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Santaris Pharma A/S Advances RNA-Targeted Drug Development Candidate Against PCSK9, an Important New Target for the Treatment of High Cholesterol
Date:4/26/2010

HOERSHOLM, Denmark and SAN DIEGO, April 26 /PRNewswire/ -- Santaris Pharma A/S, a clinical-stage biopharmaceutical company focused on the discovery and development of RNA-targeted therapies, today announced that it has advanced into drug development a discovery research candidate directed against PCSK9 (proprotein convertase subtilisin/kexin type 9), an important new target for the treatment of high cholesterol. Using its proprietary Locked Nucleic Acid (LNA) Drug Platform and Drug Discovery Engine, Santaris Pharma A/S identified and advanced the new drug, SPC5001, in just 18 months.

High cholesterol is a major risk factor for coronary heart disease, heart attack and stroke. According to the World Health Organization, high cholesterol is estimated to cause 18% of strokes and 56% of heart disease, globally and amounts to approximately 4.4 million deaths and 40.4 million disability-adjusted life years(1).

Santaris Pharma A/S presented preclinical data on SPC5001 last month at the PCSK9 Conference "From gene to therapeutics" in Nantes, France where many top pharmaceutical companies gathered to discuss efforts to develop therapies aimed at inhibiting the important new target PCSK9, a protein involved in regulating LDL (low-density lipoprotein) or "bad" cholesterol in the blood.

Data presented by Santaris Pharma A/S scientists showed that SPC5001 provided potent, specific and long-lasting inhibition of PCSK9 and lowered mean LDL cholesterol by 50% in non-human primates with a sustained reduction of 74% in the highest responder. SPC5001 did not change HDL (high-density lipoprotein) levels or the "good" cholesterol in the blood(2). The preclinical data suggest that SPC5001 has the potential to provide patients with a new treatment opti
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