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United States

Profile: MyoKardia Inc (MYOK.O)

MYOK.O on Nasdaq

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17 May 2019
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MyoKardia, Inc., incorporated on June 8, 2012, is a clinical-stage biopharmaceutical company. The Company focuses on the treatment of heritable cardiomyopathies, a group of rare, genetically-driven forms of heart failure that result from biomechanical defects in cardiac muscle contraction. The Company is engaged in the business of developing and commercializing therapeutics. The Company has used its precision medicine platform to generate an initial pipeline of four therapeutic programs for the chronic treatment of two forms of heritable cardiomyopathy-hypertrophic cardiomyopathy (HCM), and dilated cardiomyopathy (DCM). Additionally, the Company has three preclinical programs, HCM-2, DCM-2 and LUS-1.

The Company's lead product candidate, MYK-461, is an orally-administered small molecule designed to reduce excessive cardiac muscle contractility leading to HCM. The Company is enrolling patients in a Phase II clinical trial of MYK-461, which is referred as PIONEER-HCM. In three Phase 1 clinical trials, the Company has observed favorable tolerability of single and multiple doses of MYK-461 in both healthy volunteers and HCM patients. Its MYK-491 is an orally-administered small molecule designed to treat genetic DCM by restoring normal contractility in the diseased DCM heart. The Company is in a Phase 1 clinical trial of MYK-491, its product candidate for the treatment of DCM.

The Company's precision medicine platform incorporates disease research, drug discovery and clinical expertise in a self-reinforcing cycle that enables the iterative generation of new disease targets and clinical development candidates designed to establish early clinical proof-of-concept. HCM-2 is intended to reduce cardiac muscle contractility to normal levels in HCM patients through a different mechanism than that of MYK-461. DCM-2 is intended to increase cardiac muscle contractility to normal levels in genetic DCM patients through a different mechanism than that of MYK-491. LUS-1 is intended to counteract a muscle disruption that results in impaired relaxation of the heart, a biomechanical defect found in specific HCM and genetic DCM patient subgroups, as well as in other less common heritable cardiomyopathies.

The Company competes with Amgen Inc., Bayer AG, Bristol-Myers Squibb Company, C.H. Boehringer Sohn AG & Co. KG, Gilead Sciences, Inc., Novartis AG, Takeda Pharmaceutical Company Limited, Gilead Sciences, Inc., Heart Metabolics Limited, Array BioPharma Inc., Kasiak Research Pvt Ltd., Vericel Corp. and Zensun (Shanghai) Sci. &Tech. Co., Ltd.

Company Address

MyoKardia Inc

333 Allerton Ave
SOUTH SAN FRANCISCO   CA   94080-4816
P: +1650.7410900
F: +1302.6365454

Company Web Links