metabolic disorders

Metabolic disorders refer to a broad class of diseases that affect the processes that the body uses to get or make energy from ingested food. Metabolic disorders are quite common and underlie some of the major diseases in the Western world, such as diabetes and cardiovascular diseases, both of which reduce quality of life and life expectancy in the affected patients.

Cardiovascular diseases, such as heart disease and stroke, are the leading cause of death in the world. Often these diseases are the clinical manifestation of atherosclerosis caused by prolonged high levels of plasma LDL-cholesterol. Worldwide, 4.4 million deaths (about 7.9% of the total) and 40.4 million disability-adjusted life years (DALYs) (2.8% of the total) were estimated to be due to non-optimal cholesterol. The relative impact of attributable deaths and DALYs attributable to non-optimal cholesterol (i.e. mean >3.8 mmol/l) - was highest in the European sub-regions, where mean cholesterol levels were highest.

SPC4955: a novel treatment for high cholesterol
SPC4955 is a specific inhibitor of apoB-100, the primary apolipoprotein of plasma LDL-cholesterol particles (bad cholesterol) and key to their formation. Inhibition of apoB-100 is known to effectively lower plasma LDL-cholesterol in humans, suggesting that SPC4955 could become an effective novel therapy for patients with hyperlipidemia, in particular a sub-segment of severely hyperlipidemic patients that are not adequately managed by current cholesterol-lowering drugs.

In preclinical studies in rodents and primates, SPC4955 potently and dose-dependently inhibited its target mRNA, which caused significant and long-lived reductions in plasma levels of LDL-cholesterol and triglycerides.

In 2009, Santaris Pharma A/S selected SPC4955 as a drug-development candidate and it is currently in Phase 1 clinical trials to assess safety, tolerability, pharmacokinetics, and pharmacodynamics of SPC4955.

PCSK9: a novel treatment for hyperlipidemia 
Santaris Pharma A/S is developing a compound to inhibit PCSK9, a protein that controls the number of low-density lipoprotein receptors that remove LDL cholesterol particles from the bloodstream.

microRNA targets: a novel treatment for hyperlipidemia 
Santaris Pharma A/S also holds a partnership with miRagen to develop drug candidates against a limited number of microRNA targets for the treatment of cardiovascular disease.  miRagen licenses rights to utilize LNA Drug Platform for specific microRNA targets implicated in cardiovascular disease. Santaris Pharma A/S received a minority equity interest in miRagen and is eligible to  receive milestone payments and royalties.