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Regulus, GSK Ink Second miRNA Rx Collaboration


Regulus Therapeutics said this week that it has formed a new collaboration with British drugs giant GlaxoSmithKline to develop drugs targeting microRNA-122, with hepatitis C infection as the lead indication for the effort.

Under the deal, Regulus will receive upfront payments and early-stage milestones, with the potential to earn more than $150 million, as well as tiered royalties up to double digits on the worldwide sale of products resulting from the alliance.

Additional terms were not disclosed.

Regulus said that it expects to have identified a miR-122-targeting clinical candidate in the second half of the year, and file an investigational new drug application in 2011.

MiR-122 is the most abundantly expressed miRNA in the liver, and has been linked to cholesterol regulation and lipid metabolism, as well as HCV replication. A number of other companies are looking to it as a therapeutic target, most notably Santaris Pharma, which recently completed a phase I study using a locked nucleic acid-based drug called SPC3649 to down-regulate the miRNA as a treatment for HCV (see RNAi News, 5/7/2009).

Notably, GlaxoSmithKline had an option to SPC3649, but let it expire last year (see RNAi News, 12/3/2009). Now, it seems, the big pharma had interest in the target, but opted to pursue it through an already-established partnership.

Regulus and GlaxoSmithKline have been working together on miRNA therapeutics since 2008, when they struck a deal in inflammatory diseases such as rheumatoid arthritis and inflammatory bowel disease (see RNAi News, 4/17/2008).

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