Denmark-based Santaris Pharma, which has U.S.-based operations in San Diego, just announced it has received the green signal from the U.S. Food & Drug Administration to conduct phase two trials for miravirsen, a micro ribonucleic acid-targeted drug intended for the treatment of Hepatitis C virus, a liver disease.
Privately held Santaris is a clinical stage biopharmaceutical company, which uses a proprietary locked-nucleic acid (LNA) drug platform to discover and develop RNA- targeted therapies to treat infectious diseases and metabolic disorders.
Last year the company opened its San Diego office, which focuses on business and clinical development. Nearly 10 out of 75 employees work out of San Diego.
“Micro-RNAs are developing a lot of excitement. Ours is the first micro-RNA drug to reach clinical trials for treating Hepatitis C,” said Arthur Levin, vice president of the firm.
Micro-RNAs are now thought of as an important class of small RNAs encoded in the genome. They control the expression of sets of genes and entire pathways, like master regulators.
Levin explained that most small molecule drugs such as aspirin and estrogen bind to protein and change the way it works, where as Santaris’ technology enables it to attach to the RNA target, inhibiting it from making the disease-causing protein.
“So developing RNA-targeted medicines is a whole new way of attacking diseases where previously small molecule drugs were not able to reach,” Levin pointed out.
Santaris’ LNA-based drugs are small in size and bind tightly to their RNA target. Levin said that instead of attacking the protein, Santaris focuses on treating the RNA coating the protein, which is the company’s window of opportunity.
Approximately 3 million to 4 million Americans are chronically infected with Hepatitis C, and each year an additional 40,000 people join those ranks. According to the American Association for the study of liver diseases, the current treatment on the market is effective in only half of those treated.
Aside from its own research areas, Santaris has also made steady progress in the last few years by collaborating with big name pharma companies such as Pfizer (NYSE: PFE), Shire (Nasdaq: SHPGY) and Glaxo SmithKline (NYSE: GSK) as well as others such as Enzon (Nasdaq: ENZN) and miRagen Therapeutics.
These partnerships focus on a range of therapeutic areas including cancer, cardiovascular disease, infectious and inflammatory diseases, and rare genetic disorders.
While 2008 was a banner year for Santaris, when it attained clinical stage status, Levin considers 2010 to be an even bigger year, since miravirsen has advanced to phase two clinical trials.
Nagappan is a San Diego-based business writer.