MyoKardia Goes Solo as Sanofi Ends Precision Heart Drug Deal

Xconomy San Francisco — 

MyoKardia rode a partnership with Sanofi to the public markets a few years ago. But the two companies are cutting ties today, with MyoKardia regaining full rights to its drug programs and assuming all the upside—and risk—of their success or failure.

MyoKardia (NASDAQ: MYOK) said the deal, which started in September 2014, will wrap up in April. At that point, the South San Francisco, CA-based firm will fully control all of its experimental drugs, including the heart disease drugs mavacamten and MYK-491—its only programs in human clinical testing. Its share price is down about 7 percent, to $45.39, this morning.

Sanofi struck the deal with MyoKardia to co-develop small molecule pills for people with common genetic mutations that trigger certain heart diseases. The French company has paid MyoKardia some $230 million over the course of the alliance. That cash has helped MyoKardia get mavacamten and MYK-491 into late- and mid-stage testing, respectively, and bring other drugs into its pipeline as well.

In return, Sanofi got equity in MyoKardia, rights in Europe to a group of experimental drugs (including mavacamten), and U.S. rights to MYK-491, which is being developed for dilated cardiomyopathy (DCM), the most common form of cardiomyopathy. Sanofi held 11.22 percent of MyoKardia when it went public at $10 per share in October 2015 and remained the company’s second largest shareholder, with a similar-sized stake, as of a proxy filing in April 2018. Shares traded at close to $49 apiece before today’s news.

“This has been a very successful collaboration,” CEO Tassos Gianakakos said on a conference call Wednesday morning.

Gianakakos said the move wasn’t related to any problems with the company’s clinical data. Rather, MyoKardia balked at expanding the Sanofi deal. It didn’t want to give up U.S. rights to mavacamten, and it didn’t want to launch more trials to test its drugs in other types of diseases. “Neither of these options make sense for our business at this time,” Gianakakos said on the call. Sanofi then notified MyoKardia of plans to end the deal. The French pharma will maintain a “de minimis” royalty on U.S. sales of mavacamten going forward, MyoKardia chief business officer Jake Bauer said.

The news comes as MyoKardia prepares, over the next 12 to 24 months, to disclose its most significant data yet. Results from a Phase 3 study, EXPLORER, of mavacamten in hypertrophic cardiomyopathy—a genetic defect that leads to the thickening of heart tissue, irregular heartbeats, and, potentially sudden, fatal strokes in young adults—should come in the second half of 2020. Data from other studies of mavacamten and MYK-491 are expected before that.

MyoKardia has $412 million in cash on hand, enough to get through the EXPLORER data, according to Gianakakos. He added that MyoKardia isn’t looking for a cash grab, like a new partnership, elsewhere. It plans to own, and sell, its drugs. “We are building a company here for the long haul,” he said.

Here’s more on MyoKardia, which Third Rock Ventures started up in 2012.