- After four years of working together, MyoKardia will begin 2019 without its long-time pharma partner Sanofi, which said it would not extend a licensing and collaboration deal and would kick back ex-U.S. rights on three heart failure drugs to the biotech.
- MyoKardia CEO Tassos Gianakakos said on a Wednesday investor call that the termination was unrelated to any clinical data or safety concerns Sanofi had. Rather, the big pharma sought to expand the deal to include U.S. commercial rights for its leading therapeutic candidate, mavacamten, which MyoKardia was unwilling to do.
- MyoKardia's stock was down more than 14% Wednesday morning. Sanofi has a sizable equity stake in MyoKardia, which the company will look to sell down over time, Gianakakos said. The pharmaceutical giant owned about four million shares as of March 31, 2018, according to MyoKardia's latest proxy statement, which represented about 11% of the company's total shares.
MyoKardia, a South San Francisco, California-based biotech, grew up with Sanofi by its side. The French pharma fed $230 million into MyoKardia over the course of their deal.
Four academic and hospital leaders in muscle biology and cardiovascular genetics formed the company in September 2012. Two years later, in August 2014, Sanofi's subsidiary Aventis struck the collaboration deal that has now come to an end.
When that deal started, MyoKardia was a private company that had yet to start a Phase 1 study. Four years later, the company is publicly traded, mavacamten has advanced and is now in the middle of a late-stage pivotal trial in hypertrophic cardiomyopathy. MYK-491, another candidate, has moved from discovery to an ongoing Phase 2a proof-of-concept study.
The third drug in the collaboration, HCM-2, remains preclinical and aims to lower cardiac muscle contractility in hypertrophic cardiomyopathy patients.
While MyoKardia's leader said the deal's termination resulted over an unwillingness for the company to divvy up U.S. rights on mavacamten, Sanofi portrayed it as a matter of resource allocation.
"This was a difficult but necessary decision given the robustness of Sanofi's late-stage pipeline and our need to focus our resources on our highest priority programs," a Sanofi spokesperson said in an emailed statement to BioPharma Dive.
Without Sanofi, MyoKardia will attempt to go through the clinic to approval and commercialization on its own.
MyoKardia's R&D costs will increase with Sanofi out of the picture. The big pharma was funding 50% of mavacamten development costs and 100% of MYK-491. MyoKardia listed $412 million in cash and equivalents at the end of September, which company leaders expect will be enough to carry it into 2020 when accounting for increased R&D spending.
Gianakakos highlighted multiple expected clinical milestones over the next two years.
If all goes to plan, mavacamten will have readouts for treating obstructive hypertrophic cardiomyopathy from an open-label extension study in the first quarter of 2019, and Phase 3 topline data in the second half of 2020. MYK-491 will also read out proof-of-concept data in dilated cardiomyopathy in the fourth quarter of 2019.
Cardiomyopathies are a leading cause of heart failure. While mavacamten is further along in development than MYK-491, the latter may provide greater revenue potential, as dilated cardiomyopathy has a larger patient population than hypertrophic cardiomytopathy.
MyoKardia will report fourth quarter results in late February.