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Sanofi, MyoKardia partner to develop new therapies for genetic heart disease

Sanofi has entered into collaboration with US-based MyoKardia to discover and develop new therapies for patients with heritable heart diseases called cardiomyopathies, the most common forms of heart muscle disease.

The deal will see Sanofi provide about $200m in equity investments, milestone payments and research and development services over the next four years.

Sanofi has already paid MyoKardia a $45m up front, which includes both an unspecified licensing fee and an equity investment.

The collaboration is centered on three programs carried out by MyoKardia, of which two are focused potential treatments for hypertrophic cardiomyopathy (HCM), while the other on dilated cardiomyopathy (DCM).

Both 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.

Additionally, the two firms 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.

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

"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," Gianakakos said.

"The collaboration extends MyoKardia’s competitive advantage, allowing advancement of a broad portfolio of important treatments for patients."

Under the deal, MyoKardia will be responsible for the research and worldwide development activities through early human efficacy studies as well as lead worldwide development and US commercial activities for the two HCM programs, where it has retained product rights.

Sanofi will lead global development and commercial activities for DCM where it has received worldwide rights, and ex-US regulatory and commercial activities to the two HCM programs where it has ex-US commercialization rights.