Sanofi and MyoKardia Announce Groundbreaking Collaboration to Develop Targeted Therapies for Patients with Genetic Heart Disease

Up to $200 Million Collaboration to Support Pioneering Science and New Treatments

Paris and South San Francisco, CA - Sanofi and MyoKardia, Inc., a privately-held company leading the development of precision therapies for genetic heart disease, announced today 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 contraction.

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 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 2/3 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 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.”

About Genetic Cardiomyopathies
HCM and DCM are types of heritable heart diseases that are caused by mutations in the genes of the proteins that are primarily responsible for the contraction of the heart muscle. HCM is the leading cause of sudden cardiac death in young adults, and DCM, the leading genetic illness requiring heart transplantation, together are believed to affect nearly 800,000 children and adults in the U.S. alone. Available treatments for patients suffering from these forms of heart disease have been approved for other illnesses and only treat the symptoms of their disease. As their disease progresses, it can require invasive procedures including heart transplantation, and can lead to sudden cardiac death and complications associated with heart failure, including stroke.

HCM is believed to be the most common heritable cardiovascular disease, thought to affect one in 500 people in the general population. HCM is characterized by an abnormal thickening and stiffening of the walls of the heart’s main pumping chamber, the left ventricle. Disease-causing HCM mutations make it difficult for the heart to relax and refill with fresh blood between beats, causing shortness of breath and sometimes dizziness, fainting or chest pain. About one-fourth of HCM patients experience an abnormal heart rhythm called atrial fibrillation that profoundly increases the risk of potentially debilitating or even fatal stroke.

Genetically-defined DCM, estimated to occur in one in 2,500 people in the general population, is a condition in which the heart becomes enlarged and weakened, and is unable to pump enough blood to the rest of the body. DCM is also known to be associated with harmful blood clots, which can cause strokes, and with irregular and harmful heartbeats and sudden cardiac death. More than 20 percent of heart transplantations are performed every year for patients with genetic DCM.

Source: Sanofi