Sanofi And MyoKardia Partner To Target Genetic Heart Disease

Sanofi announced its global R&D collaboration with MyoKardia to discover and develop therapeutics for heritable heart diseases known as cardiomyopathies.

The partnership will leverage MyoKardia’s technology that aims to correct the disruptive effects of disease mutations on heart muscle contraction. MyoKardia currently has three programs that address genetic forms of cardiomyopathy. Two of these programs focus on hypertrophic cardiomyopathy (HCM), while one tackles dilated cardiomyopathy (DCM). The agreement includes up to $200 million in equity investments and milestone payments, of which $45 million has already been paid, as well as R&D services through 2018. The partners will spread the load of development costs equally after an initial demonstration of efficacy in patients. Sanofi will fund the development costs of the DCM program.

Tassos Gianakakos, CEO of MyoKardia, said, “MyoKardia's research represents the first hope for targeted treatments that address the primary cause of each patient's disease. 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 will lead the research and global development program through early human efficacy studies. The company will also drive development and commercialization of the two HCM programs in the U.S., while Sanofi will be responsible for regulatory activities outside the U.S. Finally, Sanofi will hold the option to co-promote the programs for potential expanded cardiovascular diseases in the U.S. MyoKardia will be given a similar option for the DCM program in the U.S.

“We are very excited to embark on this deep collaboration with MyoKardia, to identify first-in-class, life-changing therapies for HCM and DCM patients,” said Dr. Kathy Bowdish, VP of Global R&D and Head of Sanofi’s Sunrise initiative.

Both HCM and DCM are caused by genetic mutations in proteins that are mainly responsible for heart muscle contraction. HCM is the most common cause of sudden cardiac death in young adults. DCM is the leading genetic illness that requires heart transplantation. Together, HCM and DCM are thought to afflict almost 800,000 children and adults in the U.S.