MyoKardia is developing precision therapies for genetic heart disease, concentrating on hypertrophic and dilated cardiomyopathies (HCM and DCM). Its lead candidate, MYK-461, is in Phase 1 clinical trials and targets a common molecular mechanism driving HCM by correcting overcontraction and restoring cardiac contractility to normal levels. The company plans to use genetic screening to identify patients whose disease is caused by the specific mechanism targeted by MYK-461. Proceeds from the financing will be used to advance and expand MyoKardia’s portfolio of potential therapies for HCM and DCM. MyoKardia launched in 2012 by Third Rock Ventures and has helped organize the Sarcomeric Human Cardiomyopathy Registry (SHaRe) to support its precision‑medicine efforts. The company emphasizes combining cardiovascular genetics with heart muscle biochemistry to classify heritable cardiovascular diseases and match therapies to patients. MyoKardia is developing a pipeline of novel small-molecule therapeutics that target genetic cardiomyopathies, initially focusing on hypertrophic (HCM) and dilated cardiomyopathy (DCM). Its proprietary drug discovery platform combines cardiovascular genomics and muscle biology to enable mutation-specific sarcomeric allosteric modulators that rebalance contractility. The company aims to stop and potentially reverse disease progression and to extend this genetically targeted approach to a broader spectrum of cardiovascular disease, including heart failure. MyoKardia leverages expertise in sarcomere genetics, in‑vivo and in‑vitro disease models, next‑generation biochemical and biophysical assays, and medicinal chemistry to advance multiple programs. The company was launched in 2012 with funding from Third Rock Ventures and is founded by James Spudich, Leslie Leinwand, Christine Seidman, and Jonathan Seidman, with Charles Homcy serving as interim CEO. It announced a $38 million Series A financing to support progression of its preclinical programs toward clinical development.

Total raised

$84M

Funding rounds

2

Latest round

Series B

Latest activity

Apr 2015