Safety qualms in MyoKardia mid-stage study cause investors to pause
Ahead of the pivotal data readout of MyoKardia’s lead drug, mavacamten, in patients with obstructive hypertrophic cardiomyopathy (HCM), investors took issue with the drug’s safety profile in a separate Phase II non-obstructive HCM study.
Mavacamten is expected to break new ground in heart disease — a field monopolized by large pharmaceutical companies largely due to the long, arduous and expensive trials that are commonplace in heart drug development. Unlike other developers focusing on common heart disorders, MyoKardia’s treatment is also targeting a so-far untapped disease — hypertrophic cardiomyopathy — a genetic condition in which heart muscle thickens, making it harder for the organ to pump blood to the rest of the body.
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