both drug candidates. Metabolic disorders candidates SPC-4955 is a novel treatment for cholesterol. SPC-4955 inhibits the protein that is necessary for the formation of plasma LDL cholesterol particles. This has the potential to be used as treatment for patients with hyperlipidemia. The PCSK9 program also has the potential to treat patients with hyperlipidemia. It inhibits the protein which controls the number of receptors responsible for removing LDL cholesterol particles from the blood. Infectious disease Santaris developed a microRNA targeting drug for hepatitis C, miravirsen (SPC3649), which entered Phase II clinical trials in 2010. The drug targets miR-122, a host factor necessary for viral replication of the hepatitis C virus in host liver cells; because miravirsen targets a host factor rather than the virus itself, there are no indications of the virus developing resistance. The U.S. Food and Drug Administration approved a multiple dosing study, by injection, to treatment naive patients for phase II testing. Rare genetic disorders Santaris had a collaboration with Shire to discover and develop new RNA-based medicines to treat rare genetic disorders. Collaborations Santaris partnered with several pharmaceuticals companies that wanted to develop LNA oligonucleotides for mRNA and microRNA targets. Pfizer and Santaris entered a collaboration pact in 2009, which was expanded in 2011. It also partnership with Enzon for cancer drug targets, Shire for lead candidates of five rare, undisclosed genetic disorders, miRagen to develop treatments targeting microRNAs associated with cardiovascular disease, and GlasoSmithKline for four viral disease programs and clinical trials. Publications Santaris employees have co-authored articles published in Nature (2008), and Science (2009). These have outlined the results of experiments using LNA oligonucleotides to down-regulate the expression of endogenous microRNAs in primates. Timeline 2003: Santaris founded through a merger of Cureon and Pantheco. 2004: Began cancer drug development – LNA-based drugs SPC3042 targeting Survivin and SPC2968 targeting HIF-1alpha. 2005: Began a miRNA research and drug development program. 2006: Partnership with Enzon for cancer therapeutics. 2007: Commencement of preclinical development of SPC3649, a microRNA-targeted drug for the treatment of Hepatitis C. Established commercial partnership with GlaxoSmithKline for global research and development and of up four programs in viral diseases. Enzon files IND and completes two Phase I/II US studies of advanced cancer research with EZN-2968. 2008: Biotech grant from Danish Advanced Technology for microRNA antagonist research for 45m DKK. Santaris Pharma named one of the “Fierce 15” Biotech Companies of 2008 by FierceBiotech. Advanced to