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MyoKardia, Sanofi Ink $200M+ Deal for Targeted Heart Drugs

Xconomy San Francisco — 

Third Rock Ventures started up MyoKardia in 2012 around the idea of making genetically targeted drugs for certain forms of heart disease. Two years and $38 million in venture funding into the effort, French pharmaceutical giant Sanofi is buying into the concept.

South San Francisco, CA-based MyoKardia announced this morning that it’s inked a collaboration with Sanofi to co-develop targeted treatments for inheritable forms of cardiomyopathy. Sanofi has already paid MyoKardia a $45 million up front, consisting of both an unspecified licensing fee and an equity investment. Sanofi could provide another $200 million in milestone payments, R&D services, and equity funding over the next four years.

The deal is centered on three programs. Two are potential treatments for hypertrophic cardiomyopathy (HCM), a genetic defect that leads to the thickening of heart tissue, irregular heartbeats, and, potentially sudden, fatal strokes in young adults. Boston Celtics star Reggie Lewis died during a team shootaround more than 20 years ago from HCM, as did Loyola Marymount college basketball standout Hank Gathers, who collapsed during a conference championship game in 1990.

MyoKardia and Sanofi will also develop a treatment for dilated cardiomyopathy (DCM), the most common form of cardiomyopathy in which the heart muscle becomes weak and can’t pump enough blood. Combined, HCM and DCM affect about 800,000 people in the U.S. alone.

MyoKardia will handle the early R&D for all three programs. If the HCM drug candidates show efficacy in human patients, both companies will share the development costs. The startup has kept U.S. rights to those drugs, with Sanofi getting rights to the drugs elsewhere. Sanofi is getting full rights to the DCM candidate, and will foot the bill for its clinical development. Sanofi can also opt in to a profit split in the U.S. should the drug be used for other cardiovascular diseases, and MyoKardia can do the same for the DCM program.

MyoKardia was formed in 2012 with the help of a $38 million Series A from Third Rock—a biotech venture firm with offices in Boston and San Francisco—and a team of academic scientists who study the genetics of cardiovascular disease and muscle biology, like James Spudich of Stanford University, Leslie Leinwand of the University of Colorado, and Christine Seidman and Jonathan Seidman, at Harvard Medical School. Third Rock venture partner Charles Homcy initially led the company, before Tassos Gianakakos, a veteran of MAP Pharmaceuticals (bought by Allergan in January 2013) and founder of Codexis, was hired as CEO in October.

The basic idea is to develop small-molecule pills for certain segments of people with common genetic mutations that trigger certain heart diseases. Those drugs would be taken chronically to keep symptoms at bay. Homcy told Xconomy in 2012 that MyoKardia’s plan is to start out in well-defined genetic populations, and then expand into larger patient groups, and more complex and prevalent diseases like congestive heart failure, using genomic insights to try to streamline the development of its candidates.

“MyoKardia’s research represents the first hope for targeted treatments that address the primary cause of each patient’s disease,” Gianakakos said in a statement. “By genetically defining HCM and DCM into several underlying rare genetic diseases, MyoKardia’s candidate therapies have the potential to be developed far more efficiently than traditional cardiovascular drugs.”

MyoKardia, meanwhile, represents the second Third Rock startup that Sanofi has partnered with over the past few years. The pharma giant also cut a deal with Cambridge, MA-based Warp Drive Bio in 2012, and could acquire the startup if it hits certain milestones.

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