Sanofi and MyoKardia Inc., a privately-held company leading the development of precision therapies for genetic heart disease, announced a worldwide collaboration 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 function.

 

The collaboration, representing one of the largest research and development commitments to genetic forms of cardiomyopathy, encompasses three MyoKardia programs. Two of these programs are focused on hypertrophic cardiomyopathy (HCM) and the other is focused on dilated cardiomyopathy (DCM). The collaboration provides up to $200 million in equity investments, milestone payments and research and development services through 2018, of which $45 million has already been received in an upfront licensing fee and an initial equity investment. In addition, 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.

 

“This collaboration illustrates Sanofi’s research and development philosophy for Sunrise projects. It combines in a meaningful way the unique expertise in rare and cardiovascular diseases of our top scientists with that of the best innovators in the world, like MyoKardia’s founders and scientists, to achieve real breakthroughs in medicine,” said Dr. Elias Zerhouni, president of Global R&D for Sanofi.

 

“MyoKardia’s research represents the first hope for targeted treatments that address the primary cause of each patient’s disease,” said Tassos Gianakakos, chief executive officer, 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.”

 

Within the collaboration, 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. 

 

“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, vice president of Global R&D and head of Sunrise. “This opportunity allows us to advance our collective knowledge and bench strength in this exciting and emerging field. This creative collaboration truly demonstrates Sanofi’s open innovation model.”

 

Date: September 17, 2014

Source: Sanofi