“We are grateful for Sanofi’s support over the past four years in what was a highly productive relationship. Since entering into the collaboration, MyoKardia has become a leading research and development organization with a robust pipeline of targeted cardiovascular therapeutics,” said Tassos Gianakakos, MyoKardia’s chief executive officer. “Regaining worldwide rights enables us to capture the full value of the data being generated in the next 12-24 months as we prepare for the potential registration of mavacamten in obstructive hypertrophic cardiomyopathy and obtain proof-of-concept for MYK-491 in patients with dilated cardiomyopathy. Importantly, consolidated control over our entire portfolio allows us to make decisions about how we advance each of our therapeutic candidates in alignment with our precision medicine approach.”

MyoKardia and Sanofi initiated their collaboration in 2014 to advance up to three programs through discovery and into clinical development for the treatment of hypertrophic cardiomyopathy (HCM) and dilated cardiomyopathy (DCM). Over the course of the collaboration, MyoKardia received approximately $230 million in funding from Sanofi and has advanced mavacamten from preclinical development into a late-stage pivotal study for the treatment of HCM, and MYK-491 from discovery to a Phase 2 proof-of-concept study in patients with DCM. During the period leading up to the end of the research term, it was important for MyoKardia to maintain 100 percent of the U.S. commercial rights for mavacamten in HCM as well as additional rights in expanded indications. Sanofi subsequently provided MyoKardia with notification of its decision to conclude the collaboration in conjunction with the end of the research term.

MyoKardia is positioned to achieve several significant clinical milestones over the next 12 to 24 months and reaffirmed timing for anticipated data read-outs:

6-month and 12-month data for mavacamten in obstructive HCM (oHCM) from the ongoing PIONEER open-label extension study
Data from the Phase 2 MAVERICK study in non-obstructive HCM in the second half of this year
Topline data from the Phase 3 EXPLORER-HCM clinical trial of mavacamten in obstructive HCM (oHCM) in the second half of 2020
Phase 2a proof-of-concept data for MYK-491 in DCM before the end of the year

The company anticipates that current cash, cash equivalents and investments are sufficient to fund operations past the read-out of EXPLORER-HCM data in the second half of 2020. This reflects an anticipated increase in R&D expenses due to the termination of the Sanofi collaboration agreement. As of September 30, 2018, MyoKardia had cash, cash equivalents and investments totaling $412 million. MyoKardia will report fourth quarter and year-end 2018 financial results in late February of 2019.