Sanofi and MyoKardia, a South San Francisco-based, privately held company developing precision therapies for genetic heart disease, are collaborating to discover and develop first-of-its-kind targeted therapeutics for heritable heart diseases known as cardiomyopathies, the most common forms of heart muscle disease. The collaboration builds upon MyoKardia's pioneering science, which hopes to correct the disruptive effects that disease mutations have on heart muscle contraction.

The collaboration encompasses three MyoKardia programs, of which two are focused on hypertrophic cardiomyopathy (HCM) and the other on dilated cardiomyopathy (DCM). The collaboration provides up to $200 million in equity investments, milestone payments and R&D services through 2018, of which $45 million already has been received in an upfront licensing fee and an initial equity investment.

Sanofi and MyoKardia will equally share development costs on the HCM programs following initial demonstration of efficacy in patients, with Sanofi fully covering the development costs of the DCM program.  

The collaboration is an outgrowth of Sanofi's Sunrise initiative, a strategic partnership model that seeks to invest in early stage opportunities that align with Sanofi's expert development and commercialization abilities. The commitment of resources from Sanofi will accelerate and broaden MyoKardia's basic disease research and will support the shared commitment of bringing desperately needed therapies to patients.

"MyoKardia's research represents the first hope for targeted treatments that address the primary cause of each patient's disease," said Tassos Gianakakos, CEO, MyoKardia. "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. The collaboration extends MyoKardia's competitive advantage, allowing advancement of a broad portfolio of important treatments for patients." 

MyoKardia will drive research and worldwide development activities through early human efficacy studies. Thereafter, MyoKardia will lead worldwide development and U.S. commercial activities for the two HCM programs, where it has retained product rights, and Sanofi will lead global development and commercial activities for DCM where it has obtained worldwide rights, and ex-U.S. regulatory and commercial activities to the two HCM programs where it has ex-U.S. commercialization rights.

Sanofi also has the option to co-promote in the U.S. for potential expanded cardiovascular diseases outside of the genetically targeted indications for either of the HCM programs, with MyoKardia having the option to co-promote the DCM program in the U.S.