First microRNA Targeted Drug Reaches Phase II Clinical Trial

Santaris Pharma A/S announced today that it has advanced miravirsen (SPC3649), a microRNA targeted drug for treatment of  Hepatitis C virus (HCV) infection into Phase 2 studies to assess the safety and tolerability of the drug.

Santaris Pharma A/S also received acceptance of its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA). In addition to the United States, the Phase 2a clinical trials will be conducted in the Netherlands, Germany, Poland, Romania, and Slovakia.

Miravirsen is a specific inhibitor of miR-122, a liver specific microRNA that the Hepatitis C virus requires for replication. Miravirsen is designed to recognize and sequester miR-122, making it unavailable to the Hepatitis C virus. As a result, the replication of the virus is effectively inhibited and the level of Hepatitis C virus is reduced.

In addition to miravirsen, Santaris Pharma A/S is developing other drugs targeting mRNAs and microRNAs both internally as well as in partnerships and collaborations with miRagen Therapeutics (cardiovascular diseases), Shire plc (rare genetic disorders), Pfizer (undisclosed therapeutic areas), GlaxoSmithKline (viral disease) and Enzon Pharmaceuticals (oncology).  (read more… )

It’s only a matter of time before the first microRNA therapeutic reaches the market.

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