ALN-PCS: Hypercholesterolemia

ALN-PCS: Hypercholesterolemia

Alnylam is developing ALN-PCS, an RNAi therapeutic, to treat hypercholesterolemia, or high levels of cholesterol in the blood, that contributes to many diseases, most notably cardiovascular disease — the leading cause of death in the U.S.
ALN-PCS works by silencing the gene proprotein convertase subtilisn/kexin type 9, or PCSK9, which human genetics studies indicate to be an ideal target to treat hypercholesterolemia. Individuals with a genetically increased PCSK9 activity were found to have increased levels of LDL cholesterol, or bad cholesterol, together with an increased risk of cardiac disease. In contrast, individuals with genetically less PCSK9 activity were found to have significantly lower levels of LDL cholesterol and a significantly reduced risk of cardiac disease. Because of its novel mechanism of action, we believe that ALN-PCS has the opportunity to treat hypercholesterolemia in a way that is un-addressable by current therapies, such statin drugs.
In 2007, we advanced ALN-PCS in pre-clinical studies and we have seen very promising results, including a greater than 50% reduction in levels of LDL cholesterol in non-human primates. As we advance this program towards a Phase I study in humans, we look forward to demonstrating safety and tolerability in healthy volunteers. In this same early clinical study, we may also have the opportunity to evaluate the efficacy of ALN-PCS in reducing LDL cholesterol levels.

ALN-PCS Clinical Timeline

• Disease Info

Hypercholesterolemia is a condition characterized by very high levels of cholesterol in the blood. The body needs cholesterol to build cell membranes, make certain hormones, and produce compounds that aid in fat digestion. Too much cholesterol, however, increases a person's risk of developing a form of heart disease called coronary artery disease. This condition occurs when excess cholesterol in the bloodstream is deposited in the walls of blood vessels. The abnormal buildup of cholesterol forms clumps (plaque) that narrow and harden artery walls. As the clumps grow, they can clog the arteries and restrict the flow of blood to the heart. The buildup of plaque in coronary arteries causes a form of chest pain called angina and greatly increases a person's risk of having a heart attack.