Les Laboratories Servier, miRagen sign new agreement

SURESNES, France, & BOULDER, Colo.— Les LaboratoriesServier and miRagen Therapeutics, Inc. have announced the formation of anagreement to advance the research, development and commercialization of threedrug candidates, including two of miRagen's lead programs (miR-208 andmiR-15/195) and a third yet-to-be-identified target, for cardiovasculardisease. As a result of the agreement, Servier grants worldwide rights,excluding the United States and Japan.

"Our agreement with Servier not only provides validation ofour lead programs in cardiac disease, but further underscores the potential ofour innovative technology platform to deliver compelling drug candidates,"William S. Marshall, Ph.D., President and Chief Executive Officer of Miragen,said in a press release. "We are delighted to partner with Servier, whosedemonstrated leadership and expertise in the development of cardiovasculardrugs are truly impressive. By combining our strengths, we hope to translatethe potential of microRNA targeting into life-changing medicines for patientsin need."

Per the agreement, Servier will pay miRagen up to $45million in total upfront, research support and near-term milestone paymentsover the course of the next three years, and miRagen will also be eligible toreceive royalties on sales, dependent upon the successful outcome of thecollaboration. Factoring in additional clinical and commercial milestones andclinical development support for the successful development of the threecompounds, the deal could be valued at approximately $1 billion.

The twocompanies will collaborate on the research and development efforts, and Servierwill take responsibility for all costs related to the global development,regulatory approval and commercialization of the candidates worldwide, with theexclusion of the U.S. and Japanese markets. miRagen will retain rights in theUnited States and Japan, as well as the option to co-sponsor any Phase IIIprograms should miRagen, singularly or in partnership with another company,seek marketing approvals for any of the three targets in those two countries.

"We are very pleased with this new partnership, whichdemonstrates once again our ability to explore truly innovative treatments forpatients suffering from cardiovascular diseases. Indeed, these diseases stillrepresent the number one cause of mortality in most of the world," EmmanuelCanet, M.D., Ph.D., Head of R&D at Servier, noted in a press release.

miRagen's two lead programs both have a cardiovascularorientation. miR-208 has been shown to play an important role in thepathogenesis and progression of heart failure, and research shows thattherapeutic inhibition of it might improve cardiac function and survival ratesin events of heart failure. Chemically synthesized inhibitors of miR-208 haveproven to be capable of suppressing pathological cardiac remodeling in modelsof high blood pressure-induced heart failure, while also enhancing cardiacfunction and survival. Research on the company's other lead program,miR-15/195, has shown that inhibition of miR-15 might stimulatecardiomyogenesis, the process by which new heart muscle cells are formed.Inhibition of miR-15 can also spare cardiomyocytes from death during myocardialinfarction.

The two lead programs use the Locked Nucleic Acid (LNA) DrugPlatform of Santaris Pharma A/S, which miRagen licensed the rights to in June2010, to identify and sleect drug candidates against miRagen's proprietarymicroRNA targets for the treatment of cardiovascular disease. The agreement hasbeen expanded to allow miRagen to develop additional targets and give Servieraccess to the LNA technology as well.

"We are pleased to expand our alliance with Miragen toinclude additional cardiovascular targets and provide access to SantarisPharma's LNA technology in order to help Servier and Miragen to developLNA-based microRNA-targeted drugs for the treatment of cardiovascular disease,"Søren Tulstrup, President and CEO of Santaris Pharma A/S, said in a pressrelease. "This agreement further validates that Santaris Pharma's LNA DrugPlatform is the technology-of-choice for developing RNA-targeted medicines."

SOURCE: miRagen Therapeutics, Inc. press release