Sanofi has signed a deal worth up to $200m with US company MyoKardia for first-in-class therapies for cardiomyopathies, a group of diseases affecting the heart muscle.
Cardiomyopathies are typically caused by genetic mutations found within families that affect the proper functioning of the heart – for example by affecting its size or shape or its ability to conduct electrical impulses – although similar symptoms can be caused by stress, infections or as a side effect from drug treatment.
Sanofi has licensing rights to three of MyoKardia’s programmes in the two most common inherited cardiomyopathies, including two projects looking at hypertrophic cardiomyopathy (HCM) and one focusing on dilated cardiomyopathy (DCM).
HCM affects one in 500 people and is characterised by a thickening of the heart muscle, while in DCM – which is more rare with an incidence of around one in 2,500 – the heart muscle gets stretched and thin.
Both types lead to shortness of breath, chest pain and palpitations, secondary symptoms which can generally be managed by treatment and allow patients to lead a normal life. In a minority, however, the heart changes can lead to arrhythmia and sudden cardiac death.
MyoKardia’s approach targets the genetic mutations that are the underlying causes of cardiomyopathy, addressing the fundamental mechanisms of the disease. The company development programmes are being overseen by former AstraZeneca (AZ) clinical development lead Jonathon Fox, who joined the US firm as chief medical officer last year.
Sanofi has already paid $45m to MyoKardia in licensing fees and an equity investment, with the remainder due through 2018 in the form of milestone payments, additional equity investments and R&D services.
The companies will split development costs on the two HCM programmes after proof of principle has been demonstrated in patients, while Sanofi will take full responsibility for the development costs of the DCM programme.
Elias Zerhouni, president of global R&D at Sanofi, said the collaboration combines “the unique expertise in rare and cardiovascular diseases of our top scientists with that of the best innovators in the world…to achieve real breakthroughs in medicine.”
The deal with MyoKardia has been set up under Sanofi’s Sunrise initiative, which focuses on investing in very early-stage external research programmes. MyoKardia is the second deal announced by Sanofi Sunrise, which is also supporting natural product specialist Warp Drive Bio as part of a $225m deal announced in 2012.