molecules of the year

Myokardia/BMS cardiac myosin inhibitor

FIC, oral, QD, cardiac myosin inhibitor

FDA-approved for obstructive HCM

opt. of HTS hit for sarcomere activity inhibition

mavacamten (MYK461, CAMZYOS)

Myokardia, Brisbane, CA; Bristol Myers Squibb, New York, NY

2 mins read

Mavacamten is a first-in-class oral, selective, allosteric cardiac myosin inhibitor, discovered by Myokardia and acquired by BMS in 2020 in a whopping $13.1B transaction. In 2022, it was approved by the FDA to treat obstructive hypertrophic cardiomyopathy (HCM) in adults and, thanks to its use, led to improvements in functional capacity and symptoms. Before mavacamten, there were no disease-modifying treatments for HCM; today it has been described as life-changing for patients. The science behind mavacamten traces its roots to Jim Spudich’s lab at Stanford, which studied how motor proteins, myosin and actin, work and developed activity assays. In 1998, Spudich helped found Cytokinetics, today a $4B public company that has had several interesting programs focused on motor-protein modulation, including myosin…

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