Biomedical Sciences Blog

Hoersholm, Denmark/San Diego, California, May 4, 2011 — Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the research and development of mRNA and microRNA targeted therapies, today announced that it has advanced SPC5001 into Phase 1 clinical trials for the treatment of high cholesterol. SPC5001 is a mRNA-targeted drug that inhibits the exciting new target, Proprotein Convertase Subtilisin/Kexin type 9 (PCSK9), a protein involved in removing low density lipoprotein cholesterol (LDL-C) or “bad” cholesterol from the bloodstream.

Hoersholm, Denmark/San Diego, California, May 11, 2011 — Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the research and development of mRNA and microRNA targeted therapies, today announced that it has advanced a second drug from its cardiometabolic program, SPC4955 into Phase 1 clinical trials, for the treatment of high cholesterol. Developed using Santaris Pharma A/S proprietary Locked Nucleic Acid (LNA) Drug Platform, SPC4955 is a mRNA-targeted drug candidate that inhibits apolipoprotein B (apoB), a major protein component involved in the formation of low density lipoprotein cholesterol (LDL-C) or “bad” cholesterol.

"Advancing two drug candidates, SPC4955 and SPC5001, in the space of only a week from our cardiometabolic program into the clinic is a testament to the efficiency of our LNA Drug Platform and Drug Discovery Engine capabilities and represents an important step for patients as multiple approaches are needed to lower high cholesterol levels," said Arthur A. Levin, PhD, Vice President and Chief Development Officer of Santaris Pharma A/S. "SPC4955 aims to inhibit the production and export of LDL-C in the liver while our other clinical candidate SPC5001, increases the clearance of LDL from the blood. Working on two distinct compounds with different target pathways allows the company to address the broad spectrum of patients trying to control their high cholesterol levels."

The LNA Drug Platform and Drug Discovery Engine developed by Santaris Pharma A/S combines the Company's proprietary LNA chemistry with its highly specialized and targeted drug development capabilities to rapidly deliver potent single-stranded LNA-based drug candidates for a range of diseases including metabolic disorders, infectious and inflammatory diseases, cancer and rare genetic disorders.

With its partners, Santaris Pharma A/S also has a robust product pipeline consisting of mRNA and microRNA drug discovery and development collaborations. These include partnerships with Pfizer, Inc. (delivery of lead candidates against up to 20 targets), miRagen Therapeutics (cardiovascular diseases), Shire plc (rare genetic disorders), GlaxoSmithKline (four viral disease drug candidates) and Enzon Pharmaceuticals (eight cancer targets successfully delivered - three are now in Phase 1 clinical studies).