Platform: Locked nucleic acids (LNAs)
Company: Santaris Pharma
Partnerships: miRagen for cardiovascular disease; Shire for rare genetic disorders; Pfizer for multiple targets, including cancer; GlaxoSmithKline ($GSK) for viral diseases; and Enzon ($ENZN) for cancer.
Recent news about the impact of hepatitis C shows that effective therapies for the disease are still needed: Roughly 170 million people are infected with HCV worldwide, and HCV infection causes the greatest number of liver transplants. And a bevy of drugmakers and developers are in hot pursuit of new remedies against the liver-damaging disease.
The huge opportunity has fueled the Danish company Santaris in its quest to combat HCV with its next-generation locked nucleic acid (LNA) product miravirsen. While current medications for HCV infection attack the virus in the liver directly, miravirsen keeps HCV RNA from accumulating in the liver in the first place.
Locked nucleic acid-based, RNA-targeting drugs may help overcome some of the limitations of earlier antisense and siRNA experimental technologies. They're shorter in length than typical antisense drugs, and they have greater affinity for the target disease. These factors mean more potent drugs that are well-tolerated and have the potential for oral delivery.
Miravirsen (SPC3649)--the first microRNA-targeted therapy to enter human trials--has shown promise in Phase IIa data, where viral loads in patients dropped significantly: Four of 9 patients at the highest dose had undetectable levels of HCV during the trial, and the drop in their viral load continued beyond the end of therapy. The company plans additional clinical trials in combination with other HCV treatments.
Santaris has a number of partnerships that use its LNA technology. In October 2011, Servier and miRagen inked a deal using miRagen's microRNA drugs that use LNA technology for cardiovascular diseases. That expanded miRagen's 2010 agreement with Santaris.
At the beginning of 2011, Pfizer agreed to pay up to $614 million to Santaris, upsizing a deal that was originally between Wyeth and Santaris and covering up to 10 targets.