Sanofi, MyoKardia Sign $200 Million R&D Pact for Cardio DrugsBy
Sanofi and MyoKardia, Inc., a privately held company developing precision therapies for genetic heart disease, have formed a worldwide collaboration to discover and develop targeted therapeutics for heritable heart diseases known as cardiomyopathies, the most common forms of heart-muscle disease. The collaboration builds upon MyoKardia's 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 development and commercialization Within the collaboration, MyoKardia will drive research and worldwide development activities through early human efficacy studies. Thereafter, MyoKardia will lead worldwide development and US 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-US regulatory and commercial activities to the two HCM programs, where it has ex-US commercialization rights. Sanofi also has the option to co-promote in the US 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 US.
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 US. HCM is believed to be the most common heritable cardiovascular disease, thought to affect one in 500 people in the general population.