Santaris Pharma obtained an exclusive license from Massachusetts General Hospital (MGH) for intellectual property related to the regulation of miR-33 for the treatment of cardiovascular disorders. Santaris Pharma will utilize its locked nucleic acid (LNA) drug platform to develop a compound targeting this miRNA, which regulates HDL levels.
Last year MGH researchers published data in Science suggesting that the miR-33 family of miRNAs suppresses a protein known to be important for generation of HDL and for the removal of cholesterol from peripheral tissues including cells that form atherosclerotic plaques. They found that turning off miR-33 raises HDL levels.
“Current treatments for such cholesterol abnormalities as low circulating HDL levels are only modestly effective, and there is an urgent need for new therapeutic strategies,” notes Anders Naar, Ph.D., of the MGH Center for Cancer Research, who led the Science study. “Our discovery of miR-33 as a key regulator of HDL has provided a novel therapeutic target for antisense-based technologies to ameliorate cardiometabolic disorders.”
Santaris’ LNA drug platform utilizes single-stranded LNA chemistry. The most important features of LNA-based drugs include excellent specificity providing optimal targeting, increased affinity to targets providing improved potency, and favorable pharmacokinetic and tissue-penetrating properties that allow systemic delivery of these drugs without complex and potentially troublesome delivery vehicles, according to the firm.
Santaris has one LNA-based drug into clinical trials. In September 2010, Santaris advanced miravirsen, which targets miR-122, into Phase II studies in patients infected with hepatitis C. The firm expects to move two mRNA-targeted drugs, SPC5001 targeting PCSK9 and SPC4955 targeting apoB, for the treatment of high cholesterol into Phase I in the first half of this year.
Founded in 2003, Santaris’ R&D activities focus on infectious diseases and metabolic disorders. Partnerships include therapeutic areas like cancer, cardiovascular disease, infectious and inflammatory diseases, and rare genetic disorders. The company has collaborations with miRagen Therapeutics, Shire, Pfizer, GlaxoSmithKline, and Enzon Pharmaceuticals.