeutics, Inc. and Zhejiang Hisun Pharmaceutical Co., Ltd.