The Danish biotech company Santaris Pharma has announced in a press release that it has commenced Phase I clinical trials of the world's first microRNA medicine to be tested in man.
Candidate drug SPC3649, the first of a new class of microRNA antagonists based on Santaris Pharma's proprietary 'Locked Nucleic Acid' technology, has been developed as a potential new treatment for Hepatitis C and will be tested in up to 48 healthy male volunteers.
“The mechanism of action of this drug represents a potential breakthrough in medical science," says Keith McCullagh, Santaris Pharma CEO, in the press release. "We are excited now to be able to evaluate the drug’s efficacy and safety in human subjects. If successful, such trials may lead to the development of a new approach to the treatment of Hepatitis C and more generally, contribute to the development of a major new class of therapeutic agents.”
For the uninitiated, microRNAs are molecules which control many biological processes in cells, and are also implicated in diseases such as cancer and viral infections. MicroRNA antagonists block the actions of microRNAs and can thus potentially halt the progress of disease. SPC3649 specifically blocks microRNA-122 in liver cells, preventing the Hepatitis C virus from multiplying.
Santaris Pharma completed a USD 30m financing round in December 2007, with Gilde Healthcare Partners from the Netherlands stepping in as a new investor. Immediately thereafter came news that Santaris Pharma had entered a global strategic alliance with pharma giant GlaxoSmithKline (GSK). Part of the deal gives GSK 'an option to a compound being developed by Santaris Pharma as a potential new therapy for Hepatitis C infection', we reported on this website last December.
Santaris Pharma was formed in 2003 and currently employs 85 people. The company has exclusive rights to LNA technology, which it is using to develop new classes of RNA medicines targeted against cancer and metabolic disorders. Santaris Pharma has a co-development partnership in the cancer treatment area with US company Enzon Pharmaceuticals.